Patients with a history of hepatic sinusoid obstructive syndrome (veno-occlusive disease) within the prior 3 months
History of malabsorption syndrome or other condition that would interfere with enteral absorption
Known history of Gilbert's syndrome
Presence of GVHD overlap syndrome.
Myelodysplastic syndrome with isolated del(5q) (5q-syndrome)
Myelodysplastic syndrome (MDS), unclassifiable
Abnormal glucuronidation of bilirubin, known Gilbert's syndrome
History of reversible posterior leukoencephalopathy syndrome (RPLS)
Trisomy 21 (Down syndrome)
Malabsorption syndrome or other condition that would interfere with enteral absorption
Patients known to have one of the following concomitant genetic syndromes: Down syndrome, Bloom syndrome, ataxia-telangiectasia, Fanconi anemia, Kostmann syndrome, Shwachman syndrome or any other known bone marrow failure syndrome
Patients with Down syndrome
Patients with Down syndrome are not eligible
Down syndrome
Patients with down syndrome are excluded from this study
Patients with known symptomatic Fanconi anemia (FA), ataxia-telangiectasia (A-T) syndrome, Bloom syndrome (BS) and Nijmegen breakage syndrome (NBS) are not eligible (asymptomatic carriers are acceptable)
Subject has malabsorption syndrome or other condition which may affect an enteral route of administration
Malabsorption syndrome or other condition that would interfere with enteral absorption
Please note: patients who do not meet the above criteria because of Gilbert’s syndrome are still eligible
History of sarcoidosis syndrome
Difficulty with swallowing or an active malabsorption syndrome.
Patients must not have evidence or history of veno-occlusive disease or sinusoidal obstruction syndrome
History of known radiation sensitivity syndrome
Subjects with solitary plasmacytoma; active or history of plasma cell leukemia (PCL); Waldenstrom's macroglobulinemia; Polyneuropathy, Organomegaly, Endocrinopathy, Monoclonal protein, Skin changes (POEMS) syndrome; or symptomatic amyloidosis
Malabsorption syndrome or other conditions that preclude enteral route of administration
Has active hemolytic anemia, plasma cell leukemia, Waldenstrom's macroglobulinemia, POEMS syndrome, amyloidosis, significant autoimmune, CNS or other malignant disease
Bilirubin ? 1.5 x ULN (unless considered due to Gilbert's syndrome or hemolysis)
POEMS syndrome
Diagnosis of autoimmune disorder, including RA, SLE, or Sjogren's syndrome
Malabsorption syndrome or other condition that precludes enteral route of administration
Subjects with Gilbert's syndrome;
Myelodysplastic or myeloproliferative syndrome other than MDS.
Acute coronary syndrome within 6 months prior to starting treatment
POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, and skin changes)
Participants with Gilbert's syndrome will be eligible for the study
Mycosis fungoide/Sezary syndrome
History of Steven's Johnson's syndrome or toxic epidermal necrolysis syndrome.
History of nephrotic syndrome
Diagnosis of any of the following cancers:\r\n* POEMS syndrome (plasma cell dyscrasia with polyneuropathy, organomegaly, endocrinopathy, monoclonal protein [M-protein] and skin changes)\r\n* Non-secretory myeloma (no measurable protein on serum free lite assay)\r\n* Plasma cell leukemia\r\n* Human T-cell lymphotrophic virus (HTLV)1/HTLV2 positive
Patients with nevoid BCC syndrome (Gorlin syndrome) should not enroll in this study
Acute coronary syndrome
POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, and skin changes)
POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, and skin changes)
Myelodysplastic Syndrome (MDS)
Have a history of Gilbert's syndrome.
Pre-existing renal disease including glomerulonephritis, nephritic syndrome, Fanconi Syndrome or renal tubular acidosis.
Any history of serotonin syndrome (SS) after receiving 1 or more serotonergic drugs
Known history of Gilbert's syndrome.
Has polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, and skin changes (POEMS) syndrome, monoclonal gammopathy of unknown significance, smoldering myeloma, solitary plasmacytoma, amyloidosis, Waldenstrom macroglobinemia or immunoglobulin M (IgM) myeloma, or lymphoplasmacytic lymphoma (LPL).
Myelodysplastic syndrome
Patients with Down’s syndrome
ENROLLMENT TO THE DOSE ESCALATION, EXPANSION AND PART II: History of Gilbert’s syndrome
AML that progressed out of myelodysplastic syndrome (MDS) is eligible if the patient did not receive treatment directed at the MDS
Patients with 5q- syndrome should have failed to respond to or progressed on treatment with lenalidomide, where available and indicated
Patients with Down syndrome
History of hemolytic-uremic syndrome.
History of malabsorption syndrome or other condition that would interfere with enteral absorption
TBL < 2.5 mg/dL, except for participants with Gilbert's syndrome.
Malabsorption syndrome or other condition precluding enteral route of administration
Erythema multiforme, toxic epidermal necrolysis, or Stevens-Johnson syndrome
Known history of Gilbert's Syndrome.
Subjects with known POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, and skin changes) or light-chain (AL) amyloidosis.
Clonal cytogenetic abnormalities consistent with pre-myelodysplastic syndrome (pre-MDS) or MDS on marrow examination (e.g. Monosomy 7).
Myeloproliferative/myelodysplastic syndrome other than CMML. CMML with t(5;12) that have not yet received imatinib.
POEMS syndrome (plasma cell dyscrasia with polyneuropathy, organomegaly, endocrinopathy, monoclonal protein [protein] and skin changes)
Malabsorption syndrome or other conditions that would interfere with intestinal absorption
Any evidence of myelodysplastic syndrome or marrow chromosomal changes suggesting myelodysplasia (-7, -5 etc)
Diagnosis of Waldenstrom's macroglobulinemia, POEMS (polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin changes) syndrome, plasma cell leukemia, primary amyloidosis, myelodysplastic syndrome, or myeloproliferative syndrome.
History of sarcoidosis syndrome
Plasma cell dyscrasia with polyneuropathy, organomegaly, endocrinopathy, monoclonal protein (M-protein), and skin changes (POEMS) syndrome
History of known malabsorption syndrome or prior surgery(ies) that may lead to malabsorption
Malabsorption syndrome or other condition that precludes oral route of administration of lenalidomide.
History of multiple sclerosis or other demyelinating disease and/or Eaton-Lambert syndrome (para-neoplastic syndrome)
Diagnosis of Waldenstrom’s macroglobulinemia, POEMS (polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin changes) syndrome, plasma cell leukemia, myeloproliferative syndrome, or primary amyloidosis (with the exception of patients whose amyloidosis has been documented as a complication of MM, who will be evaluated on a case-by-case basis for trial participation)
POEMS syndrome (plasma cell dyscrasia with polyneuropathy, organomegaly, endocrinopathy, monoclonal protein and skin changes)
>= grade 3 neuropathy and/or polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, skin changes (POEMS syndrome)
Malabsorption syndrome or other condition that precludes enteral route of administration
Bilirubin ? 3.0 x ULN, unless due to Gilbert’s syndrome (unless considered due to leukemic organ involvement)
Subject has received treatment with a hypomethylating agent and/or other chemotherapeutic agent either conventional or experimental for myelodysplastic syndrome (MDS) or AML
Subject has a malabsorption syndrome or other condition that precludes enteral route of administration
Any history of serotonin syndrome after receiving serotonergic drugs
Documented history of carcinoid syndrome
Patients with Gilbert's syndrome will be eligible for the study; the diagnosis of Gilbert's syndrome is suspected in people who have persistent, slightly elevated levels of unconjugated bilirubin without any other apparent cause; a diagnosis of Gilbert's syndrome will be based on the exclusion of other diseases based on the following criteria:\r\n* Unconjugated hyperbilirubinemia noted on several occasions\r\n* No evidence of hemolysis (normal hemoglobin, reticulocyte count, and lactate dehydrogenase [LDH])\r\n* Normal liver function tests\r\n* Absence of other diseases associated with unconjugated hyperbilirubinemia
Subjects with history of or active symptoms of carcinoid syndrome
Any history of serotonin syndrome (SS) after receiving serotonergic drugs
Patients with a diagnosis of POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, and skin changes) or plasma cell leukemia (> 2.0 x 10^9/L circulating plasma cells by standard differential)
Bilirubin: =< 1.5 x ULN (except for subjects with Gilbert’s Syndrome or of non-hepatic cause)
Malabsorption syndrome or other condition that precludes enteral route of administration
Known reversible posterior leukoencephalopathy syndrome (RPLS)
History of concomitant genetic syndrome such as Fanconi anemia, Kostmann syndrome, Shwachman-Diamond syndrome or any other known bone marrow failure syndrome
History of multiple sclerosis or other demyelinating disease, Eaton-Lambert syndrome (para-neoplastic syndrome), history of hemorrhagic or ischemic stroke within the last 6 months, or alcoholic liver disease;
A hypomethylating agent, venetoclax and/or chemo therapeutic agent for Myelodysplastic syndrome (MDS).
Participant has a malabsorption syndrome or other condition that precludes enteral route of administration.
History of Gilbert’s syndrome
Direct bilirubin (bili) =< 40% if total bili > ULN in patients with Gilbert’s syndrome
Any history of serotonin syndrome (SS) after receiving serotonergic drugs
History of malabsorption syndrome or other condition that would interfere with enteral absorption
POEMS syndrome (plasma cell dyscrasia with polyneuropathy, organomegaly, endocrinopathy, monoclonal protein [M-protein] and skin changes)
Diagnosis of amyloidosis, polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin changes (POEMS), Waldenstrom’s macroglobulinemia
History of sarcoidosis syndrome
Patients with non-secretory multiple myeloma, active plasma cell leukemia, defined as either having 20% of peripheral white blood cells comprised of CD138+ plasma cells, or an absolute plasma cell count of 2 x 10^9/L, known amyloidosis, or known POEMS syndrome (plasma cell dyscrasia with polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, and skin changes)
Patients with respiratory distress syndrome
Has any history of serotonin syndrome after receiving 1 or more serotonergic drugs
Diagnosis of smoldering MM, Waldenström's macroglobulinemia, POEMS (polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin changes) syndrome, plasma cell leukemia, primary amyloidosis, myelodysplastic syndrome, or myeloproliferative syndrome.
Any other significant medical condition not under control, including any acute coronary syndrome within the past 6 months
Has history of reversible posterior leukoencephalopathy syndrome
Malabsorption syndrome or other condition that precludes enteral route of administration
Histologic diagnosis of Richter’s syndrome (RS)
History of sarcoidosis syndrome
Total bilirubin =< 1.5 X upper limit of normal (ULN), for patients with congenital unconjugated hyperbilirubinemia (Crigler-Najjar syndrome type 1 and 2, Gilbert syndrome) that transient hyperbilirubinemia can occur due to physiological condition, as long as there is clear documentation of diagnosis, allowed to be enrolled if direct (conjugated) bilirubin is 1.5 X ULN
• Bilirubin ? 1.5 times institutional ULN; does not apply to patients diagnosed with Gilbert's syndrome.
Participant has a malabsorption syndrome or other condition that precludes enteral route of administration.
Polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, and skin changes (POEMS) syndrome
GROUP 6: Progressive gastrointestinal disease as defined by all of the following items:\r\n* Disease duration of scleroderma =< 2 years.\r\n* Documented severe malabsorption syndrome requiring nutritional support; severe malabsorption syndrome is > 10% weight loss and on total parenteral nutrition (TPN) or enteral feedings\r\n* High score on distention/ bloating scale (>= 1.60 out of 3.00) on gastrointestinal (GI) questionnaire
>= grade 3 neuropathy and/or POEMS syndrome (plasma cell dyscrasia with polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, and skin changes)
Malabsorption syndrome or other condition that precludes enteral route of administration
Patients with Gilbert's syndrome or other heritable diseases of bile processing.
POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, and skin changes)
Has a paraneoplastic syndrome other than syndrome of inappropriate antidiuretic hormone secretion (SIADH) (hyponatremia)
Bilirubin: =< 1.5 x ULN (except for subjects with Gilbert’s syndrome or of non-hepatic origin)
POEMS syndrome requiring therapy, previously treated or untreated
Known drug-related, inherited, or acquired procoagulant disorder including prothrombin gene mutation 20210, antithrombin III deficiency, protein C deficiency, protein S deficiency and antiphospholipid syndrome but not including heterozygosity for the Factor V Leiden mutation or the presence of a lupus anticoagulant in the absence of other criteria for the antiphospholipid syndrome
Bilirubin =< 2 X ULN (3 X if known history of Gilbert's syndrome)
Clonal cytogenetic abnormalities consistent with pre-myelodysplastic syndrome (pre-MDS) or MDS on marrow examination (e.g. monosomy 7)
Known Gilbert’s syndrome
History of sarcoidosis syndrome
Patients with Gilbert’s syndrome will be eligible for the study; a diagnosis of Gilbert’s syndrome will be based on the exclusion of other diseases based on the following criteria:\r\n* Unconjugated hyperbilirubinemia noted on several occasions\r\n* No evidence of hemolysis (normal hemoglobin, reticulocyte count, and lactate dehydrogenase [LDH])\r\n* Normal liver function tests\r\n* Absence of other diseases associated with unconjugated hyperbilirubinemia
Malabsorption syndrome or other condition that precludes enteral route of administration
Patients with known Gilbert syndrome are not eligible
history of erythema multiforme, toxic epidermal necrolysis or Stevens-Johnson syndrome;
POEMS syndrome (plasma cell dyscrasia with polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, and skin changes)
Has active cytokine release syndrome from CTL019 infusion
Patients with known Gilbert's syndrome or reduced UGT1A1 activity.
Malabsorption syndrome or other condition that precludes enteral route of administration
Personal history of Gilbert’s syndrome
Subjects with known polyneuropathy-organomegaly-endocrinopathy-M protein-skin lesions (POEMS) syndrome, and subjects with myeloma and amyloidosis will be eligible if they have measurable disease
Malabsorption syndrome or other condition that would interfere with intestinal absorption are excluded
Down syndrome
Direct bilirubin must be < 2 mg/dL unless the elevation is known to be due to Gilbert's syndrome or acute GVHD (aGVHD) within 3 days of enrollment
History of primary amyloidosis, hyperviscosity or POEMS syndrome (plasma cell dyscrasia with polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, and skin changes)
Individuals with Down syndrome
Participants with malabsorption syndrome or any other condition that precludes enteral administration.
Malabsorption syndrome or other condition that precludes enteral route of administration
Malabsorption syndrome or other conditions that preclude enteral route of administration
History of serotonergic syndrome
Total bilirubin (T-Bil) =< 1.5 X ULN (prior diagnosis or past history consistent with Gilbert's syndrome is an exception)
Any history of Stevens-Johnson syndrome
Patients with an inherited cancer syndrome or a medical history suggestive of an inherited cancer syndrome remain eligible
No prior history of myelodysplastic syndrome or other myeloid malignancy
Patients with known Gilbert’s syndrome
Subjects with Gorlin syndrome
Prior treatment of myelodysplastic syndrome or myeloproliferative neoplasm with hypomethylating agent acceptable
Bilirubin equal or less than 1.5 (unless Gilbert's syndrome)
Patients with concomitant genetic syndrome: patients with Down syndrome, Fanconi anemia, Kostmann syndrome, Shwachman syndrome or any other known bone marrow failure syndrome
History of Gilbert's syndrome
Malabsorption syndrome, such as Crohn’s disease
No history of Steven’s Johnson’s syndrome, toxic epidermal necrolysis (TEN)s syndrome, or motor neuropathy
RANDOMIZED PHASE II (ARMS K AND L): No history of Steven’s Johnson’s syndrome, TENs syndrome, or motor neuropathy
History of Gilbert’s syndrome
Patients with a diagnosis of myelodysplastic syndrome/myeloproliferative neoplasm, unclassifiable (MDS/MPN-U) that require therapy
Acute IA defined as duration of clinical syndrome of <30 days.
POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, and skin changes)
Patients with active Richter's syndrome (>10% large B-cells in marrow).
Total bilirubin =< 2 X ULN (exceptions may apply to benign non-malignant indirect hyperbilirubinemia such as Gilbert syndrome)
Known drug-related, inherited, or acquired procoagulant disorder including prothrombin gene mutation 20210, antithrombin III deficiency, protein C deficiency, protein S deficiency and antiphospholipid syndrome but not including heterozygosity for the factor V Leiden mutation or the presence of a lupus anticoagulant in the absence of other criteria for the antiphospholipid syndrome
History of malabsorption syndrome e.g., pancreatic insufficiency, celiac disease, tropical sprue
Patient with FA must have moderately severe aplastic anemia (AA) myelodysplastic syndrome (MDS) or acute leukemia with or without chromosomal abnormalities
Myelodysplastic syndrome with multi-lineage dysplasia with or without chromosomal abnormalities
Myelodysplastic syndrome
History of multiple sclerosis or other demyelinating disease, Eaton-Lambert syndrome (para-neoplastic syndrome), history of hemorrhagic or ischemic stroke within the last 6 months, or alcoholic liver disease;
Subject has gastric outlet syndrome or persistent/recurrent vomiting.
Known history of myelodysplastic syndrome/leukemia at any time
Patients with myelodysplastic syndrome, aplastic anemia or hemoglobinopathies will be eligible to participate regardless of disease status if plan is to proceed to HCT
Criteria 3 POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, and skin changes)
Criteria 5 Myelodysplastic syndrome
History of motor neuropathy considered to be of autoimmune origin (e.g., Guillain-Barre Syndrome, Myasthenia Gravis).
Known presence of intermediate- or high-grade myelodysplastic syndrome.
Subject has plasma cell leukemia or Waldenstrom’s macroglobulinemia or POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, and skin changes) or amyloidosis
Myelodysplastic syndrome (MDS) or myelofibrosis;
POEMS syndrome
Myelodysplastic syndrome (MDS): primary or therapy related
Difficulty with swallowing, or an active malabsorption syndrome.
Difficulty with swallowing or an active malabsorption syndrome
Patients with HNPCC (Lynch Syndrome)
Symptomatic amyloidosis, active 'polyneuropathy, organomegaly, endocrinopathy, myeloma protein, and skin changes' (POEMS) syndrome, active plasma cell leukemia at the time of screening.
Diagnosis of myelodysplastic syndrome.
History of myelodysplastic syndrome or organ transplantation
POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, and skin changes)
Subject has plasma cell leukemia, Waldenstrom's macroglobulinemia, POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, and skin changes), or primary amyloidosis
History of Gilbert syndrome
Has any history of serotonin syndrome after receiving serotonergic drugs
Down syndrome
Kostmann syndrome
Shwachman syndrome
No known Gilbert’s syndrome or known homozygosity for UGAT1A1*28 polymorphism
History of Gilbert's syndrome
Have received a hypomethylating agent for myelodysplastic syndrome (MDS).
POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, monoclonal protein and skin changes)
Patient has a history of Stevens-Johnson-Syndrome (SJS) or Toxic Epidermal Necroloysis (TEN)
POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, and skin changes)
Patients with plasma cell leukemia, polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin changes (POEMS) syndrome, or amyloidosis are excluded from this trial
Subject has Acute Respiratory Distress Syndrome (ARDS)
Patient with and without Down syndrome are eligible and must have one of the following:\r\n* Second or greater relapse;\r\n* Primary refractory disease with at least 2 prior induction attempts;\r\n* First relapse refractory to at least one prior re-induction attempt\r\n* Any relapse after HSCT\r\n* First relapse with no prior re-induction attempt in setting of Down syndrome\r\n* Note: Patients with Down syndrome are eligible with any disease status including first relapse with no prior re-induction attempt; patients without Down syndrome are NOT eligible if in first relapse with no prior re-induction attempt
Patients known to have one of the following concomitant genetic syndromes: Bloom syndrome, ataxia-telangiectasia, Fanconi anemia, Kostmann syndrome, Shwachman syndrome, or any other known bone marrow failure syndrome
Documented Gilbert’s syndrome
Diagnosis of Down syndrome (Trisomy 21)
History of Gilbert’s syndrome
Patients with a history of myelodysplastic syndrome (MDS)
Patients with any of the following diagnoses:\r\n* Acute promyelocytic leukemia (APL)\r\n* Down syndrome\r\n* Fanconi anemia, Kostmann syndrome, Shwachman syndrome or any other known bone marrow failure syndrome\r\n* Wilson's disease and any other disorder of copper metabolism\r\n* Juvenile myelomonocytic leukemia (JMML)
Patients with Li Fraumeni syndrome are excluded from the study
Direct bilirubin ?2 X institutional ULN (unless due to known Gilbert's syndrome or compensated hemolysis directly attributable to CLL)
Radiosensitivity syndrome (scleroderma, dermatomyositis, other genetic syndrome that predisposes to adverse radiotherapy complications)
Total bilirubin =< 2 x ULN (patients with Gilbert syndrome may be included at the discretion of the principal investigator [PI] or where hemolysis has been excluded)
Patients with a family history or Li-Fraumeni syndrome will not be eligible
History of posterior reversible encephalopathy syndrome
Patients with Fanconi anemia, Kostmann syndrome, Shwachman syndrome, or other inherited bone marrow failure syndromes are not eligible
Patients who have experienced bowel perforation, neurologic involvement, Guillain Barré syndrome, Myasthenia Gravis, Steven Johnson syndrome and other intractable events or grade 4 non-laboratory toxicity
Patients must not have > grade 2 neuropathy and/or POEMS syndrome (plasma cell dyscrasia with polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, and skin changes)
MDS, myelodysplastic syndrome/myeloproliferative neoplasia overlap disorders (including chronic myelomonocytic leukemia [CMML], and MDS/myeloproliferative neoplasm [MPN] unclassifiable syndromes)
Diagnosis of polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin changes (POEMS) syndrome
Malabsorption syndrome or other condition that would interfere with intestinal absorption
Patients with malabsorption syndrome or other condition that would interfere with intestinal absorption
Patients with a history of toxic epidermal necrolysis (TEN) or Stevens-Johnson syndrome are excluded
Pre-existing nephritic syndrome
History of another primary invasive cancer, hematologic malignancy, or myelodysplastic syndrome that has not been in remission for at least 1 year
Subject has Down syndrome.
Presence of GVHD overlap syndrome as per NIH guidelines.
For Cohort A: Has myelodysplastic syndrome
Patients with concomitant genetic syndrome: such as patients with Fanconi anemia, Kostmann syndrome, Shwachman syndrome or any other known bone marrow failure syndrome. Patients with Down Syndrome will not be excluded.
History of Stevens-Johnson syndrome or toxic epidermal necrolysis syndrome.
Known prolymphocytic leukemia or history of, or currently suspected, Richter's syndrome.
Known prolymphocytic leukemia or history of, or currently suspected, Richter's syndrome.
Richter syndrome
POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, and skin changes)
Patients with peripheral arterial disease with intermittent claudication or Leriches Syndrome
Subjects who have history of toxic epidermal necrolysis or Stevens-Johnson syndrome;
Had prior serotonin syndrome
Patients with concomitant genetic syndrome: such as patients with Fanconi anemia, Kostmann syndrome, Shwachman syndrome or any other known bone marrow failure syndrome. Patients with Down Syndrome will not be excluded.
Presence of known intermediate- or high-grade myelodysplastic syndrome
Patients must not have known Gilbert’s syndrome
Known history or presence of Sweet Syndrome at screening
Plasma cell leukemia or POEMS (polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, and skin changes) syndrome
Presence of a malabsorption syndrome possibly affecting drug absorption (e.g., Crohn’s disease or chronic pancreatitis)
History of Steven's Johnson's syndrome or toxic epidermal necrolysis syndrome.
History of posterior reversible encephalopathy syndrome.
Known Gilbert's syndrome
No history of erythema multiforme, toxic epidermal necrolysis or Stevens-Johnson syndrome
POEMS syndrome
Acquired Immune Deficiency Syndrome (AIDS) based upon current CDC definition
Patients must not have POEMS syndrome (plasma cell dyscrasia with polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, and skin changes)
Any history of serotonin syndrome after receiving serotonergic drugs.
History of sarcoidosis syndrome
Have ongoing or recent (?6 months) hepatorenal syndrome.
Subjects with monoclonal gammopathy of undetermined significance (MGUS), smoldering multiple myeloma (SMM), primary amyloidosis, Waldenstrom's macroglobulinemia, or POEMS syndrome (plasma cell dyscrasia with poly neuropathy, organomegaly, endocrinopathy, monoclonal protein, and skin changes)
Malabsorption syndrome or poor upper gastrointestinal integrity
History of malabsorption syndrome or other condition that would interfere with enteral absorption
POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, and skin changes)
Myelodysplastic syndrome
Gastrointestinal tract disease or defect with associated malabsorption syndrome
Patients who have a known inherited syndrome as the cause for hormone over secretion.
Patients with Cushing's syndrome due to ectopic ACTH secretion or ACTH-independent (adrenal) Cushing's syndrome.
Patients must not have a known history of Gilbert’s syndrome or known homozygosity for the UDP glucuronosyltransferase 1 family, polypeptide A1 (UGT1A1)*28 allele
Have active metabolic or digestive illnesses such as malabsorptive disorders (Crohn’s, Celiac disease, irritable bowel syndrome [IBS]), renal insufficiency, hepatic insufficiency, cachexia, or short bowel syndrome
History of Osler-Weber-Rendu syndrome or hereditary hemorrhagic telangiectasia
Known history of myelodysplastic syndrome (MDS).
Patients must not have been diagnosed with myelodysplastic syndrome or with any malignancy other than neuroblastoma
History of motor neuropathy considered to be of autoimmune origin (e.g., Guillain-Barre syndrome, myasthenia gravis)
Known to have a hypercoagulability syndrome (e.g.: antithrombin III, deficiency, anticardiolipin syndrome etc)
Patients with Down syndrome, acute promyelocytic leukemia, juvenile myelomonocytic leukemia, Fanconi anemia, Kostmann syndrome, Shwachman syndrome, or other bone marrow failure syndromes are not eligible
History of concomitant genetic syndrome such as Fanconi anemia, Kostmann syndrome, Shwachman-Diamond syndrome or any other known bone marrow failure syndrome
Patients with active tumor lysis syndrome (TLS) either from laboratory or clinical changes
Malabsorption syndrome or other condition that precludes enteral route of administration
Wolff-Parkinson White syndrome or the presence of an intra-cardiac defibrillator
Pre-existing renal disease including glomerulonephritis, nephritic syndrome, Fanconi syndrome or renal tubular acidosis
Maldigestion/malabsorption syndrome pre-dating the diagnosis of pancreatic cancer.
Cushing’s syndrome
POEMS syndrome (plasma cell dyscrasia with polyneuropathy, organomegaly, endocrinopathy, monoclonal protein [M-protein] and skin changes)
Serum glutamate pyruvate transaminase (SGPT) =< 200 IU/ml (unless Gilbert's syndrome)
POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, and skin changes)
Myelodysplastic syndrome
Diagnosed with smoldering MM, monoclonal gammopathy of undetermined significance, Waldenstrom macroglobulinemia, plasma cell leukemia, polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin changes (POEMS) syndrome or amyloidosis
Evidence of Gilbert’s syndrome or known homozygosity for the UGT1A1*28 allele (special screening not required)
Participant has a malabsorption syndrome or other condition that precludes enteral route of administration.
Patients who have a history of myelodysplastic syndrome
Li-Fraumeni Syndrome
Diagnosis of Waldenstrom's macroglobulinemia, POEMS (polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin changes) syndrome, plasma cell leukemia, primary amyloidosis, myelodysplastic syndrome, or myeloproliferative syndrome.
Subject has malabsorption syndrome or other condition that precludes enteral route of administration
Subjects with a significant history of malabsorption (e.g. celiac sprue, short bowel syndrome, or other, as determined by the treating physician) are excluded
Active gastrointestinal tract disease with malabsorption syndrome
Patients must have a confirmed diagnosis of non-M3 AML; antecedent myelodysplastic syndrome (MDS) is acceptable
History of reversible posterior leukoencephalopathy syndrome (RPLS)
History of Gilbert’s syndrome
Malabsorption syndrome or other condition that precludes enteral route of administration
Known history of Gilbert's Syndrome or any current hyperbilirubinemia of any cause
History of malabsorption syndrome or other condition that would interfere with enteral absorption Exclusion Criteria Related to Medications:
History of sarcoidosis syndrome.
Patients with myelodysplastic syndrome/acute myeloid leukaemia.
POEMS syndrome (plasma cell dyscrasias with polyneuropathy, organomegaly, endocrinopathy, monoclonal protein [M-protein] and skin changes) or amyloid light-chain (AL) amyloidoses
Down Syndrome
Subject has active malignant tumors other than AML or myelodysplastic syndrome (MDS).
Malabsorption syndrome or other conditions that would interfere with intestinal absorption
Polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, skin (POEMS) syndrome
Has a known history of Gilbert's Syndrome
Diagnosis of Myelodysplastic Syndrome.
Polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin changes (POEMS) syndrome
Clinical evidence of nephrotic syndrome prior to enrollment
History of Gilbert’s syndrome
History of Gilbert's syndrome
Presence of chronic diarrhea (> grade 1 by Common Toxicity Criteria [CTC] criteria), short bowel syndrome, pancreatic insufficiency, or malabsorption
History of Gilbert’s syndrome
Participants with known disorders due to a deficiency in bilirubin glucuronidation (e.g. Gilbert’s syndrome)
History of sarcoidosis syndrome
Diagnosis of Waldenstrom's macroglobulinemia, POEMS syndrome, plasma cell leukemia, primary amyloidosis, myelodysplastic syndrome, or myeloproliferative syndrome
History of Gilbert's syndrome.
Total bilirubin =< 2 x ULN (except for patients with uridine diphosphate glucuronosyltransferase 1A1 [UGT1A1] promoter polymorphism, i.e. Gilbert syndrome, confirmed by genotyping or invader UGT1A1 molecular assay prior to study enrollment; patients enrolled with Gilbert syndrome must have total bilirubin < 3 ULN)
POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, and skin changes)
Participants with known disorders due to a deficiency in bilirubin glucuronidation (e.g. Gilbert’s syndrome)
History of Gilbert's syndrome
Patients with history of Down's syndrome, Fanconi anemia or other known marrow failure condition
Serum bilirubin =< 1.5 x ULN (unless Gilbert’s syndrome and evidence of hemolysis)
History of immune system abnormalities such as hyperimmunity (e.g., autoimmune diseases) that required systemic immunosuppression therapy and hypoimmunity (e.g., myelodysplastic disorders, marrow failures, acquired immunodeficiency syndrome (AIDS), ongoing pregnancy, transplant immunosuppression)
Any active or chronic corneal disorder and Sjogren's syndrome.
Diagnosis of Waldenstrom's macroglobulinemia, polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin changes (POEMS) syndrome, plasma cell leukemia, primary amyloidosis, myelodysplastic syndrome, or myeloproliferative syndrome.
Individuals with Down syndrome
Bilirubin < to 2.0 x normal (except Gilbert’s Syndrome)
Prior treatment of myelodysplastic syndrome or myeloproliferative neoplasm acceptable
History of immune system abnormalities such as hyperimmunity (e.g., autoimmune diseases) and hypoimmunity (e.g., myelodysplastic disorders, marrow failures, acquired immune deficiency syndrome [AIDS], ongoing pregnancy, transplant immunosuppression)
Any other significant medical condition not under control, including any acute coronary syndrome within the past 6 months
History of Gilbert’s syndrome
Myelodysplastic syndrome with fibrosis (MF 3)
Total bilirubin =< 2.0 (exceptions may apply to benign non-malignant indirect hyperbilirubinemia such as Gilbert syndrome)
Carcinoid Syndrome
Has a known history of, or active, neurologic paraneoplastic syndrome
Polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin changes (POEMS) syndrome
Nephrotic syndrome
Bilirubin =< 2 mg/dL (exceptions may apply to benign non-malignant indirect hyperbilirubinemia such as Gilbert syndrome)
No known malabsorption syndrome
Patients with a known mutation in p53 (Li Fraumeni syndrome)
Known procoagulant disorder including prothrombin gene mutation 20210, antithrombin III deficiency, protein C deficiency, protein S deficiency and antiphospholipid syndrome but not including heterozygosity for the Factor V Leiden mutation or the presence of a lupus anticoagulant in the absence of other criteria for the antiphospholipid syndrome
Down syndrome
Kostmann syndrome
Shwachman syndrome
A pathologically confirmed diagnosis of leukemia or myelodysplastic syndrome (MDS)
Myelodysplastic syndrome (MDS): primary or therapy related; or
Patients with Down syndrome and deoxyribonucleic acid (DNA) fragility syndromes (such as Fanconi anemia, Bloom syndrome) are excluded
Received prior treatment with HMA or chemotherapy for antecedent myelodysplastic syndrome (MDS)
Presence of chronic GvHD at Screening (including acute-chronic overlap syndrome).
Previously untreated for Myelodysplastic Syndrome (MDS)
Known presence of myelodysplastic syndrome
Concomitant genetic syndrome or other known bone marrow failure syndrome
Down syndrome
Serum bilirubin ? 2 x ULN unless due to Gilbert's syndrome or hemolysis or considered to be related to leukemia;
Known presence of myelodysplastic syndrome
History of Gilbert's syndrome.
Patients with Down syndrome are excluded.
Bilirubin ? 2.0 times ULN, unless subject has known Gilbert's syndrome
Neurologic disease including multiple sclerosis, Eaton-Lambert syndrome, demyelination
Myelodysplastic Syndrome (MDS) at any stage.
Patients with nevoid BCC syndrome (Gorlin syndrome) may enroll in this study but must meet the criteria for locally advanced or metastatic disease listed above
Prior treatment with decitabine for myelodysplastic syndrome (MDS) or AML
History of Gilbert’s syndrome
Known history of Gilbert’s syndrome
Known presence of myelodysplastic syndrome
Patients with a known history of Gilbert’s syndrome
4. Known history of Gilbert's Syndrome.
Sézary syndrome
History of malabsorption syndrome or other condition that would interfere with enteral absorption
Active disease transformation (ie, Richter's Syndrome); subjects with Richter's Syndrome that has resolved > 2 years from signing the ICD are eligible.
History of Stevens-Johnson syndrome characterized by a desquamating rash while taking thalidomide or similar drugs.
Myelodysplastic syndrome\r\n* =< 55 years of age and >= 10% blasts, not responsive to hypomethylating agents and/or conventional therapy
Subjects with posterior leukoencephalopathy syndrome
Bilirubin greater than 1.5 mg/dl (>26 µmol/L) unless elevation is thought to be due to hepatic infiltration by AML, Gilbert syndrome, or hemolysis.
History of neurological conditions that would confound assessment of treatment-emergent neuropathy other than =< grade 1 peripheral neuropathy including multiple sclerosis or other demyelinating disease and/or Eaton-Lambert syndrome (para-neoplastic syndrome); diabetes is allowed
Diagnosis of myelodysplastic syndrome (MDS) or a hematologic malignancy.
Subjects who have Gorlin syndrome
Malabsorption syndrome or other condition that would interfere with enteric absorption
History of hereditary hemorrhagic telangiectasia (HHT, Osler-Weber-Rendu syndrome)
Patients with low- and Int-1-risk myelodysplastic syndrome
Documented diagnosis of the following: Myelodysplastic syndrome lasting ? 3 months and < 3 years Disease must not be secondary to treatment with radiotherapy, chemotherapy, and/or immunotherapy for malignant or autoimmune diseases
Any pre-excitation syndromes, e.g., Wolff-Parkinson-White syndrome;
History of sarcoidosis syndrome
Patients with a family history or Li-Fraumeni syndrome will not be eligible
Patient has Down syndrome
POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, monoclonal protein and skin changes).
POEMS (plasma cell dyscrasia with polyneuropathy, organomegaly, endocrinopathy, monoclonal protein [M-protein] and skin changes) syndrome
History of Gilbert’s syndrome
Subject has active malignant tumors other than AML or Myelodysplastic syndrome (MDS).
POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, monoclonal protein and skin changes).
Plasma cell dyscrasia with polyneuropathy, organomegaly, endocrinopathy, monoclonal protein (M-protein), and skin changes (POEMS) syndrome
Patients with myelodysplastic syndrome
POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, and skin changes)
Patients with myelodysplastic syndrome
Nephrotic syndrome
Diagnosis of Waldenstrom's macroglobulinemia, POEMS (polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin changes) syndrome, plasma cell leukemia, myeloproliferative syndrome, or primary amyloidosis (with the exception of patients in whom amyloidosis has been documented as a complication of MM, who will be evaluated on a case-by-case basis for trial participation)
Patients with a known germline mutation of PTPN11 (Noonan’s Syndrome) are not eligible
POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, and skin changes)
POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, and skin changes)
Immediate family member with a known or suspected Familial Cancer Syndrome (including but not limited to hereditary breast and ovarian cancer syndrome, hereditary non-polyposis colorectal cancer syndrome and familial adenomatous polyposis).
Subject has acute chest syndrome
total bilirubin ?1.5 X institutional ULN unless due to Gilbert's syndrome, controlled autoimmune hemolytic anemia or immune thrombocytopenia
History of congenital platelet function defect (e.g., Bernard-Soulier syndrome, Chediak-Higashi syndrome, Glanzmann thrombasthenia, storage pool defect)
History of hepatic sinusoid obstructive syndrome (venoocculsive disease) within the prior 3 months.
History of malabsorption syndrome or other condition that would interfere with enteral absorption or results in the inability or unwillingness to swallow pills
Known primary immunodeficiencies, either cellular (e.g., DiGeorge syndrome, T-negative severe combined immunodeficiency [SCID]) or combined T- and B-cell immunodeficiencies (e.g., T- and B-negative SCID, Wiskott Aldrich syndrome, ataxia telangiectasia, common variable immunodeficiency)
Participants with malabsorption syndrome or any other condition that precludes enteral administration.
POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, and skin changes).
Patients with myelodysplastic syndrome.
Malabsorption syndrome
History of malabsorption syndrome or other condition that would interfere with enteral absorption
Myelodysplastic syndrome (MDS)
POEMS syndrome (plasma cell dyscrasia with polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, and skin changes)
Diagnosis of smoldering multiple myeloma, Waldenstrom's macroglobulinemia, POEMS syndrome, plasma cell leukemia, primary amyloidosis, myelodysplastic syndrome, or myeloproliferative syndrome
Has known gastrointestinal condition or procedure that could interfere with swallowing or the oral absorption of tolerance of IXAZOMIB - Diagnosis of smoldering multiple myeloma, Waldenstrom's macroglobulinemia, POEMS syndrome, plasma cell leukemia, primary amyloidosis, myelodysplastic syndrome, or myeloproliferative syndrome
Patients with a history of hereditary hemorrhagic telangiectasia (Osler-Weber-Rendu syndrome)
Patients with an immunodeficiency syndrome
POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, and skin changes)
Total bilirubin < 1.5 x upper limit of reference range (ULRR), unless the patient meets the criteria for Gilbert’s syndrome
Total bilirubin > 1.5 x ULN (unless due to lymphoma or isolated, predominantly indirect hyperbilirubinemia due to Gilbert's syndrome)
POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, and skin changes)
Bilirubin equal or less than 1.5 (unless Gilbert's Syndrome)
History of malabsorption syndrome or other condition that would interfere with enteral absorption
hyperviscosity syndrome due to monoclonal gammopathy
Acute coronary syndrome
Patients diagnosed with Sezary syndrome; Sezary syndrome is equivalent to mycosis fungoides that develops to stage IVA/B with B2 (high blood tumor burden) involvement, and as such requires a more aggressive treatment regimen than Valchlor or TSEBT
Patients with any of the following diagnoses are not eligible:\r\n* Acute promyelocytic leukemia (APL)\r\n* Down syndrome\r\n* Fanconi anemia, Kostmann syndrome, Shwachman syndrome or any other known bone marrow failure syndrome\r\n* Wilson's disease and any other disorder of copper metabolism\r\n* Juvenile myelomonocytic leukemia (JMML)
Active polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin changes (POEMS) syndrome.
Polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin changes (POEMS) syndrome
History of Gilbert’s syndrome
Active or a history of Tourette’s syndrome or tic disorder
Have a confirmed diagnosis of sleep apnea or restless leg syndrome
Patients with Down syndrome
Known malabsorption syndrome, short bowel or chologenic diarrhea not controlled by specific therapeutic means
Patients with Down syndrome, acute promyelocytic leukemia, juvenile myelomonocytic leukemia, Fanconi anemia, Kostmann syndrome, Shwachman syndrome, or other bone marrow failure syndromes are not eligible
Malabsorption syndrome or other condition that would interfere with enteral absorption
Known myelodysplastic syndrome
Known myelodysplastic syndrome.
Acute Coronary Syndrome (ACS) (angina or MI) in last 6 months.
Diagnosis of myelodysplastic syndrome (MDS).
All patients who present for an initial consultation at the Dana Farber Cancer Institute (DFCI) for myelodysplastic syndrome (MDS) or a hematologic malignancy (transplantation consultation excluded)
Sleep apnea or restless leg syndrome (RLS)
Acute coronary syndrome as defined by active chest pain, dynamic electrocardiogram (ECG) changes, troponin greater than 2.5
Co-morbidity with immunosuppressive disease such as acquired immune deficiency syndrome (AIDS)
Known Mirizzi syndrome.
History of irritable bowel syndrome (IBS)
Delayed gastric emptying syndrome
Patients with Sjogren's syndrome
Has difficulty swallowing medications, or known history of malabsorption syndrome;
Ongoing or recent hepatorenal syndrome.
Current Myelodysplastic syndrome only subjects
History of neuroleptic malignant syndrome
Patients with diagnosis of chronic fatigue syndrome (CFS)
POEMS syndrome (plasma cell dyscrasia with polyneuropathy, organomegaly, endocrinopathy, monoclonal protein [M-protein] and skin changes)
Patients with a diagnosis of obesity hypoventilation syndrome
POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, and skin changes)
Subjects with a history of myelodysplastic syndrome
Subjects who have currently active hepatic or biliary disease (with the exception of patients with Gilbert's syndrome)
Diagnosed with hematological malignancy or marrow failure syndrome such as but not limited to: aplastic anemia, myelodysplastic syndrome or leukemia
Have a clinical diagnosis of sleep apnea or restless leg syndrome
Patients with acute/chronic GVHD overlap syndrome
History of irritable bowel syndrome (IBS)
Myelodysplatic Syndrome
History of Guillain-Barre syndrome
History of Guillain-Barre syndrome
History of malabsorption syndrome (e.g., pancreatic insufficiency, celiac disease, or tropical sprue)
Have a history of Guillain-Barre syndrome (GBS)
Bone marrow failure disorders:\r\n* Paroxysmal nocturnal hemoglobinuria (PNH)\r\n* Hereditary bone marrow failure disorders include Diamond-Blackfan anemia, Shwachman- Diamond syndrome, Kostmann syndrome, and congenital amegakaryocytic thrombocytopenia\r\n* Other non-malignant hematologic or immunologic disorders that require transplantation:\r\n** Quantitative or qualitative congenital platelet disorders (including but not limited to congenital megakaryocytopenia, absent-radii syndrome, Glanzmann’s thrombasthenia)\r\n** Quantitative or qualitative congenital neutrophil disorders (including but not limited to chronic granulomatous disease, congenital neutropenia)\r\n** Congenital primary immunodeficiencies (including but not limited to severe combined immunodeficiency syndrome, Wiskott-Aldrich syndrome, CD40 ligand deficiency, T-cell deficiencies)\r\n** Hemoglobinopathies (including sickle cell disease and thalassemia)
Patients with known or suspected Gilbert’s syndrome at the time of study enrollment
Down syndrome
Short gut syndrome
Any malabsorption disease/syndrome
History of atrophic gastritis, pernicious anemia, or Zollinger-Ellison syndrome
Subjects who have Gorlins syndrome
Subjects with treatment-related myelodysplastic syndrome.
Solitary bone or extramedullary plasmacytoma as the only evidence of plasma cell dyscrasia, or monoclonal gammopathy of undetermined significance (MGUS), smoldering multiple myeloma (SMM), primary amyloidosis, Waldenstrom's macroglobulinemia, POEMS syndrome or active plasma cell leukemia
POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, and skin changes)
A defined myelodysplastic syndrome(s) (MDS)
History of malabsorption syndrome (e.g., pancreatic insufficiency, celiac disease, or tropical sprue)
Diagnosis of Down's Syndrome
Histologic confirmation of leukemia or myelodysplatic syndrome (MDS) at the time of diagnosis or recurrence
Individuals with “Li Fraumeni syndrome” defined as one of the following:\r\n* Carriers of a germline protein 53 (p53) mutation\r\n* Members of families meeting classic Li-Fraumeni syndrome (LFS) criteria by family history without an identifiable p53 mutation\r\n* Obligate carrier by pedigree (these individuals can be offered testing but are still eligible if they defer); the following examples describe “obligate carriers by pedigree”\r\n** A child of a parent with known p53 mutation that is diagnosed with cancer\r\n** An individual with a sibling and a child who are p53 positive OR\r\n* Individuals with an inherited cancer predisposition syndrome as defined by one of the following:\r\n** Hereditary retinoblastoma with a germline retinoblastoma (Rb) mutation\r\n** Diagnosis of hereditary paraganglioma/pheochromocytoma syndrome with a germline succinate dehydrogenase (SDH) mutation\r\n** Diagnosis of multiple endocrine neoplasia, type 1 or 2, with a germline multiple endocrine neoplasia (MEN) mutation\r\n** New diagnosis of opsoclonus-myoclonus with a negative cancer work-up upon presentation of symptoms\r\n** Familial neuroblastoma with a germline anaplastic lymphoma kinase (ALK) mutation\r\n** Rapid-onset obesity with hypothalamic dysfunction, hypoventilation and autonomic dysregulation (ROHHAD syndrome) or congenital central hypoventilation syndrome (CCHS) with or without a germline paired–like homeobox 2B (PHOX 2B) mutation\r\n** Von Hippel-Lindau with a Von Hippel-Lindau (VHL) mutation\r\n** Women with an abnormal cell-free deoxyribonucleic acid (DNA) test (i.e. a non-invasive prenatal test [NIPT] to detect chromosomal abnormalities) and no cancer diagnosis\r\n** Other rare cancer predisposition syndromes at the discretion of the treating physician and study physicians\r\n* IF APPLICABLE: individuals with any of the above-listed cancer predisposition syndromes (apart from Li Fraumeni syndrome) are likewise eligible in the absence of a known mutation if they are an obligate carrier by pedigree
Individuals with “Li Fraumeni syndrome” defined as one of the following:\r\n* Carriers of a germline p53 mutation OR\r\n* Members of families meeting classic LFS criteria by family history without an identifiable p53 mutation OR\r\n* Obligate carrier by pedigree (these individuals can be offered testing but are still eligible if they defer); the following examples describe “obligate carriers by pedigree”\r\n** A child of a parent with known p53 mutation that is diagnosed with cancer\r\n** An individual with a sibling and a child who are p53 positive OR\r\n* Individuals with an inherited cancer predisposition syndrome as defined by one of the following:\r\n** Hereditary retinoblastoma with a germline Rb mutation\r\n** Diagnosis of hereditary paraganglioma/pheochromocytoma syndrome with a germline SDH mutation\r\n** Diagnosis of multiple endocrine neoplasia, type 1 or 2, with a germline MEN mutation\r\n** New diagnosis of opsoclonus-myoclonus with a negative cancer work-up upon presentation of symptoms\r\n** Familial neuroblastoma with a germline ALK mutation\r\n** Rapid-onset obesity with hypothalamic dysfunction, hypoventilation and autonomic dysregulation (ROHHAD syndrome) or congenital central hypoventilation syndrome (CCHS) with or without a germline PHOX 2B mutation\r\n** Von Hippel-Lindau with a VHL mutation\r\n** Other rare cancer predisposition syndrome at the discretion of the treating physician and study physicians\r\n* IF APPLICABLE: individuals with any of the above-listed cancer predisposition syndromes (apart from Li Fraumeni syndrome) are likewise eligible in the absence of a known mutation if they are an obligate carrier by pedigree
History of Stevens-Johnson syndrome
History of Stevens-Johnson syndrome
Subject has plasma cell leukemia, Waldenstrom's macroglobulinemia, POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, and skin changes), or primary amyloidosis
Patients with respiratory distress syndrome
Patients who have experienced an acute coronary syndrome or angina in the past 6 months
PATIENT: Patients with respiratory distress syndrome
Has been diagnosis of active acquired immunodeficiency syndrome (AIDS) or hepatitis B/C and other significant disease or disorders
Patients with unstable cardiopulmonary conditions or respiratory distress syndrome
History of Steven’s Johnson’s syndrome, toxic epidermal necrolysis syndrome (TENs) or motor neuropathy
Polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin changes (POEMS) syndrome.
Pre-existing renal disease including glomerulonephritis, nephritic syndrome, Fanconi syndrome or renal tubular acidosis
Subjects with Gilbert's syndrome or resolving autoimmune hemolytic anemia may have a bilirubin up to 3.0 x ULN and are still eligible
POEMS syndrome [plasma cell dyscrasia with polyneuropathy, organomegaly, endocrinopathy, monoclonal protein (M-protein) and skin changes]
POEMs syndrome (polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, and skin changes).
Patients with respiratory distress syndrome
Patients with respiratory distress syndrome
History of Crohn's disease, celiac sprue or other malabsorption syndrome which may interfere with digestion and absorption of broccoli sprout extract
POEMS (Polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy and skin changes) syndrome, monoclonal gammopathy of unknown significance, smoldering myeloma, solitary plasmacytoma, amyloidosis, Waldenström macroglobulinemia, or IgM myeloma.
POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, and skin changes)
Myelodysplastic Syndrome
Bilirubin >1.5 x ULN, unless prior diagnosis and documentation of leukemic organ involvement, ongoing hemolysis, or Gilbert's syndrome
History of Stevens-Johnson syndrome or toxic epidermal necrolysis syndrome.
History of Guillain-Barre syndrome or Stevens-Johnson syndrome