Refractory anemia with ringed sideroblasts (RARS)
Active myeloma as defined as the presence of calcium, renal failure, anemia and bone (CRAB) criteria: hypercalcemia, renal insufficiency, anemia and/or bone disease
Patients with anemia (hemoglobin [HEM] < 10 g/dl) at baseline will be excluded unless otherwise approved by the PI or PI’s designee
Primary immunodeficiency disorder or other nonmalignant inherited disease (except Fanconi anemia) treatable by allogeneic HCT
Acquired aplastic anemia: severe aplastic anemia (SAA) is defined as follows:\r\n* Bone marrow cellularity < 25%, or marrow cellularity < 50% but with < 30% residual hematopoietic cells\r\n* Two out of three of the following (in peripheral blood): neutrophils < 0.5 x 10^9/L; platelets < 20 x 10^9/L; reticulocytes < 20 x 10^9/L\r\n* SAA diagnostic criteria may be applied to assessment at initial diagnosis or follow-up assessments
Fanconi anemia
Patients with a secondary condition (sickle cell anemia) that cause priapism
Inherited bone marrow failure syndromes. At minimum, the diagnosis of Fanconi anemia must be excluded by diepoxybutane (DEB) or equivalent testing on peripheral blood or marrow.
Patients with idiopathic aplastic anemia and Fanconi anemia; (patients with aplastic anemia associated with paroxysmal nocturnal hemoglobinuria [PNH] or inherited marrow failure syndromes, except Fanconi anemia, will be allowed)
History of hemolytic anemia or bleeding diathesis or positive direct antiglobulin test.
Patients that have active hemolytic anemia
Bone marrow failure syndromes, except for Fanconi anemia
Patients with Fanconi anemia; at a minimum, the diagnosis of Fanconi anemia must be excluded by diepoxybutane (DEB) or equivalent testing on peripheral blood or marrow in patients younger than 30 years of age; (additional mutational testing may have been performed in a clinical or research capacity on a per patient basis but is not considered an exclusion criteria)
Acquired bone marrow failure syndromes, except for Fanconi anemia
Patients that have active hemolytic anemia
Fanconi Anemia or other underlying bone marrow failure syndrome
MRI MONITORING SUB-STUDY: Known history of severe renal insufficiency, asthma, allergic conditions, sickle cell anemia, chronic hemolytic anemia, and gastrointestinal disorders
No clinically apparent alternative explanation for thrombocytopenia and anemia
Patients with sickle-cell anemia or thalassemia major.
Diagnosis of Fanconi anemia must be excluded by mitomycin C or diepoxybutane chromosomal breakage testing on peripheral blood at a CLIA-approved laboratory (not required for patients with a genetic mutation consistent with DC)
Patients treated on this study will have:\r\n* Acute leukemia in 1st or 2nd complete remission (CR)\r\n* MDS (myelodysplastic syndrome), specific subtypes of RA (refractory anemia) or RARS (refractory anemia with ringed sideroblasts) subtypes\r\n* Hodgkin or indolent non-Hodgkin’s lymphoma with chemosensitive disease\r\n* Myeloma without morphological evidence of disease, or a PR to the most recent therapy\r\n* Myeloproliferative disorders with at least a PR to current therapy\r\n* Aplastic anemia\r\n* A hematological or oncological disease (not listed) that meets the criteria reviewed above
In patients 18-60 years of age, moderately severe aplastic anemia is defined as having at least one of the following:\r\n* Platelet count < 20 x 10^9/L\r\n* ANC < 5 x 10^8/L\r\n* Hgb < 8 g/dL
Diagnosis of Fanconi anemia
Patients with Fanconi anemia (FA) must have aplastic anemia (AA), myelodysplastic syndrome without excess blasts, or high risk genotype as defined below\r\n* Aplastic anemia is defined as having at least one of the following when not receiving growth factors or transfusions:\r\n** Platelet count < 20 x 10^9 IL\r\n** Absolute neutrophil count (ANC) < 5 x 10^8/L\r\n** Hemoglobin (Hgb) < 8 g/dL\r\n* Myelodysplastic syndrome with multilineage dysplasia with or without chromosomal anomalies\r\n* High risk genotype (e.g. IVS-4 or exon 14 FANCC mutations, or BRCA1 or 2 mutations)
Bone marrow failure syndromes, except for Fanconi anemia
Acquired bone marrow failure syndromes except for Fanconi anemia or dyskeratosis congenita
Patients with myelodysplastic syndrome, aplastic anemia or hemoglobinopathies will be eligible to participate regardless of disease status if plan is to proceed to HCT
Patient has anemia due to HbS or HbC disease, alpha or beta thalassaemia
Diamond Blackfan anemia;
REGISTRATION TO TREATMENT (STEP 1): No active hemolytic anemia requiring immunosuppressive therapy or other pharmacologic treatment; patients who have a positive Coombs test but no evidence of hemolysis are NOT excluded from participation
REGISTRATION TO TREATMENT (STEP 2): No active hemolytic anemia requiring immunosuppressive therapy or other pharmacologic treatment; patients who have a positive Coombs test but no evidence of hemolysis are NOT excluded from participation
Fanconi anemia (FA)
Participants with a diagnosis of MDS secondary to paroxysmal nocturnal hemoglobinuria (PNH), aplastic anemia, or another inherited bone marrow failure disorder
Auto-immune hemolytic anemia
Patients that have active hemolytic anemia
Fanconi anemia
Primary immunodeficiency disorder or other nonmalignant inherited disease (except aplastic anemia and Fanconi anemia) treatable by allogeneic HCT
Patients with Aplastic anemia and Fanconi anemia
Non-malignant disorders deemed curable by allogeneic transplantation: a. primary immune deficiencies, b. severe aplastic anemia not responding to immune suppressive therapy, c. osteopetrosis d. selected cases of erythroid disorders such as ?0 ?0 thalassemia major, sickle cell disease, Diamond-Blackfan anemia. e. congenital/hereditary cytopenia, including Fanconi Anemia before any clonal malignant evolution (MDS, AML). Note: Subjects will be eligible if they meet either item 5 OR item 6.
Known clinically significant anemia due to iron, vitamin B12, or folate deficiencies, or autoimmune or hereditary hemolytic anemia, or gastrointestinal bleeding
Is undergoing transplant for the treatment of nonmalignant hematological disorders (for example: aplastic anemia, sickle cell anemia, thalassemias, Fanconi anemia).
Fanconi Anemia
Diagnosis of aplastic anemia, pure red cell aplasia, myelodysplasia or any of the other bone marrow failure states.
Patients with Fanconi anemia or other cancer-predisposition syndromes
History of hemolytic anemia or thalassemia
No active hemolytic anemia requiring immunosuppressive therapy or other pharmacologic treatment; patients who have a positive Coombs test but no evidence of hemolysis are NOT excluded from participation
Patients with sickle-cell anemia or thalassemia major.
Patients with history of Down's syndrome, Fanconi anemia or other known marrow failure condition
Diagnosis of severe aplastic anemia: eligibility to be discussed with principal investigator (PI) and service chief; such patients will be assessed in Arm B
Patients that have active hemolytic anemia
Fanconi anemia (FA)
Anemia below lower limit of normal or anemia requiring transfusion support as per center standard
Anemia
Active hemolytic anemia requiring immunosuppressive therapy or other pharmacologic treatment; NOTE: patients who have a positive Coombs test but no evidence of hemolysis are NOT excluded from participation
Active autoimmune haemolytic anemia.
Diagnosis of Fanconi anemia must be excluded in patients younger than 18 years of age by diepoxybutane testing on peripheral blood or comparable testing on marrow.
Other indications for HCT, including Fanconi anemia, other form of inherited bone marrow failure diseases, metabolic disorder, hemoglobinopathy, or immune deficiency
Severe active anemia (a hemoglobin < 8 documented by labs drawn within 3 months of first study treatment)
Have hypothyroidism and anemia
Active or unstable metabolic conditions such as brittle diabetes or severe anemia (hemoglobin < 8 g/dl)
Hemolytic anemia
Patient with aplastic anemia will be excluded
Active hemolytic anemia
History of atrophic gastritis, pernicious anemia, or Zollinger-Ellison syndrome
Patients with sickle cell anemia and other hemolytic anemia
Persons with any other condition (such as lichen planus, Fanconi anemia, heavy tobacco use, etc) making them at higher risk for oral cancer development
Subjects at increased risk for radiation toxicities, such as known collagen vascular disease (example, scleroderma, Sjogren's disease, etc) or other inherited radiation hypersensitivity syndromes (example, Gorlin syndrome, Fanconi anemia, ataxia-telangiectasia, etc.)
Subjects treated, or anticipated to be treated, with a calcineurin inhibitor (because concomitant use of sirolimus and a calcineurin inhibitor increases the risk of calcineurin inhibitor-induced hemolytic uremic syndrome/thrombotic thrombocytopenic purpura/thrombotic microangiopathy [HUS/TTP/TMA]).
History of hemolytic-uremic syndrome.
History of thrombotic microangiopathy, hemolytic-uremic syndrome or thrombotic thrombocytopenic purpura
Intolerance of protocol agents as follows:\r\n* Known or presumed intolerance of gemcitabine, vorinostat or sorafenib\r\n* Experienced any of the following toxicities with prior gemcitabine administration (if given): capillary leak syndrome, posterior reversible encephalopathy, hemolytic uremic syndrome, thrombotic thrombocytopenic purpura, unexplained dyspnea or other evidence of severe pulmonary toxicity, or severe hepatic toxicity
History of thrombotic microangiopathy, hemolytic-uremic syndrome or thrombotic thrombocytopenic purpura
History of thrombotic microangiopathy, hemolytic-uremic syndrome, or thrombotic thrombocytopenic purpura
History of thrombotic microangiopathy, hemolytic-uremic syndrome or thrombotic thrombocytopenic purpura.
History of microangiopathic hemolysis, thrombotic thrombocytopenic purpura (TTP) or hemolytic-uremic syndrome (HUS)
Underlying renal disease with a high risk of disease recurrence in the transplanted kidney, including:\r\n* Focal segmental glomerulosclerosis (FSGS)\r\n* Type I or II membranoproliferative glomerulonephritis\r\n* Hemolytic-uremic syndrome/thrombotic thrombocytopenic purpura
Presently with or a history of acute promyelocytic leukemia (APML), idiopathic thrombocytopenic purpura (ITP), thrombotic thrombocytopenic purpura (TTP), or hemolytic uremic syndrome (HUS)
History of thrombotic microangiopathy, hemolytic-uremic syndrome or thrombotic thrombocytopenic purpura
Uncontrolled autoimmune hemolytic anemia or thrombocytopenia.
History of autoimmune hemolytic anemia or autoimmune thrombocytopenia.
Untreated autoimmunity such as autoimmune hemolytic anemia, or immune thrombocytopenia
Uncontrolled autoimmune hemolytic anemia or thrombocytopenia
Uncontrolled autoimmune hemolytic anemia (AIHA) or idiopathic thrombocytopenia purpura (ITP)
Uncontrolled autoimmune hemolytic anemia or thrombocytopenia
Active and uncontrolled autoimmune hemolytic anemia (AIHA) or autoimmune thrombocytopenia (ITP) requiring daily prednisone dose of >= 20 mg.
Patients should not have active or uncontrolled autoimmune hemolytic anemia or immune thrombocytopenia
Active, uncontrolled autoimmune phenomenon (autoimmune hemolytic anemia or immune thrombocytopenia) requiring steroid therapy > 20 mg prednisone daily or equivalent, within 7 days of starting venetoclax
Active, uncontrolled autoimmune hemolytic anemia or immune thrombocytopenia requiring steroid therapy
Active, uncontrolled autoimmune phenomenon (autoimmune hemolytic anemia or immune thrombocytopenia) requiring steroid therapy with > 20 mg daily of prednisone dose or equivalent
Active, uncontrolled autoimmune phenomenon (autoimmune hemolytic anemia or immune thrombocytopenia) requiring steroid therapy
Active autoimmune disorder (with the exception of autoimmune hemolytic anemia or ITP)
Active, uncontrolled autoimmune hemolytic anemia or immune thrombocytopenia requiring steroid therapy
Autoimmune anemia and/or thrombocytopenia poorly responsive to corticosteroids or other standard therapy
Patients who exhibit any active or on-going autoimmune processes including, but not limited to, autoimmune hemolytic anemia or immune thrombocytopenia purpura, are NOT eligible for participation
Patients with uncontrolled Autoimmune Hemolytic Anemia (AIHA) or autoimmune thrombocytopenia (ITP).
Patients with active autoimmune anemia or autoimmune thrombocytopenia are NOT eligible
Autoimmune hemolytic anemia (AIHA) or idiopathic thrombocytopenia purpura (ITP) that is uncontrolled or requires >20 mg daily (QD) of prednisone (or equivalent) to maintain hemoglobin >8.0 g/dL or platelets >10,000 ?L without transfusion support
Uncontrolled autoimmune hemolytic anemia or thrombocytopenia
Uncontrolled autoimmune hemolytic anemia (Hgb < 11g/deciliter) or idiopathic thrombocytopenic purpura (< 100,000/µl)
Uncontrolled autoimmune hemolytic anemia or idiopathic thrombocytopenic purpura (ITP) resulting in (or as evidenced by) declining platelet or hemoglobin (Hgb) levels within the 4 weeks prior to first dose of study drug
Autoimmune anemia or thrombocytopenia that is poorly responsive to corticosteroids
Uncontrolled autoimmune hemolytic anemia or immune thrombocytopenic purpura (ITP) resulting in (or as evidenced by) declining platelet or hemoglobin (Hgb) levels within the 4 weeks prior to first dose of study drug
Uncontrolled autoimmune hemolytic anemia or idiopathic thrombocytopenia purpura.
Uncontrolled autoimmune hemolytic anemia or idiopathic thrombocytopenia purpura.
Autoimmune anemia and/or thrombocytopenia that is poorly responsive to corticosteroids or other standard therapy.
Patients with active or uncontrolled autoimmune hemolytic anemia or idiopathic thrombocytopenic purpura (ITP) are excluded; patients who have transfusion-dependent thrombocytopenia or bleeding/coagulation disorders that may increase the risk of life-threatening bleeding are excluded
Uncontrolled autoimmune hemolytic anemia or idiopathic thrombocytopenic purpura (ITP) resulting in (or as evidenced by) declining platelet or hemoglobin (Hgb) levels within the 4 weeks prior to first dose of study drug
Patients with uncontrolled autoimmune hemolytic anemia (AIHA) or autoimmune thrombocytopenia (ITP)
Uncontrolled autoimmune hemolytic anemia or thrombocytopenia
History of autoimmune hemolytic anemia or autoimmune thrombocytopenia.
Autoimmune disease with the exception of controlled/treated hypothyroidism, disease-related immune thrombocytopenic purpura, or hemolytic anemia
Untreated autoimmunity such as autoimmune hemolytic anemia, or immune thrombocytopenia
Autoimmune disease related to CLL, e.g., idiopathic thrombocytopenic purpura (ITP) or autoimmune hemolytic anemia, is permitted if not requiring active treatment
Active, uncontrolled autoimmune hemolytic anemia or immune thrombocytopenia requiring steroid therapy
Uncontrolled autoimmune hemolytic anemia or thrombocytopenia
Participants with uncontrolled autoimmune hemolytic anemia or immune thrombocytopenia
Subjects with uncontrolled autoimmune hemolytic anemia or idiopathic thrombocytopenic purpura (ITP) resulting in declining platelet or hemoglobin levels within the 4 weeks prior to first dose of study drug are not eligible
Uncontrolled autoimmune hemolytic anemia or idiopathic thrombocytopenia purpura.
Uncontrolled autoimmune hemolytic anemia (AIHA) or idiopathic thrombocytopenia purpura (ITP) that is uncontrolled or requiring > 20 mg once daily (QD) of prednisone (or equivalent) to maintain hemoglobin > 8.0 g/dL or platelets > 10,000 ?L without transfusion support
Active, uncontrolled autoimmune hemolytic anemia or idiopathic thrombocytopenic purpura (ITP)
Uncontrolled autoimmune hemolytic anemia or thrombocytopenia
Patients with uncontrolled autoimmune hemolytic anemia or autoimmune thrombocytopenia
Uncontrolled autoimmune hemolytic anemia (AIHA) or immune thrombocytopenia purpura (ITP)
Patients with uncontrolled autoimmune hemolytic anemia (AIHA) or autoimmune thrombocytopenia (ITP)
Uncontrolled autoimmune hemolytic anemia or thrombocytopenia
Uncontrolled autoimmune hemolytic anemia or thrombocytopenia
Uncontrolled autoimmune hemolytic anemia or idiopathic thrombocytopenic purpura (ITP) resulting in (or as evidenced by) declining platelet or hemoglobin (Hgb) levels within the 4 weeks prior to first dose of study drug
Uncontrolled autoimmune anemia or thrombocytopenia
Suffering from an autoimmune disease (including refractory immune thrombocytopenia-ITP or refractory autoimmune hemolytic anemia-AIHA)
Patients with autoimmune hemolytic anemia or autoimmune thrombocytopenia will be eligible for treatment
Has autoimmune cytopenia (anemia, thrombocytopenia, leukopenia).
Uncontrolled autoimmune hemolytic anemia or idiopathic thrombocytopenic purpura (ITP) resulting in (or as evidenced by) declining platelet or hemoglobin (Hgb) levels within the 4 weeks prior to first dose of study drug
total bilirubin ?1.5 X institutional ULN unless due to Gilbert's syndrome, controlled autoimmune hemolytic anemia or immune thrombocytopenia
Active autoimmune hemolytic anaemia (AIHA) and idiopathic thrombocytopenic purpura (ITP) requiring corticosteroid therapy greater than 25 mg prednisone (or equivalent) or chemotherapy.
Known active, uncontrolled autoimmune hemolytic anemia or immune thrombocytopenia requiring steroid therapy
Active, uncontrolled autoimmune phenomenon autoimmune hemolytic anemia, idiopathic thrombocytopenic purpura (AIHA, ITP) requiring steroid therapy
Subjects with Gilbert's syndrome or resolving autoimmune hemolytic anemia may have a bilirubin up to 3.0 x ULN and are still eligible
Uncontrolled autoimmune hemolytic anemia (AIHA) or idiopathic thrombocytopenia purpura (ITP)