30 closest matches · ranked by relevance
Autoimmune Pancytopenia · Autoimmune Lymphoproliferative Syndrome (ALPS) · Evans Syndrome
Primary: Number of Participants With Grade 3 and 4 Toxicities of Administration of Oral Sirolimus — 1 participants
Anemia, Aplastic · Red-Cell Aplasia, Pure · Anemia, Diamond-Blackfan
Primary: Response to Rituximab — 0; 2; 9 participants
Severe Aplastic Anemia, Refractory · Severe Aplastic Anemia, Relapse
Primary: Number of Participants With Hematological Response at 6 Months — 0; 18 Participants
Diarrhea · Gastrointestinal Complications · Unspecified Childhood Solid Tumor, Protocol Specific
Primary: Number of Participants With Treatment-free Remission at 1 Year After Study Completion — 2 Participants
Immunosuppresion · Thrombocytopenia · Pancytopenia
Primary: Hematologic Response — 38; 20; 3 Participants
Aplastic Anemia
Primary: Participants no Longer Meeting Criteria for Severe Aplastic Anemia. — 18; 17; 9; 9 participants
Autoimmune Hemolytic Anemia
Primary: Percentage of Participants Attaining a Complete Response at Any Visit From Week 6 to Week 12 — 20.0; 40.0 percentage of participants
Anemia, Aplastic · Amegakaryocytic Thrombocytopenia · Diamond-Blackfan Anemia
Primary: Treatment Failures
Myelodysplastic Syndrome · Aplastic Anemia
Primary: Overall Response — 8; 8; 4 participants
Aplastic Anemia
Primary: Patients Treated With Rabbit Antithymocyte Globulin (r-ATG/Thymoglobulin) and Cyclosporine (CsA) Achieving at Least a Partial Remission (PR) at 6 Months — 9 participants
Vitamin B12 Deficiency
Primary: Prevalence of Vitamin B12 Deficiency — 3; 6; 4 participants
Paraneoplastic Syndromes
Primary: Survival of Patients With Paraneoplastic Disease Who Are Treated With Tacrolimus — 48 months
Aplastic Anemia · Neutropenia · Pancytopenia
Primary: Blood Counts and Adverse Event Profile After 6 Months of Treatment. — 4; 5; 12 participants
Gaucher Disease
Primary: Number of Patients With GD Diagnosis Confirmed by : Enzyme Testing of acidβ-glucosidase Activity Activity <15% in Blood Leucocytes Completed When Necsssary by GB1…
Moderate Aplastic Anemia · Unilineage Bone Marrow Failure Disorders
Primary: Proportion of Drug Responders — 17 Participants
Anemia, Aplastic · Anemia, Hypoplastic · Thrombocytopenia
Primary: The Portion of Drug Responders as Defined by Hematologic Improvements — 17 Participants
Aplastic Anemia
Primary: Number of Patients With Engraftment Response — 9 Participants
Interstitial Lung Disease · Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis
Primary: Death From All Causes — 10; 5 Participants
Aplastic Anemia · Neutropenia · Pancytopenia
Primary: Response Rate at 6 Months — 1; 0; 0 participants
Autoimmune Pulmonary Alveolar Proteinosis
Primary: Time to Rescue WLL — 18; 30 Months — p=0.0078
Chronic Inflammatory Demyelinating Polyneuropathy
Primary: Number of Participants With Survival — 66; 64 Participants
Hemophagocytic Lymphohistiocytosis · Chronic Active Epstein-Barr Virus Infection · Chronic Granulomatous Disease
Primary: Percentage of Participants With Overall Survival (OS) — 80.4; 66.7 percentage of participants
Thrombocytopenia · Hematologic Diseases
Primary: Number of Participants With Platelet Count Recovery — 20; 22; 6 Participants
SYSTEMIC SCLERODERMA
Primary: Time to Treatment Failure — 0; 8 participants
Pemphigus
Primary: Number of Patients Achieving a Short- and Long-term Remission of Pemphigus — 23 Participants
Idiopathic Pulmonary Fibrosis, Acute Fatal Form
Primary: %Survival — 44; 7 percentage of participants
Dyskeratosis Congenita · Aplastic Anemia
Primary: Incidence of Neutrophil Engraftment — 100; 100; 96; 100 Percentage of participants
Myelodysplastic Syndrome (MDS) With Refractory Anemia (RA) · Severe Aplastic Anemia (SAA)
Primary: Number of Participants Who Engrafted by Day 42 — 30 Participants
Anemia, Aplastic · Anemia, Hypoplastic
Primary: Number of Participants With Complete Response at 3 Months. — 0 Participants
Fanconi Anemia
Primary: Number of Participants Experiencing Graft Failure — 0; 0; 0 Participants