30 closest matches · ranked by relevance
Sickle Cell Disease · Thalassemia · Diamond-Blackfan Anemia
Primary: Count of Participants With Stable Engraftment Post Hematopoietic Cell Transplantation (HCT) — 3 Participants
Acute Undifferentiated Leukemia · Adult Acute Lymphoblastic Leukemia in Remission · Adult Acute Myeloid Leukemia in Remission
Primary: Changes in Mean Neutrophil Values (as Measured by Lab) for Arm 1 (Other Arms Were Used for Calibration Only) — 7.8 10^9 Neutrophils per Liter
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
Sickle Cell Anemia · Severe Aplastic Anemia · Paroxysmal Nocturnal Hemoglobinuria (PNH)
Primary: Number of Patients With Neutrophil Engraftment — 2 participants
Adrenoleukodystrophy · Metachromatic Leukodystrophy · Globoid Cell Leukodystrophy
Primary: Number of Patients With Donor Cell Engraftment — 26 Participants
Dyskeratosis Congenita · Aplastic Anemia
Primary: Incidence of Neutrophil Engraftment — 100; 100; 96; 100 Percentage of participants
Hematologic Malignancy
Primary: Overall Survival — 79; 49 Participants
Sickle Cell Disease
Primary: Percentage of Participants With Event-Free Survival (EFS) — 76; 69 percentage of participants
Sickle Cell Disease & Thalassemia
Primary: Safety and Tolerability Will be Measured by Acute NiCord® Infusional Toxicity. — 0; 0; 3; 0 participants
Hemoglobinopathies · Sickle Cell Disease · Thalassemia
Primary: Stable Engraftment With Donor Stem Cells in Patients With Severe Hemoglobinopathy. — 2 participants
Anemia, Aplastic · Red-Cell Aplasia, Pure · Anemia, Diamond-Blackfan
Primary: Response to Rituximab — 0; 2; 9 participants
Multiple Myeloma · Amyloidosis
Primary: Number of Participants Without Renal Allograft Rejection at 6 Months Post-transplant — 1 Participants
Sickle Cell Disease
Primary: Number of Emergency Department (ED)/Acute Care Visits — 0; 1 number of visits
Sickle Cell Disease · Thalassemia · Severe Congenital Neutropenia
Primary: Number of Patients Who Experienced Grade 3-5 Treatment Related Toxicity — 0; 0; 2 Participants
Thrombotic Microangiopathies · Atypical Hemolytic Uremic Syndrome · Multiple Organ Dysfunction Syndrome
Primary: Survival — 15 Participants — p=<0.0001
Sickle Cell Disease · Beta Thalassemia-Major
Primary: Number of Participants With Sustained Cell Engraftment of Donor Cells — 29 Participants
Blood Platelet Disorders
Primary: Response to Drug Based on Platelet Count at the End of Therapy — 91.6 percentage of participants
Hematological Neoplasms · Hematopoietic Stem Cell Transplantation
Primary: Number of Participants Experiencing Transplant Related Mortality (TRM) — 0 Participants
Sickle Cell Disease
Primary: % of Patient-years With Malignancies — 0.001 % patient-years
Leukemia · Lymphoma
Primary: Number of Participants With Absolute Neutrophil Count Engraftment — 21 participant
Hematologic Malignancies
Primary: Number of Participants With Successful Bone Marrow Engraftment — 1; 15; 1 Participants
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
Sickle Cell Disease · Thalassemia · Hemoglobinopathies
Primary: Development of GVHD Within 1 Year of BMT — 2 participants
Hematological Malignancies
Primary: Overall Survival at 6 Months Post-transplant in Patients Receiving a Partially-matched Related Donor Allogeneic Transplant After Reduced-intensity Conditioning — 22…
Lysosomal Storage Disease · Peroxisomal Disorder
Primary: Donor (Allogeneic) Hematopoietic Engraftment — 1 participants
Thrombocytopenia · Hematologic Diseases
Primary: Number of Participants With Platelet Count Recovery — 20; 22; 6 Participants
Acute Biphenotypic Leukemia · Acute Lymphoblastic Leukemia · Acute Myeloid Leukemia
Primary: Overall Survival — 28; 15; 53; 7 Participants
Sickle Cell Disease · Sickle Cell Anemia
Primary: Mean Red Blood Cells Lifespan in Participants — 64.1; 113.4; 126.0; 123.7 day — p=<0.001
Sickle Cell Disease
Primary: Number of Participants With Sufficient Collection of Hemopoietic Stem Cells (HSCs) Without Serious Adverse Events — 14 Participants
Congenital Pediatric Disorders
Primary: Overall Survival at 100 Days After Umbilical Cord Blood Transplant in Pediatric Patients. — 0.947 probability of overall survival