Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

Masonic Cancer Center, University of Minnesota38 enrolled

Overview

This is a study to collect the outcomes of stem cell transplantation for patients with hematologic diseases other than cancer.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Sickle Cell Disease Transfusion Dependent Alpha- or Beta- Thalassemia Diamond Blackfan Anemia Paroxysmal Nocturnal Hemoglobinuria Glanzmann Thrombasthenia Severe Congenital Neutropenia

Interventions

Reduced Toxicity Ablative RegimenDRUG

* Anti-thymocyte Globulin (ATG) * Fludarabine * Busulfan * Stem Cell Infusion Day 0

Reduced Intensity Preparative RegimenDRUG

* Alemtuzumab * Cyclophosphamide * Fludarabine * Total Body Irradiation (TBI) * Stem Cell Infusion Day 0

Myeloablative Preparative RegimenDRUG

* Alemtuzumab * Cyclophosphamide * Busulfan * Stem Cell Infusion Day 0

Eligibility

Sex: ALLMax age: 55 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Diagnosis of Sickle Cell Disease, Thalassemia, Diamond Blackfan Anemia or other non-malignant hematologic disorders for which a stem cell transplant is indicated * Acceptable stem cell source identified * Performance status of ≥ 70% (Karnofsky),or ≥ 70 (Lansky play score) * Creatinine \<2.0 mg/dl for adults or glomerular filtration rate \> 50 ml/min for children * Bilirubin, Aspartate Aminotransferase, Alkaline phosphatase \<5 times the upper limit of institutional normal * Absence of decompensated congestive heart failure, or uncontrolled arrhythmia and left ventricular ejection fraction \> 40% Exclusion Criteria: * active, uncontrolled infection * pregnant or breastfeeding * HIV positive

Outcomes

Primary Outcomes

incidence of graft failure

Time frame: 42 days

Secondary Outcomes

overall survival

Time frame: 6 months, 1 and 2 years

disease free survival

patient no longer needing red blood cell transfusion and/or a hemoglobin S level at that of the donor ( sickle cell disease only)