Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

• Status: Recruiting
• Trial end date: 2022-08-01
• Target enrollment: 0
• Participant gender: All

Summary

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

• Phase: N/A
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Masonic Cancer Center, University of Minnesota
• Treatments: Alemtuzumab; Antilymphocyte Serum; Busulfan; Cyclophosphamide; Fludarabine; Fludarabine phosphate; Mesna; Vidarabine

Criteria

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