Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

  • End date
    Aug 22, 2023
  • participants needed
  • sponsor
    Masonic Cancer Center, University of Minnesota
Updated on 22 April 2022
stem cell transplantation
total body irradiation
anti-thymocyte globulin
ejection fraction
blood disorder
cell transplantation
antithymocyte globulin
preparative regimen
diamond-blackfan anemia
sickle cell anemia


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

Condition Sickle Cell Disease, Transfusion Dependent Alpha- or Beta- Thalassemia, Diamond Blackfan Anemia, Paroxysmal Nocturnal Hemoglobinuria, Glanzmann Thrombasthenia, Severe Congenital Neutropenia, Shwachman-Diamond Syndrome, Non-Malignant Hematologic Disorders
Treatment Reduced Toxicity Ablative Regimen, Reduced Intensity Preparative Regimen, Myeloablative Preparative Regimen
Clinical Study IdentifierNCT02179359
SponsorMasonic Cancer Center, University of Minnesota
Last Modified on22 April 2022


Yes No Not Sure

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
Clear my responses

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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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