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Abatacept for GVHD Prophylaxis After Hematopoietic Stem Cell Transplant for Pediatric Sickle Cell Disease: A Sickle Transplant Alliance for Research Trial


Abatacept for Graft Versus Host Disease Prophylaxis After Hematopoietic Stem Cell Transplantation for Pediatric Sickle Cell Disease: A Sickle Transplant Alliance for Research Trial

Therapeutic Area Sickle Cell Disease
Principal Investigator Jason Law MD
Min Age 3 Years
Max Age 20 Years
Gender Any
Contact Erica Heiden
More Information


To assess the tolerability of the costimulation blocking agent abatacept (CTLA4-lg) when added to the standard graft versus host disease (GVHD) prophylaxis regimen of a calcineurin inhibitor and methotrexate in patients receiving early alemtuzumab followed by fludarabine, thiotepa, melphalan, and alemtuzumab for conditioning. 

Study Details

Inclusion Criteria

  • All patients and/or their parents or legal guardians must sign a written informed consent. Asset, when appropriate, will be obtained according to institutional guidelines. 
  • Must have been evaluated and adequately counseled regarding treatment options for severe SCD by a pediatric hematologist.
  • Because of the elective and non-urgent nature of hematopoietic stem cell transplantation (HSCT) for SCD, it is important that all the patients and families be counseled regarding fertility preservation measures available to them. All patients and/or their parents or legal guardians must indicated on the consent and assent forms that they have received this counseling.

Exclusion Criteria

  • Bridging (portal to portal) fibrosis or cirrhosis of the liver.
  • Parenchymal lung disease stemming from SCD or other process defined as a diffusing capacity of the lungs for carbon monoxide (DCLO) (corrected for hemoglobin) or forced vital capacity of less than 45% of predicted. Children unable to perform pulmonary function testing will be excluded if they require daytime oxygen supplementation.
  • Renal dysfunction with an estimated glomerular filtration rate (GFR) < 50% of predicted normal for age. 

Study Requirements

Pre-admission evaluations will include: A history and physical, Hb-electrophoresis CBC and blood chemistries, GFR, Rh typing, Echocardiography, pulmonary function testing, MRI of the brain, urine analysis, HLA typing. Alemtuzumab blood levels will also be calculated based on a 2mL whole blood sample. 

Post-transplant evaluations will include: Graft cell doses, study visits every 30 days until 180 days, then at day 270, then every year until 5 years post-transplant, and all pre-admission evaluations.