This trial will determine whether adding ruxolitinib to a reduced intensity conditioning (RIC) regimen reduces the rate of graft failure following haploidentical (haplo) hematopoietic cell transplant (HCT) for children and young adults with sickle cell disease (SCD).
This study will enroll and treat up to 24 participants. Recruitment is expected to last for about 2 years and participants will be followed for an additional 2 years post-HCT.
History of stroke and/or vasculopathy, including evidence of asymptomatic cerebrovascular disease for pediatric patients.
Recurrent moderate-severe acute chest syndrome (ACS)
Recurrent vaso-occlusive pain episodes requiring parenteral analgesia despite the institution of supportive care.
Need for chronic transfusion therapy to prevent vaso-occlusive complications (i.e. pain, stroke, and ACS).
Presence of an HLA-matched sibling who is willing and able to donate bone marrow.
Uncontrolled infection, evidence of active TB, Hepatitis B or C infection, or HIV seropositivity or infection.
Previous HCT or solid organ transplant.
CNS revascularization procedure, myocardial infarction, pulmonary embolus or deep vein thrombosis in the past 6 months.
Single group
Ruxolitinib
DrugAll participants will receive ruxolitinib beginning during conditioning in addition to conventional RIC and GVHD prophylaxis.
Event Free Survival
Event Free Survival (EFS) is defined as survival without a qualifying event (primary or secondary GF, second HCT or death).
Overall Survival
Overall survival will be described at 1 and 2 years post-HCT including death from any cause after HCT.
Event Free Survival
Event Free Survival (EFS) is defined as survival without a qualifying event (primary or secondary GF, second transplant or death).
Neutrophil Recovery
The time to neutrophil recovery, in days, will be reported. Neutrophil recovery is defined as the first of 3 measurements on different days when the absolute neutrophil count is ≥500/μL after nadir.
Platelet Recovery
The time to platelet recovery, in days, will be reported. Platelet recovery is defined as the first day the platelet count is ≥50,000/μL of blood, without a transfusion in the preceding 7 days with the exception of a platelet transfusion specifically to achieve a platelet threshold to allow an elective invasive procedure.
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Arkansas Children's Hospital Research Institute
Lead sponsor
Children's Healthcare of Atlanta
Collaborator
Emory University
Collaborator
Incyte Corporation
Collaborator
