Ruxolitinib-Enhanced Conditioning for Pediatric and Young Adult Patients With Symptomatic Sickle Cell Disease Undergoing Haploidentical Hematopoietic Cell Transplantation
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.
• Participants with any genotypic form of SCD aged 12 - 45 years at enrollment with ≥1 of the following:
‣ 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).
⁃ For adult patients, an echocardiographic finding of tricuspid valve regurgitant jet velocity (TRJV) ≥ 2.7 m/sec.
• Participants must have an HLA haploidentical first degree relative (parent, sibling, or half sibling) who is willing and able to donate bone marrow.
• Participants must meet institutional eligibility criteria for HCT.