Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors

* INCLUSION CRITERIA RECIPIENT: Participants must fulfill one disease category (1 or 2) and 3 1. Patients with sickle cell disease at high risk for disease related morbidity or mortality, defined by having an end-organ damage (A, B, C, D, OR E) or complication(s) not ameliorated by sickle cell-specific therapies (F): A. Stroke defined as a clinically significant neurologic event that is accompanied by an infarct on cerebral MRI ORb B. Abnormal trans-cranial Doppler examination (\>=200 cm/s); OR C. Silent cerebral infarct defined as an infarct-like lesion based on an MRI signal abnormality at least 3 mm in one dimension and visible in two planes on FLAIR or T2- weighted images (or similar image with 3D imaging) and documented neurological examination performed by a neurologist demonstrating the participant has a normal neurologic examination, or an abnormality on examination that could not be explained by the location of the brain lesion(s); OR D. Sickle cell related renal insufficiency defined by a creatinine level \>=1.5 times the upper limit of normal and kidney biopsy consistent with sickle cell nephropathy OR nephrotic syndrome OR creatinine clearance \0.4 mg/dL at baseline; OR H. Vaso-occlusive crises: more than 1 hospital admission per year while on a therapeutic dose of sickle cell treatment /medication; OR I. Acute chest syndrome (ACS): any ACS while on sickle cell treatment /medication 2. Patients with beta-thalassemia who have grade 2 or 3 iron overload, determined by the presence of 2 or more of the following: * Portal fibrosis by liver biopsy * Inadequate chelation history (defined as failure to maintain adequate compliance with chelation with deferoxamine initiated within 18 months of the first transfusion and administered at least 5 days each week) * Hepatomegaly of greater than 2 cm below the costochondral margin or by other imaging scans 3. Non disease specific * Ages \>=4 years and less than 65 years old * Fully matched human leukocyte antigen (HLA) donors at A, B, C, and DR loci (8 of 8 or 10 of 10 * Ability to comprehend and willing to sign an informed consent, assent obtained from minors when applicable. Negative serum or urine beta-HCG, when applicable * Agree to use birth control throughout the study and 3 months after abatacept or sirolimus administration. * Female subjects must agree to use a medically acceptable method of birth control such as oral contraceptive, intrauterine device, barrier and spermicide, or implant/injection from start of screening until immunosuppression is stopped. * Male subjects must agree to use effective contraception (including condoms) from start of screening until immunosuppression is stopped. DONOR: * Fully matched human leukocyte antigen (HLA) donors at A, B, C, and DR loci (8 of 8 or 10 of 10) are intended for this study. * Donors age 4 or older and \>=15 kg (or weight deemed acceptable by IR for line placement, DTM for apheresis, and pediatric consult service) eligible to donate hematopoietic stem cells, are eligible for this study. * Donors will be evaluated in accordance with existing Standard NIH Policies and Procedures for determination of eligibility and suitability for clinical donation. Donors will sign on a separate protocol, 20-H-0099 NHLBI standard of care protocol for the mobilization and collection of HSCs. Note that participation in this study is offered to all eligible donors, but is not required for a donor to make a stem cell donation, so it is possible that not all donors will enroll onto this study. EXCLUSION CRITERIA RECIPIENT: * Karnofsky or Lanksy performance status of \

Source: ClinicalTrials.gov (NCT07599176). StuddyBuddy aggregates publicly available trial information.

Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors

Summary

Eligibility Criteria