New transplant approach offers hope for sickle cell patients without a perfect donor match

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion: 1. Confirmed diagnosis of hemoglobin SS or S-βº Thalassemia sickle cell disease 2. Severe disease status as defined by presence of one or more of the following: 1. Clinically significant neurologic event (stroke) or any neurological deficit lasting \> 24 hours; or increased transcranial Doppler velocity (\>200 m/s). A stroke is defined as a sudden neurologic change lasting more than 24 hours that is accompanied by cerebral magnetic resonance imaging (MRI) changes. 2. History of ≥ 1 episodes of acute chest syndrome (ACS) in the 2-year period preceding enrollment despite the institution of supportive care measures (i.e. asthma therapy and/or hydroxyurea). 3. History of ≥ 2 severe vaso-occlusive pain crises (VOC) per year in the 2-year period preceding enrollment despite the institution of supportive care measures (i.e. a pain management plan and/or treatment with hydroxyurea). A severe VOC is defined as an episode of pain lasting more than 2 hours severe enough to require care at a medical facility. Note that priapism that lasts more than 2 hours and requires care at a medical facility is also considered a VOC. 4. Osteonecrosis of ≥ 2 joints despite the institution of supportive care measures. 5. Prior treatment with regular RBC transfusion therapy, defined as receiving ≥ 8 transfusions per year for \> 1 year to prevent vaso-occlusive clinical complications (i.e. pain, stroke, and acute chest syndrome) 3. No HLA matched sibling or 10/10 matched unrelated donor 4. Related donor who: 1. Is genotypically haploidentical on HLA-A, B, C and DRB1 loci AND 2. Meets institutional criteria 5. Failed prior hydroxyurea therapy or have intolerance to hydroxyurea 6. Meets protocol specified organ function criteria 7. Women of childbearing potential or sexually active male: Agreement to use adequate contraception prior to study entry and 6 months post-transplant. Exclusion Criteria 1. Prior stem cell transplant 2. Prior bone marrow transplant 3. Concurrent other investigational agents, chemotherapy, biological therapy or radiation therapy 4. Planned use of moderate and strong CYP3A4 inhibitors 5. Active infection 6. Major surgery within the last 30 days 7. Clinically significant liver fibrosis or cirrhosis if on chronic transfusion therapy \> 6 months 8. Active malignancy (other than non-melanoma skin cancers) 9. History of allergic reactions attributed to compounds of similar chemical or biologic composition to any in the pre- or post-transplant regimen. 10. Women of childbearing potential: pregnant or breastfeeding