Table 2.

Outcomes prioritized by the guideline panel

Question	Secondary outcome of interest
Q1: Should individuals with SCD and neurologic injury (overt stroke, SCI, or abnormal TCD) undergo MSD transplantation?	1. Improvement/normalization of TCD velocity 2. Primary ischemic stroke 3. Secondary ischemic stroke 4. New or progressive SCI 5. New or progressive CNS vasculopathy 6. HRQOL 7. Engraftment kinetics
Q2: Should individuals with frequent pain requiring interventions by a health care provider undergo MSD HSCT vs standard of care?	1. Change in frequency of acute pain episodes requiring acute care 2. Change in hospitalization frequency 3. HRQOL 4. Engraftment kinetics
Q3: Should individuals with recurrent episodes of ACS undergo MSD HSCT vs standard of care?	1. Change in frequency of ACS 2. Change in hospitalization frequency 3. Resolution or improvement in chronic lung disease 4. HRQOL 5. Engraftment kinetics
Q4: Should individuals with SCD with an indication for HSCT (as above) who do not have an MSD undergo nonmyeloablative transplantation from alternative donor vs standard supportive care?	Same as Q1-3
Q5: Should individuals with SCD undergoing allogeneic transplantation receive a TBI-based regimen (low-dose TBI ≤400 cGy) or chemotherapy-based regimen?	Same as Q1-3 Additional outcome: potential for fertility post-HSCT
Q6: What is the optimal conditioning regimen for individuals with SCD who have an indication for HSCT and a matched sibling donor (myeloablative transplantation vs reduced intensity or nonmyeloablative transplantation)?	Same as Q 1-3 Additional outcome: Potential for fertility post-HSCT
Q7: Should age be a determining factor for HSCT with MSD for individuals with SCD with the above indication?	Same as above (Q6)
Q8: In pediatric patients with SCD undergoing matched related donor HSCT with available cryopreserved matched sibling cord blood use the cord blood or BM as donor source?	Same as above (Q6)

CNS, central nervous system; HRQOL, health-related quality of life.