Indications for HSCT in SCD
| Matched sibling donor (if available) . | Matched URD transplant . | Mismatched marrow donor, haploidentical donor, unrelated cord blood transplant . |
|---|---|---|
| Consider early,* prior to or at onset of SCD symptoms, with the highest priority given to patients with HbSS and HbSβ0 thalassemia | Stroke | Recurrent stroke despite adequate chronic transfusion therapy; progressive CNS changes |
| Stroke | Elevated TCD velocity | Severe SCD symptoms and inability to tolerate supportive care resulting in symptom persistence/progression |
| Elevated TCD velocity | Recurrent acute chest syndrome despite supportive care | |
| Recurrent acute chest syndrome despite supportive care | Recurrent severe VOE despite supportive care | |
| Recurrent severe VOE despite supportive care | Red cell alloimmunization despite intervention + established indication for chronic transfusion therapy | |
| Red cell alloimmunization despite intervention + established indication for chronic transfusion therapy | Pulmonary hypertension | |
| Pulmonary hypertension | Recurrent priapism | |
| Recurrent priapism | Sickle nephropathy | |
| Sickle nephropathy | Bone and joint involvement | |
| Bone and joint involvement | ||
| Sickle retinopathy |
| Matched sibling donor (if available) . | Matched URD transplant . | Mismatched marrow donor, haploidentical donor, unrelated cord blood transplant . |
|---|---|---|
| Consider early,* prior to or at onset of SCD symptoms, with the highest priority given to patients with HbSS and HbSβ0 thalassemia | Stroke | Recurrent stroke despite adequate chronic transfusion therapy; progressive CNS changes |
| Stroke | Elevated TCD velocity | Severe SCD symptoms and inability to tolerate supportive care resulting in symptom persistence/progression |
| Elevated TCD velocity | Recurrent acute chest syndrome despite supportive care | |
| Recurrent acute chest syndrome despite supportive care | Recurrent severe VOE despite supportive care | |
| Recurrent severe VOE despite supportive care | Red cell alloimmunization despite intervention + established indication for chronic transfusion therapy | |
| Red cell alloimmunization despite intervention + established indication for chronic transfusion therapy | Pulmonary hypertension | |
| Pulmonary hypertension | Recurrent priapism | |
| Recurrent priapism | Sickle nephropathy | |
| Sickle nephropathy | Bone and joint involvement | |
| Bone and joint involvement | ||
| Sickle retinopathy |
For all genotypes, the morbidity of the disease is the driving factor in pursuing a HSCT. Preventative HSCT should be considered for children with higher-risk genotypes, HbSS, and HbSβ0. HSCT for adults with SCD is better tolerated with a low-intensity regimen, with the caveat of requiring prolonged immune suppression to maintain mixed-donor chimerism.
AVN, avascular necrosis; TCD, transcranial Doppler; VOE, veno-occlusive episodes.
Especially in children with difficult access to adequate lifelong supportive medical care, we recommend reviewing statistics for OS, DFS, GR, and GVHD with families as they weigh these options.