Table 5. Hypothetical patient profiles with varying age, donor type, and disease severity.
Data | Patient 1 | Patient 2 | Patient 3 |
---|---|---|---|
Patient data | |||
Age at transplant (year) | 6 | 6 | 16 |
Age group (year) | ≤10 | ≤10 | 11-17 |
Sex | Male | Male | Male |
KPS or Lanskya score | <90 | <90 | <90 |
HCTb-Comorbidity index (in range) | 0-2 | 0-2 | 0-2 |
Recipient CMVc serostatus | Negative | Negative | Negative |
Transplant data | |||
Donor | HLAd identical sibling | HLA mismatch relative | HLA identical sibling |
Graft type | Bone marrow | Bone marrow | Bone marrow |
Donor-recipient HLA matching | 8/8 | 7/8 | 8/8 |
Conditioning intensity | Myeloablative | Nonmyeloablative | Myeloablative |
Conditioning regimen | Flu/Bue | TBI/Cy/Flu/TTf | Flu/Bu |
Serotherapy | ATGg | ATG | ATG |
GVHDh prophylaxis | CNI+MTXi | Post-Cy+Siro± MMFj | CNI+MTX |
SCDk data | |||
Number of ACSl syndromes within 2 year pre-HCT | 0 | 0 | 2 |
Require exchange transfusion | No | No | No |
VOCm requiring hospitalization within 2 year pre-HCT | No | No | Yes |
Frequency of hospitalizations for VOC | <3 per year | <3 per year | <3 per year |
KPS: Karnofsky performance status.
HCT: hematopoietic cell transplantation.
CMV: cytomegalovirus.
HLA: human leukocyte antigen.
Flu/Bu: fludarabine + busulfan.
TBI/Cy/Flu/TT: total body irradiation + cyclophosphamide + fludarabine + thiotepa.
ATG: anti-thymocyte globulin.
GVHD: graft-versus-host disease.
CNI+MTX: calcineurin inhibitor + methotrexate.
Post-Cy+Siro± MMF: post-transplant cyclophosphamide + sirolimus + mycophenolate mofetil.
SCD: sickle cell disease.
ACS: acute chest syndrome.
VOC: vaso-occlusive crisis.