Table 2.
Conventional and reduced intensity conditioning before hematopoietic cell transplantation in adults with sickle cell disease
| Center | Preparative regimen | GVHD-prophyl | n | Age range (years) | Published outcomes
|
Latest follow-up
|
|||||
|---|---|---|---|---|---|---|---|---|---|---|---|
| Follow-up (years) | Death | Complications | Most recent follow-up (years) | IST | GVHD | % Donor chimerism | |||||
| Pittsburgh (Krishnamurti et al., 2008) [30] | BU 8 mg/kg, Flu 135 mg/m2, ATG 120 mg/kg, TLI 500 cGy | CsA, MMF | 3 | 16–18 | 1.5–3.5 | None | Pancreatitis. (n =1); GR (n =1) | 5–6 | 1 of 3 | 1 of 3 (limited cGVHD) | 0–100% |
|
| |||||||||||
| France (Kuentz et al., 2011) [26] | BU 14 mg/kg, CY 200 mg/kg, rATG 10 mg/kg | CsA, MTX | 15 | 16–27.5 | 1–16.1 | 1 (ICH) | SDH (n =1), seizures | – | – | Eight acute GVHD, two chronic GVHD | 75–100% |
|
| |||||||||||
| NHLBI (Hsieh et al., 2009, 2014) [31■■,32] | Alem 1 mg/kg, TBI 300 cGy | Sirolimus | 30 | 17–65 | 1.8–6 | 1 (ICH) after GR | GR (n =4) | 3.6, median | 11 of 26 | None | Median T cell 48% |
Alem, alemtuzumab; ATG, antithymocyte globulin; BU, busulfan; cGVHD, chronic GVHD; CsA, cyclosporine; CY, cyclophosphamide; Flu, fludarabine; Flu, fludarabine; GR, graft rejection; GVHD, graft-versus-host disease; ICH, intracranial hemorrhage; IST, immunosuppressive therapy; MMF, mycophenolate mofetil; mos, months; MTX, methotrexate; prophyl, prophylaxis; rATG, rabbit antithymocyte globulin; SDH, subdural hematoma; TAM, transplant-associated microangiopathy; TLI, total lymphoid irradiation.