Table 1.
Results of allogeneic transplantation in the treatment of Wiskott-Aldrich syndrome published 2000–2013
| Author (publication year) | Study period and follow-up | Patients | Donors | Conditioning | GVHD and survival | Other |
|---|---|---|---|---|---|---|
| Mitchell R, et al, 201339 | 1992–2008 | N=27 | aGVHD 31% cGVHD 25% 5 yr OS 81% |
|||
| Stepensky P, et al, 201338 | 1996–2011 Median follow-up 3.4 yrs (0.4–12.5) |
N=14 Median age at HCT 3.25 yrs (0.16–6.6) |
MRD, N=5 URD, N=3 UCB, N=4 Haplo, N=2 |
BU/CY, N=2 BU/CY/ATG, N=4 Other, N=8 |
aGVHD (II–IV), N=4 cGVHD, N=2 5 yr OS 92% 5 yr DFS 59.5% |
Reject, N=3 Mixed chimerism, N=2 Autoimmunity, N=2 |
| Shin CR, et al, 201240 | 1990–2009 | N=47 Median age at HCT 1990–2000 2.3 yrs 2001–2009 1.5 yrs |
MRD, N=7 URD, N=31 UCB, N=8 Haplo, N=l |
BU/CY/ATG, N=40 Other MAC, N=6 Other RIC, N=1 |
aGVHD (II–IV) 40% cGVHD, 21% 2001–2009/1990–2000 5 yr OS 90.8%/62.5% |
Reject, N=2 Mixed chimerism, N=5 Viral infection 72% Autoimmunity 55% |
| Morio T, et al, 201141 | 1998–2008 | N=23 Median age at HCT 14 months (4–84) |
UCB, N=23 | MAC, N=21 RIC, N=2 |
aGVHD, (II–IV) 47% 5 yr OS 82% |
Viral infection, N=6 |
| Moratto D, et al, 201135 | 1980–2009 | N=194 Median age at HCT 34.6 months (2–240) |
MSD 20.1% MRD 2.6% MMRD 18% URD 46.9% UCB 12.4% |
88.1% MAC 11.9% RIC |
aGVHD N=22, 11.3% cGVHD N=11 OS 84% |
Reject, N=13 Autoimmunity, N=27 |
| Mahlaoui N, et al, 201216 | Median follow-up 6 yrs (1.5–26) |
N=22 Median age at HCT 15.5 months (5–32) |
Haplo, N=12 URD, N=7 MRD, N=3 |
aGVHD N=3 cGVHD N=3 OS 77% |
Viral infection, N=4 Autoimmunity, N=6 |
|
| Friedrich W, et al, 200942 | Since 1983 Mean follow-up 11 yrs |
N=39 Mean age at HCT 1.7 yrs |
MUD, N=15 Haplo, N=15 MSD, N=9 |
BU/CY, N=8 BU/CY/ATG, N=5 Flu/BU/CY/ATG, N=8 Other, N=18 |
GVHD, N=4 cGVHD, N=3 OS 90% MSD/MRD OS 50% Haplo |
Reject, N=8 Mixed chimerism, N=2 Viral infection, N=5 Autoimmunity, N=1 |
| Al-Ghonaium A, et al, 200843 | 1993–2006 | N=14 | HLA matched, N=z10 UCB, N=4 |
OS 100% HLA matched OS 50% UCB |
||
| Ozsahin H, et al, 200834 | 1979–2001 Median follow-up 7 yrs (2–25) |
N=96 Median age at HCT 2 yrs (0.3–17.7) |
MRD, N=45 URD, N=32 MMRD, N=19 |
BU/CY, N=85 Other, N=11 |
aGVHD, N=7 cGVHD, N=7 7 yr EFS 75% overall EFS by donor MSD/URD/MMRD 88%/7l%/55% |
Mixed chimerism N=18 Autoimmunity N=19 |
| Munoz A, et al, 200747 | 1989–2006 Median follow-up 101 months (8–204) |
N=13 | MRD, N=3 URD, N=9 Haplo, N=1 |
BU/CY, N=3 BU/CY/ATG, N=8 Other, N=2 |
aGVHD (II–IV), N=4 cGVHD, N=2 OS, 9/13 alive |
Reject, N=l Mixed chimerism, N=2 |
| Pai SY, et al, 200649 | 1990–2005 | N=23 | MRD, N=5 URD, N=16 Haplo, N=2 |
BU/CY, N=6 BU/CY/ATG, N=13 Other MAC, N=4 |
aGVHD (II–IV), N=1 cGVHD, none OS 78.2% entire cohort |
Mixed chimerism, N=6 Viral infection, N=8 Autoimmunity, N=3 |
| Kobayashi R, et al, 200648 | 1985–2004 | N=57 Median age at HCT 1.6 yrs (0.4–19) |
MRD, N=11 MMRD, N=10 URD, N=21 UCB, N=15 |
BU/CY/ATG, N=20 BU/CY, N=13 XRT containing, N=14 Other, N=10 |
aGVHD (II–IV) 36.8% cGVHD 40.4% 5 yr OS 73.7% 5 yr FFS 65.7% |
Reject, N=6 Mixed chimerism, N=1 |
| Antoine C, et al, 200345 | 1968–1999 Median follow-up 7 years (all non-SCID) |
N=103 | MSD, N=33 MFD, 5 MMRD, N=45 URD, N=20 |
BU/CY (all but 10 non-SCID patients) | 3 yr OS 81% MSD 3 yr OS 43% MMRD |
|
| Filipovich AH, et al, 200144 | 1968–1996 Median follow-up 42 months (2–304) |
N=170 Median age at HCT MSD 3 yrs (<1–16) MFD 2 yrs (<1–17) URD 2 yrs (<1–32) |
MSD, N=55 MFD, N 48 MUD, N=67 |
82% no TBI 77% non-TCD |
5 yr OS 70% By donor: MSD 87%, MFD 52%, MUD 71% |
Reject, N=6 Viral infection, N=3 |
Abbreviations: HCT, hematopoietic cell transplantation; MSD, matched sibling donor; MFD, matched family donor; MMRD, mismatched related donor; MUD, matched unrelated donor; URD, related donor; BM, bone marrow; UCB, umbilical cord blood; Haplo, haploidentical; BU, busulfan; CY, Cytoxan; ATG, anti-thymocyte globulin; XRT, radiation; TBI, total body irradiation; MAC, myeloablative conditioning; RIC, reduced intensity conditioning; OS, overall survival; EFS, event free survival; FFS, failure free survival; aGVHD, acute graft-versus-host-disease; cGVHD, chronic graft-versus-host disease; yr, year; MRD, matched related donor; DFS, disease free survival; Flu, fludarabine; SCID, severe combined immunodeficiency; TCD, T-cell depleted.