Summary of published allo-HSCT outcome data for adult (and adolescent) PID patients
| Reference . | No. of patients . | PID subtype . | Age at HSCT, y (range) . | Donor (no.) . | Conditioning . | OS . | EFS . | TRM . | Engraftment . | Median follow-up (range) . |
|---|---|---|---|---|---|---|---|---|---|---|
| Albert et al 20184 | 18 | 6 CGD 12 other PID | 18 y (15-22) | MRD (2) MUD (5) 1Ag MMUD (11) | Full Bu/Cy (2); Full Bu/Flu (1); Sub Bu/Flu (7); Flu/Mel ±TT (1); Treo/Flu ±TT (7). All had serotherapy. | 94% | 94% | 6% | 100% | 5 y (2-9 y) |
| Fox et al 20185 | 29 | 11 CGD 18 other PID | 24 y (17-50) | MRD (11) MUD (13) 1Ag MMUD (5) | Flu/Mel/Alem (20) Flu/Bu/ATG (8) Flu/Bu/Alem (1) | 89% at 1yr 85% at 3yrs | 90% | 14% | 100% | 3.5 y (4 mo to 12 y) |
| Jin et al 201818 | 8 | Primary HLH | 25 y (18-54)* | HaploID (6) MUD (2) | TBI/VP16/Cyclo (6); VP16/Flu/Bu/ATG (2) | 88% | NS | 12% | 100% | 27 mo |
| Leiding et al 201819 | 5 (AYA) 10 (ped <12 y) | STAT1 GOF | 29 y (18-35) 8 y (1-17) | MRD (4); MUD (8); MMUD (1); HaploID (2)‡; UCB (2, 2‡). | Flu/Mel/Alem (4); Bu/Cy (3); Flu/Bu or Treo/ATG or Alem (6); Various other (4‡) | 20% at 1yr (AYA) 60% at 1yr (ped) 40% at 1yr (all) | NS NS NS | NS | 50%† | NS |
| Parta et al 201720 | 17 (AYA) 20 (ped) | CGD | 24 y (18-32) 8 y (4-17) | MRD (6); MUD (30); MMUD (1) | Bu/Alem (6); Bu/TBI/Alem (31) | 82.5% (all) | 80% (all) | 17.5% (all) | 85% (all) | 3.4 y (range, NS) |
| Shah et al 201721 | 7 | DOCK8 Defic | 20 y (7-25) | HaploID (7) | Flu/Bu/Cy + low dose TBI (7) | 86% (all) | NS | 14% | 100% | 2 y (3 mo to 5.7 y) |
| Fu et al 201622 | 30 | 1° HLH; EBV- HLH; Tu-HLH; Undef- HLH | 23 y (14–52) 19 y (14–55) 24 y (14–44) 29 y (16–32) | HaploID (23); MRD (6); MUD (1). | Bu/Cyclo/VP16 (6) for MRD TBI/Cyclo/VP16 + ATG (24) for HaploID/MUD | 63.3% at 2 y (100% in 1° HLH; 64% in EBV-HLH; 17.7% in Tu-HLH; 25% in Undef) | NS | 100% | 26 mo | |
| Wehr et al 201511 | 14 (adult) 11 (ped) | CVID | 34 y (18-50) 14 y (8-17) | MRD (14); MUD (10); MMUD (1) | BCNU/Flu/Mel (5); Flu/Mel (7); Flu/Mel/Treo (2); Flu/Bu ±TT (4); Bu/Cy ±AraC (6); Flu/Cy (1). | 57% (adults) 52% (all patients) | 44% at 1 y (all) | 79% (adult) | NS | |
| Grossman et al 201423 | 14 | GATA2 Defic | 33 y (15-46) | MRD (4); MUD (4); UCB (4); HaploID (2). | Flu/low dose TBI (8); Flu/Cy/low dose TBI (6). | 57% (all) | NS | 28% | 100% | 3.5 y (1-5 y) |
| Gungor et al 201424 | 13 (adult) 43 (ped) | CGD CGD | 21 y (18-39) 9 y (0.8 – 17) | MRD (21); MUD (25); MMUD (10). | Flu/Bu ATG or Alem (all). | 92% (adult) 96% (all) | 91% (all) | 7% | 93% (adult) | 21 mo |
| Spinner et al 201425 | 21 | GATA2 Defic | NS (15-49 y) | NS | NS | 72% at 1 y; 65% at 2 y; 54% at 4 y | NS | NS | NS | 14 mo (0-180 mo) |
| Reference . | No. of patients . | PID subtype . | Age at HSCT, y (range) . | Donor (no.) . | Conditioning . | OS . | EFS . | TRM . | Engraftment . | Median follow-up (range) . |
|---|---|---|---|---|---|---|---|---|---|---|
| Albert et al 20184 | 18 | 6 CGD 12 other PID | 18 y (15-22) | MRD (2) MUD (5) 1Ag MMUD (11) | Full Bu/Cy (2); Full Bu/Flu (1); Sub Bu/Flu (7); Flu/Mel ±TT (1); Treo/Flu ±TT (7). All had serotherapy. | 94% | 94% | 6% | 100% | 5 y (2-9 y) |
| Fox et al 20185 | 29 | 11 CGD 18 other PID | 24 y (17-50) | MRD (11) MUD (13) 1Ag MMUD (5) | Flu/Mel/Alem (20) Flu/Bu/ATG (8) Flu/Bu/Alem (1) | 89% at 1yr 85% at 3yrs | 90% | 14% | 100% | 3.5 y (4 mo to 12 y) |
| Jin et al 201818 | 8 | Primary HLH | 25 y (18-54)* | HaploID (6) MUD (2) | TBI/VP16/Cyclo (6); VP16/Flu/Bu/ATG (2) | 88% | NS | 12% | 100% | 27 mo |
| Leiding et al 201819 | 5 (AYA) 10 (ped <12 y) | STAT1 GOF | 29 y (18-35) 8 y (1-17) | MRD (4); MUD (8); MMUD (1); HaploID (2)‡; UCB (2, 2‡). | Flu/Mel/Alem (4); Bu/Cy (3); Flu/Bu or Treo/ATG or Alem (6); Various other (4‡) | 20% at 1yr (AYA) 60% at 1yr (ped) 40% at 1yr (all) | NS NS NS | NS | 50%† | NS |
| Parta et al 201720 | 17 (AYA) 20 (ped) | CGD | 24 y (18-32) 8 y (4-17) | MRD (6); MUD (30); MMUD (1) | Bu/Alem (6); Bu/TBI/Alem (31) | 82.5% (all) | 80% (all) | 17.5% (all) | 85% (all) | 3.4 y (range, NS) |
| Shah et al 201721 | 7 | DOCK8 Defic | 20 y (7-25) | HaploID (7) | Flu/Bu/Cy + low dose TBI (7) | 86% (all) | NS | 14% | 100% | 2 y (3 mo to 5.7 y) |
| Fu et al 201622 | 30 | 1° HLH; EBV- HLH; Tu-HLH; Undef- HLH | 23 y (14–52) 19 y (14–55) 24 y (14–44) 29 y (16–32) | HaploID (23); MRD (6); MUD (1). | Bu/Cyclo/VP16 (6) for MRD TBI/Cyclo/VP16 + ATG (24) for HaploID/MUD | 63.3% at 2 y (100% in 1° HLH; 64% in EBV-HLH; 17.7% in Tu-HLH; 25% in Undef) | NS | 100% | 26 mo | |
| Wehr et al 201511 | 14 (adult) 11 (ped) | CVID | 34 y (18-50) 14 y (8-17) | MRD (14); MUD (10); MMUD (1) | BCNU/Flu/Mel (5); Flu/Mel (7); Flu/Mel/Treo (2); Flu/Bu ±TT (4); Bu/Cy ±AraC (6); Flu/Cy (1). | 57% (adults) 52% (all patients) | 44% at 1 y (all) | 79% (adult) | NS | |
| Grossman et al 201423 | 14 | GATA2 Defic | 33 y (15-46) | MRD (4); MUD (4); UCB (4); HaploID (2). | Flu/low dose TBI (8); Flu/Cy/low dose TBI (6). | 57% (all) | NS | 28% | 100% | 3.5 y (1-5 y) |
| Gungor et al 201424 | 13 (adult) 43 (ped) | CGD CGD | 21 y (18-39) 9 y (0.8 – 17) | MRD (21); MUD (25); MMUD (10). | Flu/Bu ATG or Alem (all). | 92% (adult) 96% (all) | 91% (all) | 7% | 93% (adult) | 21 mo |
| Spinner et al 201425 | 21 | GATA2 Defic | NS (15-49 y) | NS | NS | 72% at 1 y; 65% at 2 y; 54% at 4 y | NS | NS | NS | 14 mo (0-180 mo) |
Adult ≥ 18 years at transplant. Ag, antigen; Alem, alemtuzumab; AraC, cytarabine; ATG, anti thymocyte globulin; BCNU, carmustine; Bu, busulfan; CGD, chronic granulomatous disease; CVID, common variable immunodeficiency; Cy, cyclophosphamide; def, deficiency; EBV, Epstein-Barr virus; EFS, event free survival; Flu, fludarabine; GOF, gain of function; HaploID, haploidentical; HLH, hemophagocytic lymphohistiocytosis; Mel, melphalan; MMUD, mismatched unrelated donor; MRD, matched related donor; MUD, matched unrelated donor; ns, not stated; OS, overall survival; PID, primary immunodeficiency; TBI, total body irradiation; Treo, treosulfan; TRM, transplant related mortality; TT, thiotepa; UCB, umbilical cord blood; VP16, etoposide.
Adapted from Morris and Albert41 with permission.
Age at diagnosis.
Secondary graft failure
As second or third procedure.