HSCT characteristics and complications
| ID . | Age at HSCT (mo) . | Donor . | HLA . | Graft . | T-cell depletion∗ . | Conditioning† . | Serotherapy . | GVHD prophylaxis . | Acute GVHD . | Chronic GVHD . | Post-HSCT CT . | Outcome (mo/d after HSCT) . | Complications >HSCT . | AID >HSCT . |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1‡ | 35 | MMFD | 3/6 | BM | Yes | Bu 16 Cy 200 TT 20 (MAC) | None | None | 2 | NA | Died (d+49) | ADV | ||
| 2 | 26 | MFD | 6/6 | BM | No | Bu 20 Flu 200 (MAC) | None | CSA | No | No | Alive (mo+156) | |||
| 3 | 51 | MFD | 6/6 | BM | No | Bu 16 Flu 120 (MAC) | None | CSA | 3 | Yes | Alive (mo+125) | |||
| 4‡ | 35 | MUD | 10/10 | BM | No | Bu 19.2 Flu 160 Cy 120 (MAC) | None | CSA/MTX | 2 | Yes | Died (mo+33) | Sepsis/meningitis (chronic extensive GVHD) | ||
| 5 | 14 | MUD | 10/10 | BM | No | Bu 19.2 Flu 160 Cy 120 (MAC) | None | CSA/MTX | No | No | Alive (mo+84) | |||
| 6 | 10 | MMFD | 3/6 | PBSC | Yes | Bu 16 Flu 160 TT 10 (MAC) | None | None | No | No | Alive (mo+70) | |||
| 7 | 27 | MFD | 6/6 | BM | No | None | None | CSA | No | No | Alive (mo+221) | Disseminated BCG Psoas abscess | AIHA | |
| 8 | 16 | MFD | 6/6 | BM | No | Bu 20 Cy 200 (MAC) | None | CSA | No | No | Alive (mo+151) | |||
| 9 | 14 | MFD | 6/6 | BM | No | Bu 16 Flu 160 (MAC) | None | CSA/MTX | No | No | Alive (mo+114) | |||
| 10 | 25 | MFD | 6/6 | BM | No | Flu 150 Mel 140 (RIC) | ATG-G 20 | CSA | 1 | Yes | Alive (mo+161) | |||
| 11 | 6 | MFD | 6/6 | BM | No | Flu 150 Mel 140 (RIC) | ATG-G 20 | CSA | 3 | Yes | DLI (d+73) | Alive (mo+98) | RTI Klebsiella, pancytopenia | |
| 12 | 72 | MUD | 10/10 | PBSC | No | Bu 12.8 Cy 120 (MAC) | ATG-G 40 | CSA | No | No | Alive (mo+126) | |||
| 13 | 20 | MMFD | 4/6 | PBSC | Yes CD34+pos | Flu 150 Mel 140 (RIC) | ATG-T 20 | None | 1 | No | 2nd HSCT (mo+14) | Alive (mo+260) | AIHA§ ITP§ | |
| 14 | 47 | MUD | 10/10 | BM | No | Bu 16 Cy 200 (MAC) | Alemt. 1 | CSA/MTX | 1 | No | Alive (mo+192) | VZV (zoster) | ||
| 15 | 39 | MFD | 6/6 | PBSC | No | Flu 150 Mel 140 (RIC) | Alemt. 1 | CSA/MMF | 3 | No | Alive (mo+137) | Pneumatosis intestinalis | ||
| 16 | 2 | MUD | 10/10 | BM | No | Bu 16 Cy 200 (MAC) | Alemt. 0.6 | CSA/MTX | 2 | No | SCB (mo+9) | Alive (mo+240) | VOD, pneumonitis, nephritis | AIHA§ Alopecia totalis§ |
| 17 | 39 | MFD | 10/10 | PBSC | No | Flu 150 Mel 140 (RIC) | Alemt. 1 | CSA/ MMF | 3 | No | Alive (mo+65) | |||
| 18 | 25 | MFD | 6/6 | BM | No | Bu 16 Flu 160 (MAC) | None | CSA/MTX/ rituximab | No | No | Alive (mo+87) | VOD, pneumonitis | ||
| 19 | 22 | MUD | 10/10 | PBSC | No | Flu 150 Mel 140 (RIC) | Alemt. 1 | CSA | No | No | 2nd HSCT (mo+41) | Alive (mo+62) | ||
| 20 | 168 | MUD | 10/10 | PBSC | No | Flu 150 Mel 140 (RIC) | ATG-T 10 | CSA/ MMF | No | No | Alive (mo+28) | |||
| 21 | 28 | UCB | 5/6 | UCB | No | Treo 42 Flu 150 (RIC) | Alemt. 0.3 | CSA/ MMF | No | Yes | Alive (mo+20) | |||
| 22 | 25 | MFD | 10/10 | BM | No | Bu 16 Cy 200 (MAC) | None | CSA/ MTX | 2 | Yes | Alive (mo+125) | |||
| 23 | 42 | MFD | 6/6 | BM | No | Treo 42 Flu 160 TT 8 (MAC) | None | CSA/MTX | No | No | Alive (mo+22) | |||
| 24 | 12 | MUD | 10/10 | BM | No | Treo 42 Flu 160 (RIC) | ATG-G 45 | CSA/MTX | No | No | Alive (mo+94) | |||
| 25‡ | 31 | MUD | 10/10 | BM | No | Flu 150 Mel 140 (RIC) | Alemt. 0.6 | CSA/MMF | 4 | NA | SCB | Died (mo+8) | Acute relapsing GVHD (gut) | |
| 26 | 7 | MUD | 9/10 | PBSC | Yes CD34+pos | Treo 36 Flu 150 (RIC) | Alemt. 1 | CSA/MMF | 1 | No | Alive (mo+72) | |||
| 27 | 36 | MFD | 6/6 | UCB | No | Bu 16 Flu 150 (MAC) | None | CSA | No | No | Alive (mo+156) | VZV (zoster) | ||
| 28 | 40 | UCB | 6/6 | UCB | No | Bu 16 Cy 200 (MAC) | None | CSA/Pred | No | No | Alive (mo+96) | |||
| 29‡ | 22 | UCB | 10/10 | UCB | No | Bu 16 Flu 140 Mel 70 (MAC) | ATG-T 10 | CSA/Pred | No | NA | Died (d+20) | Neurologic decline | ||
| 30 | 20 | MUD | 10/10 | BM | No | Bu cAUC 80 Flu 150 (MAC) | ATG-T 10 | CSA/MTX | No | No | Alive (mo+108) | RTI (ADV, PIV3, metapneumovirus) | ||
| 31 | 18 | MUD | 10/10 | PBSC | No | Mel 140 Flu 150 (RIC) | Alemt. 1 | CSA/MMF | 1 | Yes | Alive (mo+24) | |||
| 32 | 192 | MMFD | 3/6 | PBSC | Yes TCRab/CD19 | Treo 42 Flu 150 TT 10 (MAC) | Alemt. 0.3 | None | 2 | No | Alive (mo+28) | GI (ADV, NV) RTI (Covid-19) | ||
| 33 | 43 | UCB | 5/6 | UCB | No | Bu 16 Cy 200 (MAC) | ATG-E 90 | CSA/pred | No | Yes | Alive (mo+156) | AIHA§ ITP§ DM Graves disease | ||
| 34 | 119 | UCB | 4/6 | UCB | No | Flu 125 Mel 140 (RIC) | Alemt. 2.3 | Tacro MTX | No | No | Alive (mo+96) | |||
| 35 | 92 | UCB | 6/6 | UCB | No | Flu 125 Mel 140 (RIC) | Alemt. 2.4 | Tacro/MTX | 2nd HSCT (mo+5) | Autologous recovery | VZV (zoster) RTI by EBV | |||
| 36‡ | 52 | UCB | 5/6 | UCB | No | Flu 150 Mel 140 TT 7 (MAC) | Alemt. 3.2 | Tacro/MTX | 3 | NA | Died (mo+8.5) | ADV, CMV (acute relapsing GVHD) | ||
| 37 | 2 | MMFD | 7/10 | BM | No | Bu cAUC 55 Cy 20 Flu 150 (RIC) | Alemt. 0.4 | PtCY d+3/+4 Tacro/MMF | No | No | 2nd HSCT (mo+17) | Alive (mo+80) | EBV (rituximab) RTI (Aspergillus) | AIHA |
| 38 | 4 | MUD | 9/10 | BM | No | Bu cAUC 79 Flu 160 (MAC) | Alemt. 0.6 | CSA/MMF | 3 | No | Alive (mo+53) | VOD | ||
| 39 | 41 | MUD | 10/10 | PBSC | No | Bu 12 Flu 250 (MAC) | Alemt. 0.6 | CSA/MTX | No | No | Alive (mo+30) | EBV+PTLD (rituximab) GI (sapovirus) Neisseria bacteremia | ||
| 40 | 26 | MUD | 9/10 | PBSC | No | Bu 12 Flu 250 (MAC) | Alemt. 0.6 | CSA/MTX | 2 | No | Alive (mo+22) | EBV (rituximab) RTI (rhinovirus/enterovirus/CV) GI (sapovirus) | ||
| 41 | 4 | MUD | 10/10 | PBSC | No | Bu12 Flu 250 (MAC) | Alemt. 0.6 | CSA/MTX | 2 | No | 2nd HSCT (mo+15) (+SCB) | Alive (mo+32) | EBV (rituximab) RTI (enterovirus/rhinovirus, ADV) | |
| 42 | 12 | MMFD | 7/10 | PBSC | No | None | None | PtCY d+3/+4 Tacro/MMF | No | No | SCB (mo+3) | Alive (mo+45) | ||
| 43 | 22 | MMFD | 6/10 | PBSC | Yes TCRab/CD19 | Treo 42 Flu 150 Thio 10 (MAC) | ATG-T 5 | Tocilizumab abatacept rituximab | No | No | CD45RA deplet. DLI (d+47, +81, +94) | Alive (mo+63) | RTI (HHV6, RSV) GI (RV, HHV6, ADV) Sepsis (Candida) Disseminated BCG | Arthritis |
| 44‡ | 37 | MUD | 10/10 | BM | No | Bu cAUC 75 Flu 160 (MAC) | ATG-T 7.5 | CSA/MMF | No | NA | Died (d+15) | MAS | ||
| 45 | 23 | MMFD | 5/10 | PBSC | Yes TCRab/CD19 | Treo 42 Flu 150 TT 10 (MAC) | ATG-G 30 | MMF, pred, ruxolitinib, vedolizumab | Yes, skin, 2 | No | Alive (mo+12) | EBV infection GI NV Zoster (VZV) RTI (rhinovirus/enterovirus, BV) | ||
| 46 | 3 | MUD | 10/10 | BM | No | Bu cAUC 79 Flu 180 (MAC) | Alemt. 0.6 | CSA/MMF | No | No | Alive (mo+85) |
| ID . | Age at HSCT (mo) . | Donor . | HLA . | Graft . | T-cell depletion∗ . | Conditioning† . | Serotherapy . | GVHD prophylaxis . | Acute GVHD . | Chronic GVHD . | Post-HSCT CT . | Outcome (mo/d after HSCT) . | Complications >HSCT . | AID >HSCT . |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1‡ | 35 | MMFD | 3/6 | BM | Yes | Bu 16 Cy 200 TT 20 (MAC) | None | None | 2 | NA | Died (d+49) | ADV | ||
| 2 | 26 | MFD | 6/6 | BM | No | Bu 20 Flu 200 (MAC) | None | CSA | No | No | Alive (mo+156) | |||
| 3 | 51 | MFD | 6/6 | BM | No | Bu 16 Flu 120 (MAC) | None | CSA | 3 | Yes | Alive (mo+125) | |||
| 4‡ | 35 | MUD | 10/10 | BM | No | Bu 19.2 Flu 160 Cy 120 (MAC) | None | CSA/MTX | 2 | Yes | Died (mo+33) | Sepsis/meningitis (chronic extensive GVHD) | ||
| 5 | 14 | MUD | 10/10 | BM | No | Bu 19.2 Flu 160 Cy 120 (MAC) | None | CSA/MTX | No | No | Alive (mo+84) | |||
| 6 | 10 | MMFD | 3/6 | PBSC | Yes | Bu 16 Flu 160 TT 10 (MAC) | None | None | No | No | Alive (mo+70) | |||
| 7 | 27 | MFD | 6/6 | BM | No | None | None | CSA | No | No | Alive (mo+221) | Disseminated BCG Psoas abscess | AIHA | |
| 8 | 16 | MFD | 6/6 | BM | No | Bu 20 Cy 200 (MAC) | None | CSA | No | No | Alive (mo+151) | |||
| 9 | 14 | MFD | 6/6 | BM | No | Bu 16 Flu 160 (MAC) | None | CSA/MTX | No | No | Alive (mo+114) | |||
| 10 | 25 | MFD | 6/6 | BM | No | Flu 150 Mel 140 (RIC) | ATG-G 20 | CSA | 1 | Yes | Alive (mo+161) | |||
| 11 | 6 | MFD | 6/6 | BM | No | Flu 150 Mel 140 (RIC) | ATG-G 20 | CSA | 3 | Yes | DLI (d+73) | Alive (mo+98) | RTI Klebsiella, pancytopenia | |
| 12 | 72 | MUD | 10/10 | PBSC | No | Bu 12.8 Cy 120 (MAC) | ATG-G 40 | CSA | No | No | Alive (mo+126) | |||
| 13 | 20 | MMFD | 4/6 | PBSC | Yes CD34+pos | Flu 150 Mel 140 (RIC) | ATG-T 20 | None | 1 | No | 2nd HSCT (mo+14) | Alive (mo+260) | AIHA§ ITP§ | |
| 14 | 47 | MUD | 10/10 | BM | No | Bu 16 Cy 200 (MAC) | Alemt. 1 | CSA/MTX | 1 | No | Alive (mo+192) | VZV (zoster) | ||
| 15 | 39 | MFD | 6/6 | PBSC | No | Flu 150 Mel 140 (RIC) | Alemt. 1 | CSA/MMF | 3 | No | Alive (mo+137) | Pneumatosis intestinalis | ||
| 16 | 2 | MUD | 10/10 | BM | No | Bu 16 Cy 200 (MAC) | Alemt. 0.6 | CSA/MTX | 2 | No | SCB (mo+9) | Alive (mo+240) | VOD, pneumonitis, nephritis | AIHA§ Alopecia totalis§ |
| 17 | 39 | MFD | 10/10 | PBSC | No | Flu 150 Mel 140 (RIC) | Alemt. 1 | CSA/ MMF | 3 | No | Alive (mo+65) | |||
| 18 | 25 | MFD | 6/6 | BM | No | Bu 16 Flu 160 (MAC) | None | CSA/MTX/ rituximab | No | No | Alive (mo+87) | VOD, pneumonitis | ||
| 19 | 22 | MUD | 10/10 | PBSC | No | Flu 150 Mel 140 (RIC) | Alemt. 1 | CSA | No | No | 2nd HSCT (mo+41) | Alive (mo+62) | ||
| 20 | 168 | MUD | 10/10 | PBSC | No | Flu 150 Mel 140 (RIC) | ATG-T 10 | CSA/ MMF | No | No | Alive (mo+28) | |||
| 21 | 28 | UCB | 5/6 | UCB | No | Treo 42 Flu 150 (RIC) | Alemt. 0.3 | CSA/ MMF | No | Yes | Alive (mo+20) | |||
| 22 | 25 | MFD | 10/10 | BM | No | Bu 16 Cy 200 (MAC) | None | CSA/ MTX | 2 | Yes | Alive (mo+125) | |||
| 23 | 42 | MFD | 6/6 | BM | No | Treo 42 Flu 160 TT 8 (MAC) | None | CSA/MTX | No | No | Alive (mo+22) | |||
| 24 | 12 | MUD | 10/10 | BM | No | Treo 42 Flu 160 (RIC) | ATG-G 45 | CSA/MTX | No | No | Alive (mo+94) | |||
| 25‡ | 31 | MUD | 10/10 | BM | No | Flu 150 Mel 140 (RIC) | Alemt. 0.6 | CSA/MMF | 4 | NA | SCB | Died (mo+8) | Acute relapsing GVHD (gut) | |
| 26 | 7 | MUD | 9/10 | PBSC | Yes CD34+pos | Treo 36 Flu 150 (RIC) | Alemt. 1 | CSA/MMF | 1 | No | Alive (mo+72) | |||
| 27 | 36 | MFD | 6/6 | UCB | No | Bu 16 Flu 150 (MAC) | None | CSA | No | No | Alive (mo+156) | VZV (zoster) | ||
| 28 | 40 | UCB | 6/6 | UCB | No | Bu 16 Cy 200 (MAC) | None | CSA/Pred | No | No | Alive (mo+96) | |||
| 29‡ | 22 | UCB | 10/10 | UCB | No | Bu 16 Flu 140 Mel 70 (MAC) | ATG-T 10 | CSA/Pred | No | NA | Died (d+20) | Neurologic decline | ||
| 30 | 20 | MUD | 10/10 | BM | No | Bu cAUC 80 Flu 150 (MAC) | ATG-T 10 | CSA/MTX | No | No | Alive (mo+108) | RTI (ADV, PIV3, metapneumovirus) | ||
| 31 | 18 | MUD | 10/10 | PBSC | No | Mel 140 Flu 150 (RIC) | Alemt. 1 | CSA/MMF | 1 | Yes | Alive (mo+24) | |||
| 32 | 192 | MMFD | 3/6 | PBSC | Yes TCRab/CD19 | Treo 42 Flu 150 TT 10 (MAC) | Alemt. 0.3 | None | 2 | No | Alive (mo+28) | GI (ADV, NV) RTI (Covid-19) | ||
| 33 | 43 | UCB | 5/6 | UCB | No | Bu 16 Cy 200 (MAC) | ATG-E 90 | CSA/pred | No | Yes | Alive (mo+156) | AIHA§ ITP§ DM Graves disease | ||
| 34 | 119 | UCB | 4/6 | UCB | No | Flu 125 Mel 140 (RIC) | Alemt. 2.3 | Tacro MTX | No | No | Alive (mo+96) | |||
| 35 | 92 | UCB | 6/6 | UCB | No | Flu 125 Mel 140 (RIC) | Alemt. 2.4 | Tacro/MTX | 2nd HSCT (mo+5) | Autologous recovery | VZV (zoster) RTI by EBV | |||
| 36‡ | 52 | UCB | 5/6 | UCB | No | Flu 150 Mel 140 TT 7 (MAC) | Alemt. 3.2 | Tacro/MTX | 3 | NA | Died (mo+8.5) | ADV, CMV (acute relapsing GVHD) | ||
| 37 | 2 | MMFD | 7/10 | BM | No | Bu cAUC 55 Cy 20 Flu 150 (RIC) | Alemt. 0.4 | PtCY d+3/+4 Tacro/MMF | No | No | 2nd HSCT (mo+17) | Alive (mo+80) | EBV (rituximab) RTI (Aspergillus) | AIHA |
| 38 | 4 | MUD | 9/10 | BM | No | Bu cAUC 79 Flu 160 (MAC) | Alemt. 0.6 | CSA/MMF | 3 | No | Alive (mo+53) | VOD | ||
| 39 | 41 | MUD | 10/10 | PBSC | No | Bu 12 Flu 250 (MAC) | Alemt. 0.6 | CSA/MTX | No | No | Alive (mo+30) | EBV+PTLD (rituximab) GI (sapovirus) Neisseria bacteremia | ||
| 40 | 26 | MUD | 9/10 | PBSC | No | Bu 12 Flu 250 (MAC) | Alemt. 0.6 | CSA/MTX | 2 | No | Alive (mo+22) | EBV (rituximab) RTI (rhinovirus/enterovirus/CV) GI (sapovirus) | ||
| 41 | 4 | MUD | 10/10 | PBSC | No | Bu12 Flu 250 (MAC) | Alemt. 0.6 | CSA/MTX | 2 | No | 2nd HSCT (mo+15) (+SCB) | Alive (mo+32) | EBV (rituximab) RTI (enterovirus/rhinovirus, ADV) | |
| 42 | 12 | MMFD | 7/10 | PBSC | No | None | None | PtCY d+3/+4 Tacro/MMF | No | No | SCB (mo+3) | Alive (mo+45) | ||
| 43 | 22 | MMFD | 6/10 | PBSC | Yes TCRab/CD19 | Treo 42 Flu 150 Thio 10 (MAC) | ATG-T 5 | Tocilizumab abatacept rituximab | No | No | CD45RA deplet. DLI (d+47, +81, +94) | Alive (mo+63) | RTI (HHV6, RSV) GI (RV, HHV6, ADV) Sepsis (Candida) Disseminated BCG | Arthritis |
| 44‡ | 37 | MUD | 10/10 | BM | No | Bu cAUC 75 Flu 160 (MAC) | ATG-T 7.5 | CSA/MMF | No | NA | Died (d+15) | MAS | ||
| 45 | 23 | MMFD | 5/10 | PBSC | Yes TCRab/CD19 | Treo 42 Flu 150 TT 10 (MAC) | ATG-G 30 | MMF, pred, ruxolitinib, vedolizumab | Yes, skin, 2 | No | Alive (mo+12) | EBV infection GI NV Zoster (VZV) RTI (rhinovirus/enterovirus, BV) | ||
| 46 | 3 | MUD | 10/10 | BM | No | Bu cAUC 79 Flu 180 (MAC) | Alemt. 0.6 | CSA/MMF | No | No | Alive (mo+85) |
ADV, adenovirus; Alemt., alemtuzumab (mg/kg body weight); ATG-E, horse anti–thymocyte globulin (mg/kg body weight); ATG-G, rabbit anti–T-lymphocyte globulin (mg/kg body weight); ATG-T, rabbit antithymocyte globulin (mg/kg body weight); Bu, busulfan (mg/kg body weight); BV, bocavirus; cAUC, cumulative area under the curve (mg/L × h); CSA, cyclosporine A; CV, coronavirus; Cy, cyclophosphamide; DM, diabetes mellitus; Flu, fludarabine (mg/m2 body surface area); GI, gastrointestinal infection; Mel, melphalan; MFD, matched family donor; MMF, mycophenolate mofetil; MMFD, mismatched family donor; MTX, methotrexate; NA, not applicable; NV, norovirus; PBSC, peripheral blood stem cells; PIV3, parainfluenza virus type 3; Pred, prednisolone; PtCY, post-HSCT cyclophosphamide; PTLD, posttransplant lymphoproliferative disease; RTI, respiratory tract infection; Tacro, tacrolimus; TCRab, T-cell receptor αβ; Treo, treosulfan (g/m2 body surface area); TT, thiotepa (mg/kg body weight); VOD, veno-occlusive disease; UCB, umbilical cord blood.
In vitro depletion.
Definitions of conditioning (supplemental Material).
Deceased patient.
Resolved after rituximab treatment.