Table 3.
HCT data and post-HCT complications for the 30 DADA2 patients
| Patient ID | Year of HCT | Age at HCT (y)/sex | Indication for HCT | HLA match/graft source | Conditioning | GvHD prophylaxis | CD34+ dose (× 106/kg) | aGvHD/grade | cGvHD | Other comp | Last donor chimerism | Last follow-up (m) |
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| P1* | 2009 | 3/M | RCA, neutropenia | MSD BM | Bu/Cy (MAC) | MMF/CsA | 7.5 |
Gut 3 |
No | ITP, SOS on D + 60 | 3 y > 95% | 114 |
| P2 | 2018 | 28/F | PRCA | MSD BM | Bu/Flu (MAC) | CsA | 4.3 | No | No | None | 2 y 91% (myeloid 100%) | 26 |
| P3 | 2016 | 3/F | PRCA | MRD BM (ADA2 carrier) | Bu/Flu/TT (MAC) | MTX/CsA | 5.4 | No | No | None | 2 y 100% | 48 |
| P4 | 2016 | 23/M | PRCA | MRD PB (unknown ADA2 status) | Bu/Flu (MAC) | MMF/PTCy | 10.5 | Skin 1 | Skin + mouth, mild | None | 6 m 100% | 41 |
| P5 | 2015 | 7/M | PRCA | 10/10 MUD BM | Bu/Cy/ATG (MAC) | MTX/CsA | TNC = 7.5 | No | No | None | 1 y 100% | 60 |
| P6 | 2019 | 17/M | Neutropenia | 10/10 MUD PB | Bu/Cy/pentostatin/hATG (RIC) | MMF/Tacro/PTCy | 7.7 | Skin 1 | No | None | 1 y 100% | 20 |
| P7 | 2016 | 23/M | Severe lymphopenia | 10/10 MUD BM | Bu/Flu/Alem (MAC) | MTX/CsA | 6.4 | No | No | None | 6 m > 95% | 50 |
| P8 | 2016 | 2/M | PRCA, recurrent CMV | 10/10 MUD BM | Flu/Treo/TT/ATG (MAC) | MTX/CsA | 8.3 | Skin 2 | No | None | 6 m 100% | 50 |
| P9 | 2016 | 5/F | RCA, neutropenia | 10/10 MUD BM | Flu/Treo/TT/Alem (MAC) | MMF/CsA | 9 | Skin 1 | No | None | 1 y 98% | 48 |
| P10 | 2016 | 11/F | Pancytopenia, autoimmunity | 10/10 MUD PB | Flu/Treo/TT/ATG (MAC) | MTX/CsA | 3.2 | Skin 1 | No | Steatosis hepatis | 1 y 100% | 50 |
| P11 | 2019 | 28/F | Pancytopenia | 10/10 MUD BM | Flu/Treo/TT/Alem (MAC) | MMF/CsA | 4.8 | No | No | None | 6 m 100% | 12 |
| P12* | 2016 | 5/M | Recurrent TIA, immunodeficiency | 10/10 MUD PB (2 boosts for declining MC)** | Flu/Treo/Alem (MAC) | MMF/CsA | 8.1 | Skin 1 | No | None | 3 y > 95% | 54 |
| P13 | 2020 | 6/M | PRCA | 10/10 MUD BM | Flu/Treo/Alem (MAC) | MMF/Tacro | 5.7 | No | No | None | 6 m 100% | 6 |
| P14 | 2018 | 14/F | Neutropenia, severe lymphopenia | 10/10 MUD PB | Flu/Treo/Alem (MAC) | MMF/CsA | 15 | Skin 1 | No | Mild bronchiectasis | 2 y 100% | 22 |
| P15 | 2016 | 4/M | PRCA, neutropenia | 10/10 MUD BM | Flu/Mel/Alem (RIC) | MTX/Tacro | 3.2 | No | No | ITP D + 42 | 1 y 98% | 52 |
| P16 | 2003 | 4/M | Refractory SAA | 10/10 MUD BM (1st HCT from affected MSD) | Flu/TBI/Alem (RIC) | MTX/CsA | 1.4 | No | No | None | 3 y 100% | 200 |
| P17 | 2019 | 6/F | Immune dysregulation | 10/10 MUD BM | Flu/Treo/TT/Alem (MAC) | CsA | 10.8 | Skin 1 | Skin, moderate | None | 3 m 96% | 11 |
| P18.1 | 2018 | 7/M | RCA, neutropenia | 10/10 MUD PB | Bu/Cy/pentostatin/hATG (RIC) | MMF/Tacro/PTCy | 9 | No | No | Secondary GF | ||
| P18.2 | 2019 | 10/10 MUD PB | Flu/Cy/Alem (NMA) | CsA/PTCy | 3.7 | No | No | Unstable graft | ||||
| P18.3 | 2019 | 10/10 MUD PB CD34 selected + 2 week post-HCT DLI | Flu/rATG (NMA) | None | 7.4 | Skin, liver, gut 2 | No | NRH, siderosis | 8 m 100% | 24 | ||
| P19.1 | 2017 | 19/M | Neutropenia | 10/10 MUD BM | Bu/Cy/pentostatin (RIC) | MMF/Siro/PTCy | 4.5 | No | No | SOS on D + 21, secondary GF | ||
| P19.2 | 2018 | 10/10 MUD PB | Flu/Alem (NMA) | CsA | 8.5 | No | No | Stable NRH | 1 y 100% | 36 | ||
| P20 | 2014 | 23/F | Neutropenia | 10/10 MUD BM | Flu/Mel/Alem (RIC) | Prednisone/CsA | TNC = 2.1 | No | No | None | 2 y 98% | 76 |
| P21 | 2018 | 13/F | Diffuse large B-cell lymphoma | Haplo brother PB | Flu/Mel/TT/ATG/rituximab (RIC) | alpha–beta TCD | 7 | No | No | None | 1 y 100% | 30 |
| P22 | 2013 | 20/F | Neutropenia | Haplo sister BM (ADA2 carrier) | Flu/Bu/Cy/TBI200 (MAC) | Tacro/MMF/PTCy | 0.5 | No | No | None | 3 y 100% | 78 |
| P23 | 2015 | 5/F | Neutropenia | 9/10 MMUD BM | Bu/Flu/TT/ATG/ rituximab (MAC) | MTX/CsA | 4.2 | Skin/gut 2 | Gut, mild | None | 1 y 100% | 45 |
| P24 | 2018 | 9/F | Neutropenia, AIHA | 9/10 MMUD BM | Bu/Flu/Alem/Rituximab (MAC) | MMF/CsA/PTCy | 5.6 | No | No | SOS on D + 12, ARDS D + 54 | 1 m 100% | 2 (dead) |
| P25 | 2020 | 4/F | PRCA | 9/10 MMUD PB | Flu/Treo/TT/ATG (MAC) | MTX/CsA | 4.5 | Skin 2 | No | None | 1 m 100% to 11 m 55% | 12 |
| P26 | 2018 | 24/F | Neutropenia | 9/10 MMUD BM | Flu/Treo/TT/ATG/ rituximab (MAC) | MTX/CsA | 3 | No | No | None | 2 y 100% | 51 |
| P27 | 2012 | 8/F | Pancytopenia | 9/10 MMUD BM | Flu/Treo/Alem (MAC) | MTX/CsA | 8.6 | No | No | None | 5 y 100% | 98 |
| P28.1 | 2016 | 16/F | RCA, neutropenia | 9/10 MMUD BM | Flu/Treo/TT/ATG (MAC) | MTX/CsA | 1.4 | No | No | Primary GF | ||
| P28.2 | 9/10 MMUD PB (different donor) | Flu/TT/ATG (RIC) (alpha–beta TCD) | MTX/CsA | 4.9 | No | No | None | 1 m 100% | 42 | |||
| P29* | 2007 | 9/M | PRCA | 9/10 MMUD PB (1st HCT from affected MSD) | Flu/TBI450/Alem (RIC) | MTX/CsA | 7.9 | Skin 2 | Skin + liver moderate | AIHA D + 70 bridging liver fibrosis | 3 y 95% | 155 |
| P30* | 2017 | 16/F | RCA, neutropenia | 9/10 MMUD BM | Flu/Mel/TT/Alem (RIC) | prednisone/CsA | TNC = 2.5 | No | Skin mild | Hepatitis | 1 y 100% | 36 |
*Siblings (1 + 12 and 29 + 30); **unconditioned boosts 1 month apart; AIHA autoimmune hemolytic anemia, Alem alemtuzumab, ARDS acute respiratory distress syndrome, hATG horse antithymocyte globulin, BM bone marrow, Bu busulfan, comp complications, CsA cyclosporine A, DLI donor lymphocyte infusion, Flu fludarabine, GF graft failure, GvHD graft versus host disease, HCT hematopoietic cell transplant, m month, MC mixed chimerism, Mel melphalan, MTX methotrexate, MMF mycophenolate mofetil, MSD HLA-matched sibling donor, MUD HLA-matched unrelated donor, MMUD HLA-mismatched unrelated donor, PB peripheral blood, NRH nodular regenerative hyperplasia, PRCA pure red cell aplasia, RCA red cell aplasia, Siro sirolimus, SOS sinusoidal obstruction syndrome, TBI total body irradiation, TNC total nucleated cell dose, Treo treosulfan, TT thiotepa, PTCy post-transplant cyclophosphamide, y year