HSCT characteristics
| ID . | Age of onset, y . | Clinical manifestations . | Age at HSCT, y . | HLA match . | Graft . | Conditioning . | Serotherapy . | GVHD prophylaxis . | Cell dose . | ANC engraftment, day . | GVHD . | Complications . | Last chimerism, % (time after transplant) . | Last FU . |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| CD27 | ||||||||||||||
| P6 | 1 | HLH | 2 | MMUD, 9 of 10 | CB | Bu, Cy | ATG, 3 × 30 mg/kg | CSA, Pred | 0.5 × 108/kg, TNC | +34 | Acute IV (gut 3-4) | Infections, ADV, EBV, HHV6, cord colitis syndrome, poor growth | 96 (4.5 y) | 9.5 y |
| P7 | 1 | Recurrent URTI, skin abscesses, meningitis | 2 | MMUD, 9 of 10 | CB | Flu, Bu (MAC) | ATG, 3 × 2.5 mg/kg | CSA, MMF | 0.7 × 108/kg, TNC | +20 | Acute I (skin 1-2), chronic limited skin | Severe infections, CMV | 100 (5 y) | 6.5 y |
| P8 | 15 | CAEBV, EBV-LPD, oral ulcers, uveitis | 17 | MMUD, 8 of 10 | CB | Flu, Cy, TBI | None | CSA, MMF | NA | +5 | Acute I (skin 1-2) | HHV6 infection | 100 (4 y) | 9 y |
| P13 | 6 | Recurrent pneumonia, EBV viremia, HLH | 12 | MRD, 10 of 10 | BM | Eto + Dexa (HLH protocol) | Campath, 4 × 0.3 mg/kg | CSA, Pred | 9.1 × 106/kg, CD34 | +13 | Acute III (liver 3) | Severe infections, mucor, HLH relapse, EBV | 100 (2 y) | 2 y |
| P18 | 2 | Pneumonia, EBV-HLH/LPD, DLCBL | 2 | Haplo, 5 of 10 | PB TCRab/ CD19 depletion | Flu, Mel, TT | ATG, 3 × 10 mg/kg | MMF | 20.6 × 106/kg, CD34 | 20 | None | ADV viremia, CMV reactivation, CMV VST D+60 | 100 (2 y) | 2 y |
| P25 | 1 | EBV-LPD, EBV viremia | 4 | MSD, 10 0f 10 | BM | Flu, Bu (MAC) | None | CSA, MTX | 8.6 × 106/kg, CD34 | 16 | None | Pulmonary candidosis (cleared) | 96 (3 mo) | 1 y |
| P26 | 14 | Recurrent tonsillitis, EBV− ns-HL, EBV+ NHL, DLBCL | 18 | MMUD, 9 of 10 | PB | Flu, Mel | Campath, 5 × 20 mg total | CSA | 6.4 × 106/kg, CD34 | 12 | Acute I (skin 2) chronic limited skin | EBV, rhinovirus | 100 (1 y) | 4 y |
| P27 | 5 | Recurrent pneumonia, skin infections, arthritis, LPD | 6 | MMUD, 9 of 10 | PB CD45RA depletion | Flu, Bu, TT | ATG, 3 × 10 mg/kg | CSA | 13.2 × 106/kg, CD34 | None | None | Graft failure | ||
| P27 2nd | 6 | Same | BM | Flu, Cy | ATG, 3 × 2.5 mg/kg | CSA, MMF | 3.3 × 106/kg, CD34 | 28 | None | 100 (3 mo) | 3 y | |||
| P30 | 3 | HL | 5 | MUD, 10 of 10 | PB | Flu, Bu (MAC) | ATG, 3 × 10 mg/kg | CSA, MTX | 8 × 106/kg, CD34 | 12 | None | None | 100 (1 mo) | 10 mo |
| P32 | 8 | mc-HL | 8 | MRD, 10 of 10 | BM | Flu, Bu | ATG, 3 × 10 mg/kg | CSA, MTX | 2.3 × 106/kg, CD34 | 15 | None | 100 (1 y) | 2 y | |
| P33 | 3 | ns-HL | 3 | MRD, 10 of 10 | BM | Flu, Bu, TT | ATG, 3 × 10 mg/kg | CSA, MTX | 2.8 × 106/kg, CD34 | 17 | None | Infections, hemorrhage | 100 (2 y) | 2 y |
| CD70 | ||||||||||||||
| P34 | 3 | ns-HL stage 2, LPD, recurrent fever | 10 | Haplo, 3 of 6 | BM | Flu, Bu (MAC) | ATG,1 × 10 mg/kg; Campath, 1 × 0.5 mg/kg; RTX, 375 | CSA, MMF, Post-Cy | 4.9 × 106/kg, CD34 | 22 | Acute II (skin 2, gut 1) chronic limited skin | None | 100 (1 y) | 3.5 y |
| P37 | 3 | Recurrent otitis, recurrent fever, chronic enteritis, mc-EBV-HL, HL relapse | 18 | MUD, 10 of 10 | PB | Flu, Treo, RTX | ATG | CSA, MMF | 3.4 × 106/kg, CD34 | 20 | None | RSV pneumonia hemopericardium, acute renal failure, thrombocytopenia | 75 (6 mo) | 1 y |
| P38 | 2 | URTI, IM, mc-EBV-HL, HL relapse | 10 | MUD, 10 of 10 | PB | Flu, Treo, RTX | ATG | CSA, MMF | 3.7 × 106/kg, CD34 | 20 | Acute II (gut 1) | Acute renal failure, RSV pneumonia | 100 (3 mo) | 1 y |
| P39 | 1 | LAP, recurrent fever, PFAPA like, EBV PTLD, mosquito bite hypersensitivity | 5 | MUD, 10 of 10 | BM | Flu, Treo, TT | ATG, 3 × 10 mg/kg | CSA, MMF, MTX | 24 | Acute II (gut 1) | CMV reactivation | 100 (1 y) | 4 y | |
| P40 | 7 | Tonsillitis, URTI, EBV viremia, EBV-LPD, recurrent fever | 16 | MSD, 10 of 10 | PB | Flu, Treo, RTX | No | CSA, MMF | 7 × 106/kg, CD34 | 20 | Acute III (gut 2-3), chronic gut | CMV infection, thrombocytopenia | 83 (4 mo) | 1 y |
| P41 | 9 | LAP, EBV–B-NHL | 11 | Haplo, 5 of 10 | BM | Flu, Bu, TT | ATG | CSA, post-Cy | 15.2 × 106/kg, CD34 | 13 | None | Severe engraftment syndrome, day +166 PJP | 100 (3 mo) | Died day +166 |
| P43 | 4 | Recurrent pneumonia, chronic enteritis, EBV LAP | 7 | MRD, 10 of 10 | PB | Flu, Treo | No | CSA, MMF | 5.4 × 106/kg, CD34 | 22 | Acute II (skin, liver) | None | 99 (4 mo) | 1 y |
| P44 | 3 | LRTI, ns-HL | 6 | MRD. 10 of 10 | PB | Flu, Treo | No | CSA, MMF | Chronic skin and liver | None | 99 (4 mo) | 1 y |
| ID . | Age of onset, y . | Clinical manifestations . | Age at HSCT, y . | HLA match . | Graft . | Conditioning . | Serotherapy . | GVHD prophylaxis . | Cell dose . | ANC engraftment, day . | GVHD . | Complications . | Last chimerism, % (time after transplant) . | Last FU . |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| CD27 | ||||||||||||||
| P6 | 1 | HLH | 2 | MMUD, 9 of 10 | CB | Bu, Cy | ATG, 3 × 30 mg/kg | CSA, Pred | 0.5 × 108/kg, TNC | +34 | Acute IV (gut 3-4) | Infections, ADV, EBV, HHV6, cord colitis syndrome, poor growth | 96 (4.5 y) | 9.5 y |
| P7 | 1 | Recurrent URTI, skin abscesses, meningitis | 2 | MMUD, 9 of 10 | CB | Flu, Bu (MAC) | ATG, 3 × 2.5 mg/kg | CSA, MMF | 0.7 × 108/kg, TNC | +20 | Acute I (skin 1-2), chronic limited skin | Severe infections, CMV | 100 (5 y) | 6.5 y |
| P8 | 15 | CAEBV, EBV-LPD, oral ulcers, uveitis | 17 | MMUD, 8 of 10 | CB | Flu, Cy, TBI | None | CSA, MMF | NA | +5 | Acute I (skin 1-2) | HHV6 infection | 100 (4 y) | 9 y |
| P13 | 6 | Recurrent pneumonia, EBV viremia, HLH | 12 | MRD, 10 of 10 | BM | Eto + Dexa (HLH protocol) | Campath, 4 × 0.3 mg/kg | CSA, Pred | 9.1 × 106/kg, CD34 | +13 | Acute III (liver 3) | Severe infections, mucor, HLH relapse, EBV | 100 (2 y) | 2 y |
| P18 | 2 | Pneumonia, EBV-HLH/LPD, DLCBL | 2 | Haplo, 5 of 10 | PB TCRab/ CD19 depletion | Flu, Mel, TT | ATG, 3 × 10 mg/kg | MMF | 20.6 × 106/kg, CD34 | 20 | None | ADV viremia, CMV reactivation, CMV VST D+60 | 100 (2 y) | 2 y |
| P25 | 1 | EBV-LPD, EBV viremia | 4 | MSD, 10 0f 10 | BM | Flu, Bu (MAC) | None | CSA, MTX | 8.6 × 106/kg, CD34 | 16 | None | Pulmonary candidosis (cleared) | 96 (3 mo) | 1 y |
| P26 | 14 | Recurrent tonsillitis, EBV− ns-HL, EBV+ NHL, DLBCL | 18 | MMUD, 9 of 10 | PB | Flu, Mel | Campath, 5 × 20 mg total | CSA | 6.4 × 106/kg, CD34 | 12 | Acute I (skin 2) chronic limited skin | EBV, rhinovirus | 100 (1 y) | 4 y |
| P27 | 5 | Recurrent pneumonia, skin infections, arthritis, LPD | 6 | MMUD, 9 of 10 | PB CD45RA depletion | Flu, Bu, TT | ATG, 3 × 10 mg/kg | CSA | 13.2 × 106/kg, CD34 | None | None | Graft failure | ||
| P27 2nd | 6 | Same | BM | Flu, Cy | ATG, 3 × 2.5 mg/kg | CSA, MMF | 3.3 × 106/kg, CD34 | 28 | None | 100 (3 mo) | 3 y | |||
| P30 | 3 | HL | 5 | MUD, 10 of 10 | PB | Flu, Bu (MAC) | ATG, 3 × 10 mg/kg | CSA, MTX | 8 × 106/kg, CD34 | 12 | None | None | 100 (1 mo) | 10 mo |
| P32 | 8 | mc-HL | 8 | MRD, 10 of 10 | BM | Flu, Bu | ATG, 3 × 10 mg/kg | CSA, MTX | 2.3 × 106/kg, CD34 | 15 | None | 100 (1 y) | 2 y | |
| P33 | 3 | ns-HL | 3 | MRD, 10 of 10 | BM | Flu, Bu, TT | ATG, 3 × 10 mg/kg | CSA, MTX | 2.8 × 106/kg, CD34 | 17 | None | Infections, hemorrhage | 100 (2 y) | 2 y |
| CD70 | ||||||||||||||
| P34 | 3 | ns-HL stage 2, LPD, recurrent fever | 10 | Haplo, 3 of 6 | BM | Flu, Bu (MAC) | ATG,1 × 10 mg/kg; Campath, 1 × 0.5 mg/kg; RTX, 375 | CSA, MMF, Post-Cy | 4.9 × 106/kg, CD34 | 22 | Acute II (skin 2, gut 1) chronic limited skin | None | 100 (1 y) | 3.5 y |
| P37 | 3 | Recurrent otitis, recurrent fever, chronic enteritis, mc-EBV-HL, HL relapse | 18 | MUD, 10 of 10 | PB | Flu, Treo, RTX | ATG | CSA, MMF | 3.4 × 106/kg, CD34 | 20 | None | RSV pneumonia hemopericardium, acute renal failure, thrombocytopenia | 75 (6 mo) | 1 y |
| P38 | 2 | URTI, IM, mc-EBV-HL, HL relapse | 10 | MUD, 10 of 10 | PB | Flu, Treo, RTX | ATG | CSA, MMF | 3.7 × 106/kg, CD34 | 20 | Acute II (gut 1) | Acute renal failure, RSV pneumonia | 100 (3 mo) | 1 y |
| P39 | 1 | LAP, recurrent fever, PFAPA like, EBV PTLD, mosquito bite hypersensitivity | 5 | MUD, 10 of 10 | BM | Flu, Treo, TT | ATG, 3 × 10 mg/kg | CSA, MMF, MTX | 24 | Acute II (gut 1) | CMV reactivation | 100 (1 y) | 4 y | |
| P40 | 7 | Tonsillitis, URTI, EBV viremia, EBV-LPD, recurrent fever | 16 | MSD, 10 of 10 | PB | Flu, Treo, RTX | No | CSA, MMF | 7 × 106/kg, CD34 | 20 | Acute III (gut 2-3), chronic gut | CMV infection, thrombocytopenia | 83 (4 mo) | 1 y |
| P41 | 9 | LAP, EBV–B-NHL | 11 | Haplo, 5 of 10 | BM | Flu, Bu, TT | ATG | CSA, post-Cy | 15.2 × 106/kg, CD34 | 13 | None | Severe engraftment syndrome, day +166 PJP | 100 (3 mo) | Died day +166 |
| P43 | 4 | Recurrent pneumonia, chronic enteritis, EBV LAP | 7 | MRD, 10 of 10 | PB | Flu, Treo | No | CSA, MMF | 5.4 × 106/kg, CD34 | 22 | Acute II (skin, liver) | None | 99 (4 mo) | 1 y |
| P44 | 3 | LRTI, ns-HL | 6 | MRD. 10 of 10 | PB | Flu, Treo | No | CSA, MMF | Chronic skin and liver | None | 99 (4 mo) | 1 y |
ADV, adenovirus; ANC, absolute neutrophil count; ATG, antithymocyte globulin; BM, bone marrow; Bu, busulfan; CB, cord blood; CSA, cyclosporin A; Cy, cyclophosphamide; Dexa, dexamethasone; Eto, etoposide; Flu, fludarabine; FU, follow-up; Haplo, haploidentical; HHV6, human herpesvirus 6; MAC, myeloablative conditioning; Mel, melphalan; MMF, mycophenolate mofetil; MMUD, mismatched unrelated donor; MRD, matched related donor; MSD, matched sibling donor; MUD, matched unrelated donor; PB, peripheral blood; PJP, Pneumocystis jirovecii pneumonia; Pred, prednisolone; PTLD, posttransplant lymphoproliferative disorder; TBI, total-body irradiation; TCRab, T-cell receptor αβ; TNC, total nucleated cell; Treo, treosulfan; TT, thiotepa; VST, virus-specific T cells. See Table 1 for expansion of other abbreviations.