Table II.
Transplant related mortality and other significant toxicities.
| Age, years (gender) |
ALL risk group |
Stem cell source |
Description and timing of events | Contribution of sirolimus | Outcome |
|---|---|---|---|---|---|
| 9(M) | IRCR2 | 6/6 MSD | Pneumonia, cGVHD | Not related | Died day +789 |
| 11(M) | HRCR2 | 5/6 UCB | Started sirolimus day −3, sepsis, aspergillosis, severe VOD day +14, TAM, MSOF |
Possibly related | Died day +50 |
| 7(M) | CR4 | 3/6 UCB | Started sirolimus day −3, gram− sepsis – sirolimus stopped day +4, resp failure day +9, second gram− sepsis day +20, severe VOD day +25, MSOF, ARDS |
Possibly related | Died day +29 |
| 15(M) | CR1 | 4/6 UCB | Graft failure, second transplant CB day +59. Developed cGVHD |
Possibly related to graft failure, not related to cGVHD death |
Died day +365 of pneumonia |
| 12(F) | CR3 | 5/6 UCB | Late autoimmune haemolytic anaemia/cGVHD | Not related, not on sirolimus | Died day +365 |
| 18(M) | CR3 | 4/6 UCB | Respiratory failure day +22 with influenza, BK cystitis, adenovirus and rotavirus. Developed severe VOD, Ascites, MSOF, and pulmonary haemorrhage |
Possibly related | Died day +33 |
| 16(M) | CR3 | 5/6 UCB | Early reverible TAM without significant renal impairment, cGVHD |
Possibly related to TAM, resolved fully after stopping sirolimus. Not related to cGVHD/death |
Died day +431 |
| 1(F) | HRCR2 | 6/6 UCB | Pseudomonas sepsis, severe VOD, MSOF, pulmonary haemorrhage |
Possibly related | Died day +39 |
| 20(F) | HRCR2 | 10/10 URD | Moderate VOD (max bilirubin 42·75 µmol/l) | Possibly related, resolved completely, sirolimus not held |
Died of relapse day +239 |
| 9(M) | HRCR2 | 6/6 MSD | Moderate VOD (max bilirubin 44·46 µmol/l) | Possibly related, resolved completely after stopping sirolimus |
Died of relapse day +100 |
| 6(M) | IRCR2 | 6/6 MSD | Moderate VOD (max bili 25·65 µmol/l) | Possibly related, resolved completely after stopping sirolimus |
Alive and well, f/u day +732 |
| 18(F) | IRCR2 | 6/6 MSD | TAM, BK+ cystitis, renal failure (not requiring dialysis) |
Probably related, resolved completely after stopping sirolimus |
Alive and well, f/u day +1020 |
| 10(F) | CR3 | 5/6 UCB | Interstitial pneumonitis, BK cystitis | Possibly related, resolved completely, sirolimus not held |
Alive and well, f/u day +1315 |
| 10(M) | CR1 | 9/10 URD | CTC grade 4 pleural and grade 3 pericardial effusions, grade 3 muscle weakness and neuropathy |
Probably related (effusions), possibly related (weakness and neuropathy), rapid resolution of effusions after stopping sirolimus |
Alive and well, f/u day +998 |
| 4(M) | HRCR2 | 5/6 UCB | Late grade 4 thrombocytopenia, leucopenia and grade 3 anaemia |
Probably related, resolved completely after stopping sirolimus |
Alive and well, f/u day +851 |
IR, intermediate risk; HR, high risk; CR, complete remission; MSD, matched sibling donor; UCB, unrelated cord blood; URD, unrelated donor; cGVHD, chronic graft-versus-host disease; VOD, veno-occlusive disease; TAM, transplant-associated microangiopathy; MSOF, multi-system organ failure; ARDS, acute respiratory distress syndrome; CTC, common toxicity criteria; f/u, follow-up.