Ariagno, Sydney

Summary Recipients of haploidentical and unrelated haematopoietic cell transplants (HCTs) historically had a higher risk for graft‐versus‐host disease (GvHD). However, with improved GvHD prophylaxis, recent data suggest adult patients undergoing transplantation from unrelated or mismatched donors now experience the rates of GvHD similar to those receiving matched related donor grafts. This finding is not yet explored in paediatric patients. The objectives of this study were to: (1) compare acute and chronic GvHD incidence, mortality and GvHD‐free relapse‐free survival (GRFS) among matched related donor (MRD), matched unrelated donor (MUD) and haploidentical paediatric HCT recipients in the era of enhanced GvHD prophylaxis for high‐risk patients, (2) identify independent risk factors for GvHD and (3) evaluate the efficacy of various GvHD prophylactic regimens in our cohort. We conducted a retrospective, single‐centre medical record abstraction among patients aged 0–25 years who underwent allogeneic HCT for any indication. The results demonstrated that 5‐year cumulative incidence rates of any GvHD, all‐cause mortality and GRFS are similar across the haploidentical, MRD and MUD groups. Tacrolimus‐containing doublet/triplet regimens were associated with decreased GvHD, as compared to ciclosporin. Adding alemtuzumab also decreased risk for GvHD, without increasing relapse rates. Prospective studies with larger cohorts are warranted to further optimize outcomes for paediatric allogeneic HCT patients.

Central apnea is a major cause of death in neonates with trisomy 18 (T18) and is likely due to immaturity of the respiratory drive, similar to the pathological mechanism in apnea of prematurity. Although caffeine has long been used for apnea of prematurity, its use has not yet been reported for infants with T18. Here we describe an infant with T18 receiving comfort-focused care and palliative transport home. Of utmost importance to her family was enabling the patient to spend time at home before her death. However, immediately before transport, she developed severe central apnea, raising concern that she may not survive the transfer. Caffeine was trialed in the hopes of reducing central apnea events. Its use was successful and facilitated safe transport, allowing the patient to uneventfully transition home, where she spent several meaningful weeks with her family before her death.