By Lynda Williams, medwireNews Reporter
medwireNews: Children and adolescents who undergo allogeneic haematopoietic stem cell transplantation (HCT) for nonmalignant disease require “multidisciplinary and lifelong follow-up”, researchers recommend in The Lancet Child & Adolescent Health.
Overall, 19% of 5790 patients who underwent HCT before the age of 21 years experienced one late effect of treatment, such as neurologic adverse events, kidney failure or metabolic disorders, and 7% had two or more such effects, report Rachel Phelan (Medical College of Wisconsin, Milwaukee, USA) and co-workers.
“Support for comprehensive survivorship care programmes for these patients with complex needs should be considered”, the authors advise.
“In addition, coordination of care when transitioning from paediatric-focused to adult-focused practices is crucial due to the ongoing possibility of late-occurring chronic conditions and risk for loss to follow-up as patients age.”
The team collated information from the Center for International Blood and Marrow Transplantation Research database for paediatric patients who received HCT at one of 171 centres around the world between 2000 and 2017. Almost a third (31.3%) of transplants occurred in 2000–2005, 30.0% in 2006–2011 and 38.7% in 2012–2017.
The majority (60.5%) of patients were boys, 36.4% were White, 13.3% Hispanic and 12.7% Black. The median age at HCT was 5.5 years. The most common reason for HCT was severe aplastic anaemia (21.2%), non-severe combined immunodeficiency (SCID) primary immune deficiency (14.6%), SCID (11.2%), Fanconi anaemia (9.8%) and sickle cell disease (9.2%).
The cumulative incidence of each late effect was calculated for 7 years after HCT. The most common events were stroke or seizure, affecting 9.4% of patients, followed by renal failure (8.1%), growth disturbance (5.0%), diabetes (4.9%), hypothyroidism (3.2%), gonadal dysfunction (2.6%), cataracts (1.9%), avascular necrosis (1.6%), congestive heart failure (0.6%) and myocardial infarction (0.2%).
However, the timing of each late effect differed, Phelan and co-workers say. The incidence of neurological events, renal failure and diabetes peaked within a year of HCT and the incidence of avascular necrosis, congestive heart failure and myocardial infection stabilized after 5 years. By contrast, the incidence of other endocrine-related disorders increased throughout follow-up.
Multivariable analysis revealed that the risk factors related to late effects differed between the sequalae and included sex, age over 10 years at time of HCT, indication for HCT, having an unrelated donor, receipt of total-body irradiation or myeloablative conditioning, and a subsequent diagnosis of acute or chronic graft-versus-host disease.
For example, girls were significantly more likely than boys to have gonadal dysfunction, prompting the team to remark that “focused work is needed to improve and expand options for pre-transplant fertility preservation”.
But the researchers also found that the risk of late effects appeared to decrease over the study period “possibly indicating improvements in overall transplant approaches and supportive care.”
Phelan and co-workers conclude: “The contemporary data presented in this report highlight the fact that refining the pre-transplantation and peri-transplantation procedures alone, while crucial, is insufficient to optimise long-term transplant-related outcomes.
“Attention must be paid to ensuring that all patients have access to personalised, long-term, multidisciplinary survivorship care.”
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Lancet Child Adolesc Health 2024; doi:10.1016/S2352-4642(24)00167-6