High burden of late effects after haematopoietic stem cell transplantation in childhood: a single-centre study

Abstract

The aim of our study was to assess the cumulative incidence and severity ('burden') of late effects in a single-centre cohort of childhood haematopoietic stem cell transplantation (HSCT) survivors, at least 2 years after transplantation. The presence and severity of late effects in each survivor was documented according to the Common Terminology Criteria for Adverse Events (version 3.0). The burden of late effects was graded from mild to disabling/life-threatening. Risk factors for a high burden of late effects were assessed by univariate and multivariate logistic regression analyses. Among 162 survivors of HSCT seen in our late effects outpatient clinic, cumulative incidence of late effects was 93.2% after a median followup time of 7.2 years (range 2.0-21.0 years) after HSCT. The burden of late effects was mild, moderate, severe and disabling in 28, 41, 24 and 1% of survivors respectively. Risk factors for a severe or disabling burden of late effects were older age at HSCT (P for trend o0.001) and a conditioning regimen including irradiation OR 2.2, 95% CI 1.1-4.7, P ¼ 0.03). In conclusion, a high burden of late effects is found in childhood HSCT survivors after a median follow-up of only 7 years.

Health status of childhood leukemia survivors who received hematopoietic cell transplantation after BU or TBI: an LEA study

“…This determining role of preparative regimens is in accordance with previous studies that identified TBI as a risk factor for more frequent and more severe late effects in pediatric HSCT recipients. 2,16,17 This was not observed in a BMTSS study describing self-reported complications in mostly adult patients. 18 Child-specific adverse events like growth retardation might account for this difference.…”

Chronic disease burden and frailty in survivors of childhood HSCT: a report from the St. Jude Lifetime Cohort Study

“…[11][12][13][14] In the Bone Marrow Transplant Survivor Study (BMTSS), Armenian et al found a 79.0% prevalence of grade 1-4 and 25.5% of grade 3-4 self-reported chronic conditions among 145 childhood HSCT survivors (mean age, 24.0 years) surveyed 11 years post-HSCT. 11 A Dutch study that used clinical data to ascertain chronic conditions among 162 survivors of childhood HSCT (median age, 13.5 years) with 7 years of follow-up reported a lower prevalence of any grade or grade 3-4 chronic conditions (93.2% and 25%, respectively).…”

Adverse health events and late mortality after pediatric allogeneic hematopoietic SCT—two decades of longitudinal follow-up

“…11,12 Singlefraction TBI (sTBI) 10-12 Gy with CY (120 mg/kg) was used at the University Hospital in Huddinge during 1978-1995 and at the Helsinki University Hospital during [1978][1979][1980][1981][1982][1983]. Fractionated TBI (fTBI) was introduced in Helsinki in 1984 (10)(11)(12) Gy in 5-6 fractions) and in Huddinge in 1993 (12 Gy in 4 fractions). Patients with SAA have received CY (200 mg/kg) with or without TLI 6 Gy or fTBI 10 Gy.…”

“…Most studies have included patients with comparatively short follow-up times, often o 4 years, 6,9 or been limited by lack of follow-up data into late puberty or adulthood. 10 As long-term survival is an expected outcome for many pediatric patients undergoing HSCT, the treatment-related late toxicities are becoming increasingly important. In a retrospective cohort study, we aimed to evaluate the frequency, spectrum and risk factors of late effects in a cohort of pediatric allogeneic HSCT (allo-HSCT) survivors with longitudinal follow-up data into late puberty or adulthood.…”