Long-term health-related outcomes in survivors of childhood cancer treated with HSCT versus conventional therapy: a report from the Bone Marrow Transplant Survivor Study (BMTSS) and Childhood Cancer Survivor Study (CCSS)

Treatment-related late toxicities after pediatric allogeneic hematopoietic SCT (allo-HSCT) are increasingly important as long-term survival has become an expected outcome for many transplanted children and adolescents. In a retrospective cohort study, we assessed long-term health outcomes in 204 allo-HSCT survivors transplanted in childhood or adolescence (o 20 years) between 1978 through 2000 after a median follow-up time of 12 (range 4-28) years. Data on conditioning regimen, adverse health events (AE) and growth and hormonal substitutions (hormone replacement therapies (HRTs)) were obtained from medical records. AEs were graded retrospectively according to Common Terminology Criteria for Adverse Events v3.0. Late deaths (⩾48 months after allo-HSCT) were evaluated separately. Multivariate analysis demonstrated that chronic GVHD (P o0.000) and longer follow-up time (P o0.05) correlated with AEs, whereas CY-based conditioning was inversely correlated (P o 0.002). TBI and longer follow-up duration predicted more severe AEs (P o 0.001 and P o 0.001, respectively). HRTs were more frequent after TBI. Diabetes type II, dyslipidemia and hypertension were detected in 9, 7 and 7% of the survivors, respectively. Late deaths (n = 22) were most frequently due to pulmonary failure (n = 7), followed by secondary malignancy (n = 5). The occurrence of AEs after pediatric allo-HSCT is high and likely to increase during extended follow-up, particularly in patients who have received TBI.

Section: Discussionsupporting

confidence: 82%

Section: Discussionmentioning

confidence: 99%

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

Wilhelmsson

Vatanen

Borgström

et al. 2015

“…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.…”

Section: Discussionmentioning

confidence: 75%

“…1 It is now well known that these patients require attentive and continued follow-up into adulthood, due to late and very late complications such as endocrinopathies, growth and development disturbances, and second malignancies. The particularly high prevalence of late effects among HSCT recipients 2 should be a major concern of pediatric hematologists. This increased morbidity has often been attributed to the late toxicity of preparative regimens.…”

Section: Introductionmentioning

confidence: 99%

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

Bernard

Auquier

Herrmann

et al. 2014

The purpose of this multicenter study was to compare the long-term impact of a preparative regimen with either BUBU or TBI on health status and quality of life (QoL) in childhood acute leukemia survivors treated with hematopoietic SCT (HSCT). Two-hundred and forty patients were included. Sixty-six had received BU, while 174 had received TBI. Median follow-up from HSCT was 10.1 years. Multivariate analyses were performed to assess the occurrence of late effects according to treatment. QoL was assessed in 130 adults using SF-36 questionnaires. Patients developed fewer late complications after BU (2.35 vs 3.01, P ¼ 0.03) while the risk to present with at least one complication was equivalent in both groups (87.9% after BU and 93.1% after TBI, P ¼ 0.66). Detailed multivariate analyses revealed a lower risk of height growth failure (OR ¼ 0.2), cataract (OR ¼ 0.1) and iron overload (OR ¼ 0.2) after BU, and an increased risk of overweight (OR ¼ 3.9) and alopecia (OR ¼ 11.2). SF-36 mental and physical composite scores were similar in both treatment groups and proved significantly lower than French norms. Late effects induced by BU might differ from those experienced after TBI. Although less frequent, they are still of considerable importance and may affect patients' QoL.

“…4 However, there is a paucity of data regarding pediatric and adolescent and young adult survivors of childhood BMT and their transition to adulthood. It is critical that attention is given to this subset of BMT survivors, for they bring with them a unique set of potential complications.…”

Section: Introductionmentioning

confidence: 99%

Childhood to adult transition and long-term follow-up after blood and marrow transplantation

Cupit

Duncan

Savani

et al. 2015

The use of hematopoietic stem cell transplantation or blood and marrow transplantation (BMT) is on the increase worldwide. With BMT's increasing utilization and increasing success, the number of BMT survivors in the United States alone is expected to surpass 500 000 by the year 2030. BMT survivors are susceptible to a host of long-term side effects and complications. The pediatric and adolescent and young adult (AYA) populations comprise an increasing proportion of BMT survivors. Though these populations are both at risk of a specific set of sequelae and faced with the additional challenge of transitioning to adult care, no previous literature has addressed their specific challenges. In this review, we illustrate with clinical vignettes the need for focused and specific survivorship clinics for pediatric/AYA BMT survivors. We then focus on the following areas pertaining to pediatric BMT survivorship and care: (1) psychological health, (2) neurocognition, (3) endocrine health, (4) infertility resources, (5) issues in transition from pediatric to adult clinicians, (6) preventative services and (7) cost of care issues.