Burden of Morbidity after Allogeneic Blood or Marrow Transplantation for Inborn Errors of Metabolism: A BMT Survivor Study Report

Wadhwa, Aman; Chen, Yanjun; Hageman, Lindsey; Schlichting, Elizabeth; Funk, Erin; Hicks, Jessica; Balas, Nora; Siler, Arianna; Wu, Jessica; Francisco, Liton; Holmqvist, Anna Sällfors; Gupta, Ashish; Lund, Troy C.; Orchard, Paul J.; Armenian, Saro H.; Arora, Mukta; Bhatia, Smita

doi:10.1016/j.jtct.2021.11.023

Cited by 2 publications

(1 citation statement)

References 20 publications

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“…Our study found that younger patients (and patients with lower weights) were more likely to need ICU care, which is not surprising given patients with smaller airways have a higher risk of increased respiratory needs and intubation post-HCT ( 17 ). Our cohort had a higher proportion of patients needing PICU care compared to previously reported retrospective cohorts (38.5% vs. 10%–15%; 3 – 5 , 13 , 14 ) and is likely the result of our patient cohort being younger, lower weight and having a high proportion of patients with underlying inborn errors of metabolism, who are also have a higher risk of complications peri-transplant ( 17 – 19 ). Further, when evaluating PICU care risk by multivariate logistic regression and adjusting for underlying disease, increasing age was found to have a lower odds of needing PICU care, which is in keeping with lower age (and likely smaller weight patients) experiencing higher risk of PICU need peri-HCT ( 17 ).…”

Section: Discussionmentioning

confidence: 71%

Risk factors predicting need for the pediatric intensive care unit (PICU) post-hematopoietic cell transplant, PICU utilization, and outcomes following HCT: a single center retrospective analysis

Johnson,

Cornea,

Goldfarb

et al. 2024

Front. Pediatr.

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Hematopoietic cell transplant (HCT) is a curative treatment for multiple malignant and non-malignant disorders. While morbidity and mortality have decreased significantly over the years, some patients still require management in the pediatric intensive care unit (PICU) during their HCT course for additional respiratory, cardiovascular, and/or renal support. We retrospectively reviewed pediatric patients (0–18 years) who underwent HCT from January 2015–December 2020 at our institution to determine risk factors for PICU care and evaluate PICU utilization and outcomes. We also assessed pulmonary function testing (PFT) data to determine if differences were noted between PICU and non-PICU patients as well as potential evolution of pulmonary dysfunction over time. Risk factors of needing PICU care were lower age, lower weight, having an underlying inborn error of metabolism, and receiving busulfan-based conditioning. Nearly half of PICU encounters involved use of each of respiratory support types including high-flow nasal cannula, non-invasive positive pressure ventilation, and mechanical ventilation. Approximately one-fifth of PICU encounters involved renal replacement therapy. Pulmonary function test results largely did not differ between PICU and non-PICU patients at any timepoint aside from individuals who required PICU care having lower DLCO scores at one-year post-HCT. Future directions include consideration of combining our data with other centers for a multi-center retrospective analysis with the goal of gathering and reporting additional multi-center data to work toward continuing to decrease morbidity and mortality for patients undergoing HCT.

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Section: Discussionmentioning

confidence: 71%

Risk factors predicting need for the pediatric intensive care unit (PICU) post-hematopoietic cell transplant, PICU utilization, and outcomes following HCT: a single center retrospective analysis

Johnson,

Cornea,

Goldfarb

et al. 2024

Front. Pediatr.

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The Late Effects of Hematopoietic Stem Cell Transplants in Pediatric Patients: A 25-Year Review

Lee,

Nguyen,

et al. 2024

The Journal of Clinical Endocrinology &Amp; Metabolism

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Context A rare, large single centre study covering all long-term health outcomes of paediatric allogeneic HSCT survivors, to provide comprehensive local data, and identify gaps and future directions for improved care. Objective To document endocrine sequelae and other late effects of all HSCT recipients Design Retrospective review Setting Royal Children’s Hospital Melbourne Patients 384 children and adolescents received HSCT. 228 formed the study cohort; 212 were alive at commencement of data accrual. Intervention None Main outcome measures Incidence of endocrinopathies; fertility, growth, bone and metabolic status; subsequent malignant neoplasms (SMNs). Results Gonadotoxicity was more common in females (p<0.001). Total body irradiation (TBI) conditioning was more toxic than chemotherapy alone. All females receiving TBI or higher cyclophosphamide equivalent doses (CED) developed premature ovarian insufficiency (POI) . In males, impaired spermatogenesis +/- testicular endocrine dysfunction was associated with increasing testicular radiation exposure. Preservation of gonadal function was associated with younger age at HSCT. Of sexually active females, 22% reported spontaneous pregnancies. Short stature was common, with growth hormone axis disruption in 30% of these. Of patients exposed to thyroid radiation 51% developed nodules, 30% malignant. Metabolic disturbances included hypertension, dyslipidemias, with both excess and underweight reported. Fragility fractures occurred in 6%; avascular necrosis in 6%. 13% developed SMNs, risk continuing to rise throughout follow-up. Conclusions We confirm gonadal dysfunction, multiple endocrine and metabolic abnormalities, thyroid cancer and SMNs, as common sequelae of HSCT, and identify gaps in management - particularly the need for informed fertility counselling and pretreatment fertility preservation, evaluation and management of bone health, and underline need for early lifestyle modification, long-term surveillance, and prospective planned studies aimed at reducing complication risk.

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Burden of Morbidity after Allogeneic Blood or Marrow Transplantation for Inborn Errors of Metabolism: A BMT Survivor Study Report

Cited by 2 publications

References 20 publications

Risk factors predicting need for the pediatric intensive care unit (PICU) post-hematopoietic cell transplant, PICU utilization, and outcomes following HCT: a single center retrospective analysis

Risk factors predicting need for the pediatric intensive care unit (PICU) post-hematopoietic cell transplant, PICU utilization, and outcomes following HCT: a single center retrospective analysis

The Late Effects of Hematopoietic Stem Cell Transplants in Pediatric Patients: A 25-Year Review

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