Survival Trends in Infants Undergoing Allogeneic Hematopoietic Cell Transplant

Parikh, Suhag; Satwani, Prakash; Ahn, Kwang Woo; Sahr, Natasha; Fretham, Caitrin; Abraham, Allistair; Agrawal, Vaibhav; Auletta, Jeffery J.; Abdel‐Azim, Hisham; Copelan, Edward A.; Díaz, Miguel Ángel; Dvorak, Christopher C.; Frangoul, Haydar; Freytes, César O.; Gadalla, Shahinaz M.; Gale, Robert Peter; George, Biju; Gergis, Usama; Hashmi, Shahrukh K.; Hematti, Peiman; Keating, Amy K.; Lazarus, Hillard M.; Myers, Kasiani C.; Olsson, Richard F.; Prestidge, Timothy; Rotz, Seth J.; Savani, Bipin N.; Shereck, Evan; Williams, Kirsten M.; Wirk, Baldeep; Pasquini, Marcelo C.; Loren, Alison W.

doi:10.1001/jamapediatrics.2019.0081

Cited by 17 publications

(15 citation statements)

References 26 publications

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“…6 Despite these achievements, mortality following pediatric HCT remains significant, and efforts to identify novel risk factors for inferior outcomes are essential. 7 Social determinants of health, including poverty, are widely recognized as major contributors to both adult and pediatric health outcomes in the United States. [8][9][10] While studies in both pediatric oncology [11][12][13][14] and adult HCT [15][16][17] indicate that socioeconomic status is an independent predictor of inferior survival outcomes, including both relapse and overall survival, limited data exist exploring the pertinence of socioeconomic status in pediatric HCT.…”

Section: Introductionmentioning

confidence: 99%

Neighborhood poverty and pediatric allogeneic hematopoietic cell transplantation outcomes: a CIBMTR analysis

Bona

Brazauskas

He³

et al. 2021

Blood

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Social determinants of health, including poverty, contribute significantly to health outcomes in the United States, yet their impact on pediatric hematopoietic cell transplantation (HCT) outcomes is poorly understood. We aimed to identify the association between neighborhood-poverty and HCT outcomes for pediatric allogeneic HCT recipients in the Center for International Blood and Marrow Transplant Research (CIBMTR) database. We assembled two pediatric cohorts who received a first, allogeneic HCT from 2006-2015 at age ≤18 years; including 2053 children with malignant disease and 1696 children with non-malignant disease. Neighborhood-poverty exposure was defined a priori per U.S. Census definition as living in a high-poverty ZIP code (>=20% of persons below 100% Federal Poverty Level) and used as the primary predictor in all analyses. Our primary outcome was overall survival (OS) defined as time from HCT until death from any cause. Secondary outcomes included relapse and transplant-related mortality (TRM) in malignant disease, acute and chronic GVHD, and infection in the first 100 days post-HCT. Among children transplanted for non-malignant disease, neighborhood-poverty was not associated with any HCT outcome. Among children transplanted for malignant disease, neighborhood-poverty conferred an increased risk of TRM but was not associated with inferior OS or any other transplant outcome. Among children with malignant disease, a key secondary finding was that children with Medicaid insurance experienced inferior OS and increased TRM compared to those with private insurance. These data suggest opportunities for future investigation of household-level poverty exposures on HCT outcomes in pediatric malignant disease to inform care delivery interventions.

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Section: Introductionmentioning

confidence: 99%

Neighborhood poverty and pediatric allogeneic hematopoietic cell transplantation outcomes: a CIBMTR analysis

Bona

Brazauskas

He³

et al. 2021

Blood

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“…In 472 infants transplanted for malignant diseases in 2000–2014 and reported to the Centre for International Blood and Marrow Transplant Research (CIBMTR), 182 of whom had ALL, Parikh et al observed no improvement in the outcome measures over time, as opposed to the general improvement of transplant results for most other ages and disease groups over time. Both the rate of relapse and the rate of toxicity remained high, with a high incidence of sinusoidal obstruction syndrome (SOS) ( 18 ). These data illustrate the challenges in infant ALL with high risks of both toxicity and relapse.…”

Section: Conventional Strategies Including Haematopoietic Stem Cell T...mentioning

confidence: 99%

“…A study conducted by Tracey et al, including patients with ALL aged ≤18 years old, concluded that neither a TBI dose in excess of 13 Gy nor the addition of etoposide to cyclophosphamide could improve OS after HSCT but did increase TRM ( 18 ). TRM, as expected, was generally higher in patients >10 years old compared to in patients <10 years old ( 21 ).…”

Section: Conventional Strategies Including Haematopoietic Stem Cell T...mentioning

confidence: 99%

Acute Lymphoblastic Leukaemia in the Youngest: Haematopoietic Stem Cell Transplantation and Beyond

et al. 2022

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The ALL SCTped 2012 FORUM (For Omitting Radiation Under Majority age) trial compared outcomes for children ≥4 years of age transplanted for acute lymphoblastic leukaemia (ALL) who were randomised to myeloablation with a total body irradiation (TBI)-based or chemotherapy-based conditioning regimen. The TBI-based preparation was associated with a lower rate of relapse compared with chemoconditioning. Nevertheless, the age considered suitable for TBI was progressively raised over time to spare the most fragile youngest patients from irradiation-related complications. The best approach to use for children <4 years of age remains unclear. Children diagnosed with ALL in their first year of life, defined as infants, have a remarkably poorer prognosis compared with older children. This is largely explained by the biology of their ALL, with infants often carrying a KMT2A gene rearrangement, as well as by their fragility. In contrast, the clinical presentations and biological features of ALL in children >1 year but <4 years often resemble those presented by older children. In this review, we explore the state of the art regarding haematopoietic stem cell transplantation (HSCT) in children <4 years, the preparative regimens available, and new developments in the field that may influence treatment decisions.

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“…Despite the survival of both children reported by Martinez et al, there is an expected mortality at least 10% for children with severe immune disorders undergoing HSCT. 7 Moreover, both children in this study developed both acute and chronic graft-versus-host disease (GVHD), which was fortunately highly responsive to steroids, but with any allogeneic HSCT performed for a chronic nonmalignant condition, there is the possibility of exchanging 1 chronic disorder for another (ie, chronic GVHD). As the authors point out, the risk of GVHD might be reduced by interferon blockade during the acute phase of transplantation.…”

Section: Stella M Davies | Cincinnati Children's Hospital Medical Centermentioning

confidence: 93%

Grasping the sword of Damocles

Davies

2021

Blood

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Survival Trends in Infants Undergoing Allogeneic Hematopoietic Cell Transplant

Abstract: IMPORTANCE Studies demonstrating improved survival after allogeneic hematopoietic cell transplant generally exclude infants.OBJECTIVE To analyze overall survival trends and other outcomes among infants who undergo allogeneic hematopoietic cell transplant.

Cited by 17 publications

References 26 publications

Neighborhood poverty and pediatric allogeneic hematopoietic cell transplantation outcomes: a CIBMTR analysis

Neighborhood poverty and pediatric allogeneic hematopoietic cell transplantation outcomes: a CIBMTR analysis

Acute Lymphoblastic Leukaemia in the Youngest: Haematopoietic Stem Cell Transplantation and Beyond

Grasping the sword of Damocles

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