Pub Date : 2025-10-07DOI: 10.1016/S2352-4642(25)00267-6
Matheus van Rens , Robin van der Lee , Giovanni Barone , Fiammetta Piersigilli
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Pub Date : 2025-09-25DOI: 10.1016/S2352-4642(25)00274-3
Maria Antonietta De Ioris , Franco Locatelli
{"title":"Long-term sequelae in survivors of high-risk neuroblastoma: the price of success","authors":"Maria Antonietta De Ioris , Franco Locatelli","doi":"10.1016/S2352-4642(25)00274-3","DOIUrl":"10.1016/S2352-4642(25)00274-3","url":null,"abstract":"","PeriodicalId":54238,"journal":{"name":"Lancet Child & Adolescent Health","volume":"9 11","pages":"Pages 754-755"},"PeriodicalIF":15.5,"publicationDate":"2025-09-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145182693","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-09-25DOI: 10.1016/S2352-4642(25)00241-X
Tara O Henderson MD , Jenna K Bardwell MPH , Pei-Chi Kao MPH , Paul C Nathan MD , Wendy Landier PhD , Sogol Mostoufi-Moab MD , Tara M Brinkman PhD , Fiona Schulte PhD , Julie R Park MD , Saro H Armenian DO , Arlene Naranjo PhD , Meera Subramaniam MD , Araz Marachellian MD , Sharon J Diskin PhD , Wendy B London PhD , Lisa R Diller MD
<div><h3>Background</h3><div>New treatment regimens have resulted in increasing numbers of children who survive high-risk neuroblastoma, but late effects of cancer therapy are not well studied. We aimed to explore treatment-related risk factors associated with late effects of modern therapies for high-risk neuroblastoma.</div></div><div><h3>Methods</h3><div>This multicentre, cross-sectional cohort study of survivors of high-risk neuroblastoma was done at 88 hospitals participating in the Children's Oncology Group (COG) in North America, Australia, and New Zealand. Eligible participants were aged 5–50 years at enrolment, were enrolled on ANBL00B1, were diagnosed with high-risk neuroblastoma on or after Jan 1, 2000, were alive at least 5 years after diagnosis, and had language skills in English, French, or Spanish. Individuals with active neuroblastoma relapse (cytotoxic chemotherapy in the previous 2 years) were ineligible. Participant evaluations included an audiogram, pulmonary function test, echocardiogram, and laboratory studies. Exposures were abstracted via chart review. Prevalence of a specific late effect was calculated as the number of participants with the late effect divided by the sample size with known data for that late effect. Multivariable logistic regression models examined associations between treatment-related risk factors versus hearing loss, growth failure, underweight, and restrictive lung disease.</div></div><div><h3>Findings</h3><div>Between June 5, 2017 and Sept 17, 2021, 375 eligible participants were enrolled from 890 potentially eligible individuals. Median age at high-risk neuroblastoma diagnosis was 2·5 years (range 0·2–15·8), median age at enrolment was 12·0 years (5·0–24·0), and median time from diagnosis to enrolment was 9·0 years (5·1–18·2). All participants received chemotherapy, 363 (97%) of 375 received at least one stem cell transplant and 231 (64%) of 363 patients with known data received anti-GD2 therapy. Moderate-to-severe hearing loss was identified in 236 (72%) of 327 participants. Growth failure was identified in 87 (24%) of 360 and underweight in 190 (51%) of 373. Pulmonary function tests revealed moderate-to-severe restrictive lung disease in 17 (8%) of 207 participants. Longer follow-up was associated with a higher prevalence of late effects. Compared with single stem-cell transplant, exposure to tandem stem-cell transplant was associated with increased risk of growth failure (odds ratio [OR] 3·4 [95% CI 1·6–7·2], p=0·0016] and moderate-to-severe restrictive lung disease (OR 4·5 [1·1–18·3]; p=0·033). Neither conditioning regimen exposure (carboplatin–etoposide–melphalan <em>vs</em> busulfan–melphalan) nor anti-GD2 therapy was associated with increased risk of hearing loss, underweight, growth failure, or restrictive lung disease.</div></div><div><h3>Interpretation</h3><div>Survivors of high-risk neuroblastoma treated with modern therapies demonstrated a substantial burden of late effects, providing cr
{"title":"Late effects after high-risk neuroblastoma (LEAHRN): a multicentre, cross-sectional cohort study from the Children's Oncology Group","authors":"Tara O Henderson MD , Jenna K Bardwell MPH , Pei-Chi Kao MPH , Paul C Nathan MD , Wendy Landier PhD , Sogol Mostoufi-Moab MD , Tara M Brinkman PhD , Fiona Schulte PhD , Julie R Park MD , Saro H Armenian DO , Arlene Naranjo PhD , Meera Subramaniam MD , Araz Marachellian MD , Sharon J Diskin PhD , Wendy B London PhD , Lisa R Diller MD","doi":"10.1016/S2352-4642(25)00241-X","DOIUrl":"10.1016/S2352-4642(25)00241-X","url":null,"abstract":"<div><h3>Background</h3><div>New treatment regimens have resulted in increasing numbers of children who survive high-risk neuroblastoma, but late effects of cancer therapy are not well studied. We aimed to explore treatment-related risk factors associated with late effects of modern therapies for high-risk neuroblastoma.</div></div><div><h3>Methods</h3><div>This multicentre, cross-sectional cohort study of survivors of high-risk neuroblastoma was done at 88 hospitals participating in the Children's Oncology Group (COG) in North America, Australia, and New Zealand. Eligible participants were aged 5–50 years at enrolment, were enrolled on ANBL00B1, were diagnosed with high-risk neuroblastoma on or after Jan 1, 2000, were alive at least 5 years after diagnosis, and had language skills in English, French, or Spanish. Individuals with active neuroblastoma relapse (cytotoxic chemotherapy in the previous 2 years) were ineligible. Participant evaluations included an audiogram, pulmonary function test, echocardiogram, and laboratory studies. Exposures were abstracted via chart review. Prevalence of a specific late effect was calculated as the number of participants with the late effect divided by the sample size with known data for that late effect. Multivariable logistic regression models examined associations between treatment-related risk factors versus hearing loss, growth failure, underweight, and restrictive lung disease.</div></div><div><h3>Findings</h3><div>Between June 5, 2017 and Sept 17, 2021, 375 eligible participants were enrolled from 890 potentially eligible individuals. Median age at high-risk neuroblastoma diagnosis was 2·5 years (range 0·2–15·8), median age at enrolment was 12·0 years (5·0–24·0), and median time from diagnosis to enrolment was 9·0 years (5·1–18·2). All participants received chemotherapy, 363 (97%) of 375 received at least one stem cell transplant and 231 (64%) of 363 patients with known data received anti-GD2 therapy. Moderate-to-severe hearing loss was identified in 236 (72%) of 327 participants. Growth failure was identified in 87 (24%) of 360 and underweight in 190 (51%) of 373. Pulmonary function tests revealed moderate-to-severe restrictive lung disease in 17 (8%) of 207 participants. Longer follow-up was associated with a higher prevalence of late effects. Compared with single stem-cell transplant, exposure to tandem stem-cell transplant was associated with increased risk of growth failure (odds ratio [OR] 3·4 [95% CI 1·6–7·2], p=0·0016] and moderate-to-severe restrictive lung disease (OR 4·5 [1·1–18·3]; p=0·033). Neither conditioning regimen exposure (carboplatin–etoposide–melphalan <em>vs</em> busulfan–melphalan) nor anti-GD2 therapy was associated with increased risk of hearing loss, underweight, growth failure, or restrictive lung disease.</div></div><div><h3>Interpretation</h3><div>Survivors of high-risk neuroblastoma treated with modern therapies demonstrated a substantial burden of late effects, providing cr","PeriodicalId":54238,"journal":{"name":"Lancet Child & Adolescent Health","volume":"9 11","pages":"Pages 776-786"},"PeriodicalIF":15.5,"publicationDate":"2025-09-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145182692","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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