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Subsequent neoplasms and late mortality in children undergoing allogeneic transplantation for nonmalignant diseases

2020· article· en· W3024374829 on OpenAlexaff
Justine M. Kahn, Ruta Brazauskas, Heather R. Tecca, Stephanie Bo‐Subait, David Buchbinder, Minoo Battiwala, Mary E.D. Flowers, Bipin N. Savani, Rachel Phelan, Larisa Broglie, Allistair Abraham, Amy K. Keating, Andrew Daly, Baldeep Wirk, Biju George, Blanche P. Alter, Celalettin Üstün, César O. Freytes, Amer Beitinjaneh, Christine Duncan, Edward A. Copelan, Gerhard Hildebrandt, Hemant S. Murthy, Hillard M. Lazarus, Jeffery J. Auletta, Kasiani C. Myers, Kirsten M. Williams, Kristin Page, Lynda M. Vrooman, Maxim Norkin, Michael Byrne, Miguel Ángel Díaz, Naynesh Kamani, Neel S. Bhatt, Andrew R. Rezvani, Nosha Farhadfar, Parinda A. Mehta, Peiman Hematti, Peter J. Shaw, Rammurti T. Kamble, Raquel M. Schears, Richard F. Olsson, Robert J. Hayashi, Robert Peter Gale, Samantha Mayo, Saurabh Chhabra, Seth J. Rotz, Sherif M. Badawy, Siddhartha Ganguly, Steven Z. Pavletic, Taiga Nishihori, Tim Prestidge, Vaibhav Agrawal, William J. Hogan, Yoshihiro Inamoto, Bronwen E. Shaw, Prakash Satwani

Bibliographic record

VenueBlood Advances · 2020
Typearticle
Languageen
FieldMedicine
TopicHematopoietic Stem Cell Transplantation
Canadian institutionsUniversity of Toronto
FundersJanssen PharmaceuticalsNational Institute of Allergy and Infectious DiseasesDaiichi Sankyo EuropeNational Cancer InstituteOffice of Naval ResearchLegend BiotechKite PharmaSanofi GenzymeTakeda OncologyAgency for Healthcare Research and QualityHealth Resources and Services Administrationbluebird bioKiadis PharmaDaiichi-SankyoSwedish Orphan BiovitrumOmeros CorporationAstellas PharmaAdaptive BiotechnologiesPfizerIncyteChimerixMedacJazz PharmaceuticalsAtara BiotherapeuticsActinium PharmaceuticalsNational Institutes of HealthRegeneron PharmaceuticalsNational Center for Advancing Translational SciencesJanssen BiotechHistoGeneticsU.S. Department of DefenseSanofiGlaxoSmithKlineNational Heart, Lung, and Blood InstituteNovartis Pharmaceuticals CorporationU.S. NavyCelgeneCSL BehringBristol-Myers SquibbTerumo BCTAstraZenecaBiomedical Advanced Research and Development AuthorityAmgenAstellas Pharma US
KeywordsMedicineInternal medicineTransplantationPopulationInterquartile rangeIncidence (geometry)Cumulative incidenceAplastic anemiaPediatricsGastroenterologySurgeryBone marrow

Abstract

fetched live from OpenAlex

We examined the risk of subsequent neoplasms (SNs) and late mortality in children and adolescents undergoing allogeneic hematopoietic cell transplantation (HCT) for nonmalignant diseases (NMDs). We included 6028 patients (median age, 6 years; interquartile range, 1-11; range, <1 to 20) from the Center for International Blood and Marrow Transplant Research (1995-2012) registry. Standardized mortality ratios (SMRs) in 2-year survivors and standardized incidence ratios (SIRs) were calculated to compare mortality and SN rates with expected rates in the general population. Median follow-up of survivors was 7.8 years. Diagnoses included severe aplastic anemia (SAA; 24%), Fanconi anemia (FA; 10%), other marrow failure (6%), hemoglobinopathy (15%), immunodeficiency (23%), and metabolic/leukodystrophy syndrome (22%). Ten-year survival was 93% (95% confidence interval [95% CI], 92% to 94%; SMR, 4.2; 95% CI, 3.7-4.8). Seventy-one patients developed SNs (1.2%). Incidence was highest in FA (5.5%), SAA (1.1%), and other marrow failure syndromes (1.7%); for other NMDs, incidence was <1%. Hematologic (27%), oropharyngeal (25%), and skin cancers (13%) were most common. Leukemia risk was highest in the first 5 years posttransplantation; oropharyngeal, skin, liver, and thyroid tumors primarily occurred after 5 years. Despite a low number of SNs, patients had an 11-fold increased SN risk (SIR, 11; 95% CI, 8.9-13.9) compared with the general population. We report excellent long-term survival and low SN incidence in an international cohort of children undergoing HCT for NMDs. The risk of SN development was highest in patients with FA and marrow failure syndromes, highlighting the need for long-term posttransplantation surveillance in this population.

Fetched live from OpenAlex and de-inverted. Abstracts are not stored in this database: the inverted indexes are 8.6 GB of the frame’s 9.3 GB of text, and the host has 13 GB free.

How this classification was reachedexpand

Full frame distilled prediction

Teacher imitation

Not calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.

metaresearch head score (Codex)0.000
metaresearch head score (Gemma)0.000
Version: codex-gemma-dda1882f352aValidation status: machine_predicted_unvalidated
Candidate categoriesnone
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Observational · Consensus signal: Observational
GenreCandidate signal: Empirical · Consensus signal: Empirical
Teacher disagreement score0.028
Threshold uncertainty score0.481

Codex and Gemma teacher scores by category

CategoryCodexGemma
Metaresearch0.0000.000
Meta-epidemiology (narrow)0.0000.000
Meta-epidemiology (broad)0.0000.000
Bibliometrics0.0000.000
Science and technology studies0.0000.000
Scholarly communication0.0000.000
Open science0.0000.000
Research integrity0.0000.000
Insufficient payload (model declined to judge)0.0000.000

Machine scores (provisional)

The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.

Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.

Opus teacher head0.018
GPT teacher head0.271
Teacher spread0.253 · how far apart the two teachers sit on this one work
Validation statusscore_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from it

Classification

machine, unvalidated

Machine predicted; a candidate call from one teacher head, not a consensus.

The models applied no category: nothing in the taxonomy fit this work.
Study designObservational
Domainnot available
GenreEmpirical

How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".

Quick stats

Citations32
Published2020
Admission routes1
Has abstractyes

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