Evidence map›Paper›PMID 35840745›Full record

ArticleBone marrow transplantation2022

Late endocrine effects after hematopoietic stem cell transplantation in children with nonmalignant diseases.

L C de Kloet, J E Bense, M Y E C van der Stoep, M Louwerens, E G J von Asmuth, A C Lankester, A P J de Pagter, S E Hannema

Open access · bronzeAbstract read
PubMed Publisher
In one paragraph

Article in Bone marrow transplantation, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
16citing papers in PubMed, 2 pooled it
8.0field-weighted citation impact, top 2% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

16 citing papers in PubMed, 2 syntheses or guidelines pooled it, 23 citations in OpenAlex.

  1. Pooled it
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  8. 5-ALA/FeFrontiers in cell and developmental biology · 2026
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

8 authors at 1 institution in 1 country.

L C de Kloet *Willem-Alexander Children's Hospital, Department of Pediatrics, Division of Endocrinology, Leiden University Medical Center, Leiden, The Netherlands.
J E Bense *Willem-Alexander Children's Hospital, Department of Pediatrics, Division of Pediatric Stem Cell Transplantation Program, Leiden University Medical Center, Leiden, The Netherlands.ORCID http://orcid.org/0000-0002-7939-334X
M Y E C van der StoepDepartment of Clinical Pharmacy and Toxicology, Leiden University Medical Center, Leiden, The Netherlands.
M LouwerensDepartment of Internal Medicine, Leiden University Medical Center, Leiden, The Netherlands.
E G J von AsmuthWillem-Alexander Children's Hospital, Department of Pediatrics, Division of Pediatric Stem Cell Transplantation Program, Leiden University Medical Center, Leiden, The Netherlands.ORCID http://orcid.org/0000-0001-6256-7457
A C LankesterWillem-Alexander Children's Hospital, Department of Pediatrics, Division of Pediatric Stem Cell Transplantation Program, Leiden University Medical Center, Leiden, The Netherlands.ORCID http://orcid.org/0000-0002-6703-2993
A P J de PagterWillem-Alexander Children's Hospital, Department of Pediatrics, Division of Pediatric Stem Cell Transplantation Program, Leiden University Medical Center, Leiden, The Netherlands. LEEF@lumc.nl.ORCID http://orcid.org/0000-0002-7051-1872
S E HannemaWillem-Alexander Children's Hospital, Department of Pediatrics, Division of Endocrinology, Leiden University Medical Center, Leiden, The Netherlands.ORCID http://orcid.org/0000-0002-8996-0993
Leiden University Medical Center · NL

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

The number of children undergoing hematopoietic stem cell transplantation (HSCT) for nonmalignant diseases has increased in recent years. Endocrine complications are common after HSCT for malignant diseases, while little is known about long-term prevalence and risk factors in children transplanted for nonmalignant diseases. We retrospectively evaluated gonadal function, near adult height and thyroid function in 197 survivors of pediatric HSCT for hemoglobinopathies (n = 66), inborn errors of immunity/metabolism (n = 74) and bone marrow failure disorders (n = 57); median follow-up was 6.2 years (range 3.0-10.5). Gonadal dysfunction occurred in 55% of (post)pubertal females, was still present at last assessment in 43% and was more common after busulfan- than treosulfan-based conditioning (HR 10.6, CI 2.2-52.7; adjusted for HSCT indication). Gonadal dysfunction occurred in 39% of (post)pubertal males, was still present at last assessment in 32% and was less common in those who were prepubertal compared to (post)pubertal at HSCT (HR 0.11; CI 0.05-0.21). Near adult height was more than 2 SDS below mean parental height in 21% of males and 8% of females. Hypothyroidism occurred in 16% of patients; 4% received thyroxin treatment. In conclusion, endocrine complications, especially gonadal dysfunction, are common after pediatric HSCT for nonmalignant conditions. In females, treosulfan seems less gonadotoxic than busulfan. Careful long-term endocrine follow-up is indicated.

Indexed as

BusulfanHematopoietic Stem Cell TransplantationAdultChildFemaleHumansMaleRetrospective StudiesThyroxineTransplantation ConditioningBusulfanThyroxinetreosulfan

Identifiers

PMID35840745
OpenAlexW4285589265

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.