Evidence map›Paper›PMID 40083103›Full record

ArticleClinical and experimental pediatrics2025

Hematopoietic stem cell transplantation in pediatric patients with type VI mucopolysaccharidosis.

Vedat Uygun, Koray Yalçın, Hayriye Daloğlu, Seda Öztürkmen, Suna Çelen, Suleimen Zhumatayev, Gülsün Karasu, Akif Yeşilipek

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Clinical and experimental pediatrics, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed
–field-weighted citation impact
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

2 citing papers in PubMed.

  1. Review
  2. Advances in Therapies for Mucopolysaccharidoses.Current issues in molecular biology · 2026
    Review
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.

Vedat UygunDepartment of Pediatric Bone Marrow Transplantation Unit, Faculty of Medicine, İstinye University, Medical Park Antalya Hospital, Antalya, Turkey.
Koray YalçınDepartment of Pediatric Bone Marrow Transplantation Unit, Faculty of Medicine, Bahçeşehir University, Medical Park Göztepe Hospital, İstanbul, Turkey.
Hayriye DaloğluDepartment of Pediatric Bone Marrow Transplantation Unit, Medical Park Antalya Hospital, Antalya, Turkey.
Seda ÖztürkmenDepartment of Pediatric Bone Marrow Transplantation Unit, Medical Park Antalya Hospital, Antalya, Turkey.
Suna ÇelenDepartment of Pediatric Bone Marrow Transplantation Unit, Faculty of Medicine, Bahçeşehir University, Medical Park Göztepe Hospital, İstanbul, Turkey.
Suleimen ZhumatayevDepartment of Pediatric Bone Marrow Transplantation Unit, Medical Park Göztepe Hospital, İstanbul, Turkey.
Gülsün KarasuDepartment of Pediatric Bone Marrow Transplantation Unit, Medical Park Antalya Hospital, Antalya, Turkey.
Akif YeşilipekDepartment of Pediatric Bone Marrow Transplantation Unit, Medical Park Antalya Hospital, Antalya, Turkey.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundIt is uncertain whether hematopoietic stem cell transplantation (HSCT), versus standard enzyme replacement therapy (ERT), is effective for type VI mucopolysaccharidosis (MPS VI). PURPOSE: New related advances in HSCT prompted an examination of the transplant procedures performed in a recent cohort.

methodsThis single-center retrospective study reviewed the medical records of 17 pediatric patients with MPS VI who underwent allogeneic HSCT in 2021-2023. All conditioning regimens were myeloablative. Engraftment days, complications, and survival data were recorded. As follow-up was short, we recorded only 6-minute walk test distance before versus after HSCT.

resultsThe patients underwent transplantation at a median of 6-year postdiagnosis. All were engrafted and had a full or mixed chimerism. Enzyme levels were within normal ranges. Walking tests of all evaluable patients improved at a median 9-month follow-up.

conclusionHSCT aims to improve the disease and provides a permanent solution at the enzyme level, eliminating ERT. Our study showed that HSCT, a less expensive and permanent treatment option, should be offered to patients with MPS VI.

Indexed as

ChildEnzyme replacement therapyHematopoietic stem cell transplantationMaroteaux-Lamy syndromeMucopolysaccharidoses

Identifiers

PMID40083103
PMCPMC12326044

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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.