Evidence map›Paper›PMID 39718183›Full record

ArticleThe Lancet. Rheumatology2025

Allogeneic haematopoietic stem-cell transplantation for children with refractory systemic juvenile idiopathic arthritis and associated lung disease: outcomes from an international, retrospective cohort study.

Michael G Matt, Daniel Drozdov, Elisabeth Bendstrup, Mia Glerup, Ellen-Margrethe Hauge, Tania Masmas, Elvira Cannizzaro Schneider, Ulrike B Zeilhofer, Rolla F Abu-Arja, Kyla D Driest and 15 more

Abstract readMulticenter Study
PubMed Full text at PMC Publisher
In one paragraph

Article in The Lancet. Rheumatology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.

0numbers the graph read from it
0cells of the map it votes in
6citing 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

6 citing papers in PubMed.

  1. Article
  2. Article
  3. Review
  4. Observational
  5. Review
  6. 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

25 authors.

Michael G MattDivision of Rheumatology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
Daniel DrozdovDivision of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA; Division of Hematology/Oncology, Department of Pediatrics, Kantonsspital Aarau, Aarau, Switzerland; Division of Stem Cell Transplantation, University Children's Hospital Zurich, Zurich, Switzerland.
Elisabeth BendstrupCenter for Rare Lung Diseases, Department of Respiratory Diseases and Allergy, Aarhus University Hospital and Department of Clinical Medicine, Aarhus University Aarhus, Denmark.
Mia GlerupDepartment of Child and Adolescent Medicine, Aarhus University Hospital and Department of Clinical Medicine, Aarhus University Aarhus, Denmark.
Ellen-Margrethe HaugeDepartment of Rheumatology, Aarhus University Hospital and Department of Clinical Medicine, Aarhus University Aarhus, Denmark.
Tania MasmasDepartment of Paediatrics and Adolescent Medicine, Paediatric Haematopoietic Stem Cell Transplantation and Immunodeficiency, Copenhagen University Hospital, Copenhagen, Denmark.
Elvira Cannizzaro SchneiderDivision of Rheumatology Children's Research Center, University Children's Hospital Zurich, Zurich, Switzerland.
Ulrike B ZeilhoferDivision of Stem Cell Transplantation, University Children's Hospital Zurich, Zurich, Switzerland.
Rolla F Abu-ArjaPediatric Blood and Marrow Transplant Program, Nationwide Children's Hospital and The Ohio State University, Columbus, OH, USA.
Kyla D DriestDivision of Rheumatology, Nationwide Children's Hospital and The Ohio State University, Columbus, OH, USA.
Joseph H OvedStem Cell Transplantation and Cellular Therapies, MSK Kids, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
Karen OnelDivision of Pediatric Rheumatology, Hospital for Special Surgery, New York, NY, USA.
Christen L EbensDivision of Blood and Marrow Transplant and Cellular Therapy, Department of Pediatrics, University of Minnesota, Minneapolis, MN, USA.
Deepakbabu ChellapandianCancer and Blood Disorders Institute, Johns Hopkins All Children's Hospital, St Petersburg, FL, USA.
Shanmuganathan ChandrakasanDepartments of Pediatrics and Human Genetics, Emory University School of Medicine, and Children's Healthcare of Atlanta, Atlanta, GA, USA.
Sampath PrahaladDepartments of Pediatrics and Human Genetics, Emory University School of Medicine, and Children's Healthcare of Atlanta, Atlanta, GA, USA.
Johannes RothChildren's Hospital of Eastern Ontario and University of Ottawa, Ottawa, ON, Canada; Children's Hospital Lucerne and Faculty of Health Science and Medicine, University of Lucerne, Lucerne, Switzerland.
Susan E ProckopDana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA, USA.
Juliana SilvaGreat Ormond Street Hospital for Children, London, UK.
Andrew H SchapiroDepartment of Radiology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA; Department of Radiology, Cincinnati, OH, USA; Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, USA.
Christopher ToweDivision of Pulmonary Medicine, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA; Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, USA.
Sharat ChandraDivision of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA; Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, USA.
Alexei GromDivision of Rheumatology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA; Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, USA.
Grant S SchulertDivision of Rheumatology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA; Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, USA. Electronic address: grant.schulert@cchmc.org.
Rebecca A MarshDivision of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA; Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, USA; Pharming Healthcare, Warren, NJ, USA. Electronic address: rebecca.marsh@cchmc.org.

Funding

X-RAY CRYSTALLOGRAPHYP30CA008748 · NCI · SLOAN-KETTERING INSTITUTE FOR CANCER RES · PI SELWYN M VICKERS · 1985 to 2026
$347.4M
Pathogenesis of Systemic Juvenile Idiopathic Arthritis-Associated Lung DiseaseR01AR079524 · NIAMS · CINCINNATI CHILDRENS HOSP MED CTR · PI Grant Sanford Schulert · 2022 to 2026
$2.6M
NCI NIH HHS P30 CA008748NIAMS NIH HHS R01 AR079524
6 · The paper itself

Abstract

backgroundSystemic juvenile idiopathic arthritis-related lung disease (sJIA-LD) is a severe complication in patients with treatment-refractory systemic juvenile idiopathic arthritis (sJIA). The objective of this study was to evaluate the effect of allogeneic haematopoietic stem-cell transplantation (HSCT) in a cohort of children with sJIA-LD.

methodsThis international, retrospective cohort study was performed in nine hospitals across the USA and Europe in children with sJIA-LD who had received allogeneic HSCT. Patients' medical charts were reviewed and their data extracted using a standardised form. The outcomes assessed were allogeneic HSCT outcomes (eg, engraftment and donor chimerism, and transplant-related complications), pulmonary outcomes (eg, oxygen dependence, chest CT findings, and pulmonary function test results), and overall outcomes (eg, death, complete response, or partial response). A complete response was defined as resolution of signs of sJIA without the need for systemic immunomodulatory therapy, in addition to discontinuation of supplemental oxygen.

findingsBetween Jan 18, 2018, and Oct 17, 2022, 13 patients with sJIA-LD, who were refractory to immunosuppressive treatment and who had received an average of six different treatment agents, underwent allogeneic HSCT. Ten (77%) of 13 patients were female and three (23%) were male. The median age at diagnosis of sJIA-LD was 4·8 years (IQR 2·9-14·8) and the median age at transplantation was 9·0 years (5·0-19·0). Pre-HSCT chest CT revealed characteristic sJIA-LD. Five patients required supplemental oxygen before transplantation. Patients received various reduced toxicity or intensity conditioning regimens. Grafts were from 10/10 HLA-matched (n=6) or 9/10 HLA-mismatched (n=5) unrelated donors, a 7/10 related donor (n=1), and a matched sibling (n=1). All patients engrafted. One patient had secondary graft failure and received a second transplant from a different donor. Post-transplantation complications included acute graft-versus-host disease (n=5), bacteraemia (n=8), cytomegalovirus reactivation (n=6), and post-transplantation macrophage activation syndrome (n=3). Four patients died; two from cytomegalovirus pneumonitis, one from intracranial haemorrhage, and one from progressive sJIA-LD. At a median follow-up of 16 months (IQR 6-24), all nine surviving patients had a complete response, with no active features of sJIA, no biological therapy or corticosteroid use, and no supplemental oxygen dependence.

interpretationAllogeneic HSCT might be a valuable treatment option for patients with refractory sJIA and sJIA-LD and should be considered for children with worsening oxygen dependence or severe treatment-related morbidity.

fundingNational Institute of Arthritis and Musculoskeletal and Skin Diseases (R01-AR079525).

Indexed as

Arthritis, JuvenileHematopoietic Stem Cell TransplantationLung DiseasesAdolescentChildChild, PreschoolEuropeFemaleHumansMaleRetrospective StudiesTransplantation, HomologousTreatment Outcome

Identifiers

PMID39718183
PMCPMC11949720

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Registered trials

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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.