Evidence map›Paper›PMID 41958357›Full record

ArticleBritish journal of haematology2026

Treosulfan-fludarabine conditioning in infants with severe combined immunodeficiencies: Extended study of the UK paediatric treosulfan study.

Su Han Lum, Sinéad Greener, Irum Latif Memon, Persis Amrolia, Zohreh Nademi, Robert Chiesa, Juliana Silva, Helen Young, Stephen Owens, Eleri Williams and 6 more

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

Article in British journal of haematology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

16 authors.

Su Han LumTranslational & Clinical Research Institute, Newcastle University, Newcastle upon Tyne, UK.ORCID https://orcid.org/0000-0002-5471-029X
Sinéad GreenerTranslational & Clinical Research Institute, Newcastle University, Newcastle upon Tyne, UK.
Irum Latif MemonBone Marrow Transplant Unit, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.
Persis AmroliaBone Marrow Transplant Unit, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.ORCID https://orcid.org/0000-0003-0480-3911
Zohreh NademiTranslational & Clinical Research Institute, Newcastle University, Newcastle upon Tyne, UK.
Robert ChiesaBone Marrow Transplant Unit, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.
Juliana SilvaBone Marrow Transplant Unit, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.
Helen YoungChildren's Haematopoietic Stem Cell Transplant Unit, Great North Children's Hospital, Newcastle Upon Tyne, UK.
Stephen OwensChildren's Haematopoietic Stem Cell Transplant Unit, Great North Children's Hospital, Newcastle Upon Tyne, UK.
Eleri WilliamsChildren's Haematopoietic Stem Cell Transplant Unit, Great North Children's Hospital, Newcastle Upon Tyne, UK.
Khuen Foong NgTranslational & Clinical Research Institute, Newcastle University, Newcastle upon Tyne, UK.ORCID https://orcid.org/0000-0002-8767-3974
Terry FloodChildren's Haematopoietic Stem Cell Transplant Unit, Great North Children's Hospital, Newcastle Upon Tyne, UK.
Andrew R GenneryTranslational & Clinical Research Institute, Newcastle University, Newcastle upon Tyne, UK.
Sophie HambletonTranslational & Clinical Research Institute, Newcastle University, Newcastle upon Tyne, UK.
Kanchan RaoChildren's Haematopoietic Stem Cell Transplant Unit, Great North Children's Hospital, Newcastle Upon Tyne, UK.ORCID https://orcid.org/0000-0001-9449-8476
Mary SlatterTranslational & Clinical Research Institute, Newcastle University, Newcastle upon Tyne, UK.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Allogeneic haematopoietic stem cell transplantation (HSCT) is a curative therapy for severe combined immunodeficiency (SCID). Conditioning improves donor engraftment and freedom from immunoglobulin replacement (IgR) but increases the risks of acute and late toxicity. Treosulfan, a reduced toxicity alkylating agent, has emerged as an alternative to busulfan. In this UK multicentre study, we evaluated outcomes of 104 infants with SCID who underwent first HSCT following treosulfan-fludarabine conditioning between 2006 and 2022. After a median follow-up of 5.4 years, 5-year overall survival (OS) and event-free survivals (EFS) were 81% and 77% respectively. On multivariate analysis, molecularly undefined SCID (OS hazard ratio [HR] 5.61; EFS HR 5.55) and pre-HSCT cytomegalovirus (CMV) infection (OS HR 3.94; EFS 3.68) were independently associated with inferior OS and EFS; RAG-DCLRE1C genotypes also predicted worse EFS (HR 4.35). Cumulative incidence of endothelial cell dysfunction (ECD) was 11%. Treosulfan dose was not associated with OS, EFS, ECD or donor myeloid chimerism. Low mixed donor myeloid chimerism was observed across all treosulfan doses, but IgR freedom was achieved in 92% of survivors after first HSCT. Treosulfan-fludarabine provides excellent survival with low endothelial toxicity for SCID HSCT, with potential for optimisation via pharmacokinetic guided dosing.

Indexed as

BusulfanHematopoietic Stem Cell TransplantationSevere Combined ImmunodeficiencyTransplantation ConditioningVidarabineFemaleHumansInfantMaleUnited KingdomBusulfanfludarabinetreosulfanVidarabinefludarabineHSCTSCIDtreosulfan

Identifiers

PMID41958357
PMCPMC13176534

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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.