Evidence map›Paper›PMID 38507116›Full record

ArticleInternational journal of hematology2024

Early hematopoietic cell transplantation for familial hemophagocytic lymphohistiocytosis in a regional treatment network in Japan.

Masataka Ishimura, Katsuhide Eguchi, Motoshi Sonoda, Tamami Tanaka, Akira Shiraishi, Yasunari Sakai, Takahiro Yasumi, Takayuki Miyamoto, Ilia Voskoboinik, Kunio Hashimoto and 5 more

Abstract read
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Article in International journal of hematology, 2024. 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

15 authors.

Masataka IshimuraDepartment of Pediatrics, Graduate School of Medical Sciences, Kyushu University, 3-1-1 Maidashi, Higashi-Ku, Fukuoka, 812-8582, Japan. ischii-pediatr@kyudai.jp.ORCID http://orcid.org/0000-0002-0735-6566
Katsuhide EguchiDepartment of Pediatrics, Graduate School of Medical Sciences, Kyushu University, 3-1-1 Maidashi, Higashi-Ku, Fukuoka, 812-8582, Japan.
Motoshi SonodaDepartment of Pediatrics, Graduate School of Medical Sciences, Kyushu University, 3-1-1 Maidashi, Higashi-Ku, Fukuoka, 812-8582, Japan.
Tamami TanakaDepartment of Pediatrics, Graduate School of Medical Sciences, Kyushu University, 3-1-1 Maidashi, Higashi-Ku, Fukuoka, 812-8582, Japan.
Akira ShiraishiDepartment of Pediatrics, Graduate School of Medical Sciences, Kyushu University, 3-1-1 Maidashi, Higashi-Ku, Fukuoka, 812-8582, Japan.
Yasunari SakaiDepartment of Pediatrics, Graduate School of Medical Sciences, Kyushu University, 3-1-1 Maidashi, Higashi-Ku, Fukuoka, 812-8582, Japan.
Takahiro YasumiDepartment of Pediatrics, Kyoto University Graduate School of Medicine, Kyoto, Japan.
Takayuki MiyamotoDepartment of Pediatrics, Kyoto University Graduate School of Medicine, Kyoto, Japan.
Ilia VoskoboinikCancer Immunology Program, Peter MacCallum Cancer Centre, Melbourne, Australia.
Kunio HashimotoDepartment of Pediatrics, Graduate School of Biomedical Sciences, Nagasaki University, Nagasaki, Japan.
Shirou MatsumotoDepartment of Pediatrics, Faculty of Life Science, Kumamoto University, Kumamoto, Japan.
Shuichi OzonoDepartment of Pediatrics and Child Health, Kurume University School of Medicine, Kurume, Fukuoka, Japan.
Hiroshi MoritakeDivision of Pediatrics, Faculty of Medicine, University of Miyazaki, Miyazaki, Japan.
Hidetoshi TakadaDepartment of Child Health, Faculty of Medicine, University of Tsukuba, Tsukuba, Japan.
Shouichi OhgaDepartment of Pediatrics, Graduate School of Medical Sciences, Kyushu University, 3-1-1 Maidashi, Higashi-Ku, Fukuoka, 812-8582, Japan.

Funding

Ministry of Health, Labour and Welfare 20FC1053
6 · The paper itself

Abstract

Familial hemophagocytic lymphohistiocytosis (FHLH) is a fatal hyperinflammation syndrome arising from the genetic defect of perforin-mediated cytolysis. Curative hematopoietic cell transplantation (HCT) is needed before development of central nervous system (CNS) disease. We studied treatment outcomes of 13 patients (FHLH2 n = 11, FHLH3 n = 2) consecutively diagnosed from 2011 to 2022 by flow cytometric screening for non-myeloablative HCT in a regional treatment network in Kyushu, Japan. One patient with a novel PRF1 variant escaped screening, but all patients with FHLH2 reached diagnosis and 8 of them received HCT until 3 and 9 months of age, respectively. The earliest HCT was conducted 65 days after birth. Three pretransplant deaths occurred in newborns with liver failure at diagnosis. Ten posttransplant patients have remained disease-free, 7 of whom had no neurological involvement. Time from first etoposide infusion to HCT was shorter in patients without CNS disease or bleeding than in patients with those factors (median [range] days: 62 [50-81] vs. 122 [89-209], p = 0.016). Six of 9 unrelated patients had a PRF1 c.1090_1091delCT variant. These results suggest that the critical times to start etoposide and HCT are within 3 months after birth and during etoposide control, respectively. Newborn screening may increase the percentage of disease-free survivors without complications.

Indexed as

Hematopoietic Stem Cell TransplantationLymphohistiocytosis, HemophagocyticPerforinChild, PreschoolEtoposideFemaleHumansInfantInfant, NewbornJapanMaleTreatment OutcomeEtoposidePerforinPRF1 protein, humanHematopoietic cell transplantationHyperinflammationPerforinRapid diagnosis

Identifiers

PMID38507116

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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.