Evidence map›Paper›PMID 30519529›Full record

ArticleMolecular genetics and metabolism reports2019

Allogeneic stem cell transplantation with reduced intensity conditioning for patients with adrenoleukodystrophy.

Koji Kato, Ryo Maemura, Manabu Wakamatsu, Ayako Yamamori, Motoharu Hamada, Shinsuke Kataoka, Atsushi Narita, Shunsuke Miwata, Yuko Sekiya, Nozomu Kawashima and 11 more

Open access · goldAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular genetics and metabolism reports, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers.

0numbers the graph read from it
0cells of the map it votes in
9citing papers in PubMed
0.8field-weighted citation impact, top 27% 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

9 citing papers in PubMed, 15 citations in OpenAlex.

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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

21 authors at 3 institutions in 1 country.

Koji KatoDepartment of Hematology and Oncology, Children' Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, Japan.
Ryo MaemuraDepartment of Hematology and Oncology, Children' Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, Japan.
Manabu WakamatsuDepartment of Hematology and Oncology, Children' Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, Japan.
Ayako YamamoriDepartment of Hematology and Oncology, Children' Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, Japan.
Motoharu HamadaDepartment of Hematology and Oncology, Children' Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, Japan.
Shinsuke KataokaDepartment of Hematology and Oncology, Children' Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, Japan.
Atsushi NaritaDepartment of Hematology and Oncology, Children' Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, Japan.
Shunsuke MiwataDepartment of Hematology and Oncology, Children' Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, Japan.
Yuko SekiyaDepartment of Hematology and Oncology, Children' Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, Japan.
Nozomu KawashimaDepartment of Hematology and Oncology, Children' Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, Japan.
Kyogo SuzukiDepartment of Hematology and Oncology, Children' Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, Japan.
Kotaro NaritaDepartment of Hematology and Oncology, Children' Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, Japan.
Sayoko DoisakiDepartment of Hematology and Oncology, Children' Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, Japan.
Hideki MuramatsuDepartment of Hematology and Oncology, Children' Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, Japan.
Hirotoshi SakaguchiDepartment of Hematology and Oncology, Children' Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, Japan.
Kimikazu MatsumotoDepartment of Hematology and Oncology, Children' Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, Japan.
Yuka KoikeDepartment of Neurology, Clinical Neuroscience Branch, Brain Research Institute, Niigata University, Niigata, Japan.
Osamu OnoderaDepartment of Neurology, Clinical Neuroscience Branch, Brain Research Institute, Niigata University, Niigata, Japan.
Makiko KagaDepartment of Developmental Disorders, National Center of Neurology and Psychiatry, National Institute of Mental Health, Tokyo, Japan.
Nobuyuki ShimozawaDivision of Genomics Research, Life Science Research Center, Gifu University, Gifu, Japan.
Nao YoshidaDepartment of Hematology and Oncology, Children' Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, Japan.
Niigata University · JPGifu University · JPNational Center of Neurology and Psychiatry · JP

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

objectiveThe prognosis of adrenoleukodystrophy (ALD)with neurological involvement is generally dismal; however, allogeneic stem cell transplantation (SCT) is recognized as effective to stabilize or improve the clinical symptoms of ALD. Herein, we report the clinical outcomes of patients with ALD who consecutively underwent allogeneic stem cell transplantation with reduced intensity conditioning at our institution. PATIENTS: Sixteen patients with ALD, who were symptomatic (n = 14) or presymptomatic (n = 2), received SCT from 2010 to 2016. The stem cell source was cord blood (n = 14), or bone marrow from a human leukocyte antigen identical sibling (n = 2). The conditioning regimen prior to transplantation was reduced intensity and consisted of fludarabine (125 mg/m

resultsPrimary engraftment was obtained in 11 patients, and 4 of the 5 patients who lost the primary graft received a second cord blood transplantation and were engrafted. Five years overall and event-free survival were 90.9% and 61.1% respectively, with a median of 45 months (range 16-91). Loes score stabilized or improved by 18 months after transplantation except for patients with internal capsule involvement.

conclusionAllogeneic SCT with reduced intensity conditioning for patients with ALD was safely performed without major transplant-related complications even in symptomatic patients and neurological symptoms were stabilized after SCT in patients without internal capsule involvement.

Indexed as

AdrenoleukodystrophyALD, adrenoleukodystrophyAllogeneic stem cell transplantationATG, anti-thymocyte globulinBM, bone marrowBMT, bone marrow transplantationCB, cord bloodCMV, cytomegalovirusCSA, cyclosporine ACY, cyclophosphamideDQ, developmental quotientEBV, Epstein-Barr virusEFS, event free survivalFISH, fluorescent in situ hybridizationFLU, fludarabineGd, GadoliniumGVHD, graft-versus host diseaseHHV-6, human herpesvirus-6HLA, human leukocyte antigenIC, internal capsuleIQ, intelligence quotientLoes scoreMAC, myeloablative conditioningMEL, melphalanMRI, magnetic resonance imagingMTX, methotrexateOS, overall survivalRIC, reduced intensity conditioningSCT, stem cell transplantationVery long chain fatty acidVLCFA, very long chain fatty acid

Identifiers

PMID30519529
PMCPMC6260425
OpenAlexW2900850685

What OpenQuestion holds

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LicenceCC BY-NC-ND
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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.