Evidence map›Paper›PMID 42583025›Full record

ArticleBlood red cells & iron2026

Results from the first clinical trial of bitopertin for steroid-relapsed/refractory Diamond-Blackfan anemia.

David J Young, Tania R Machado, Bretagne C Cowling, Raymond T Doty, Sanjay Venugopal, Colin O Wu, Janis L Abkowitz, William Savage, Cynthia E Dunbar

Registry-linked trialAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Blood red cells & iron, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT05828108 (A Phase I/II, Intra-Patient Dose-Escalation Study of the Selective GlyT1 Inhibitor, Bitopertin for Steroid-Refractory Diamond-Blackfan Anemia.), which is not on this 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.

NCT05828108 phase1 / phase2completednot on this map

A Phase I/II, Intra-Patient Dose-Escalation Study of the Selective GlyT1 Inhibitor, Bitopertin for Steroid-Refractory Diamond-Blackfan Anemia.

TypeinterventionalSponsorNational Heart, Lung, and Blood Institute (NHLBI)Ran2023 to 2025Enrolled15ConditionsSteroid-refractory Diamond-Blackfan Anemia (DBA)ArmsBitopertin
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

9 authors.

David J YoungTranslational Stem Cell Biology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD, USA.ORCID 0000-0002-0885-8568
Tania R MachadoOffice of the Clinical Director, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD, USA.
Bretagne C CowlingOffice of the Clinical Director, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD, USA.
Raymond T DotyDivision of Hematology, Department of Medicine, University of Washington, Seattle, WA, USA.ORCID 0000-0002-9312-8622
Sanjay VenugopalOffice of the Clinical Director, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD, USA.
Colin O WuOffice of Biostatistics Research, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD, USA.ORCID 0000-0002-4514-0926
Janis L AbkowitzDivision of Hematology, Department of Medicine, University of Washington, Seattle, WA, USA.ORCID 0000-0002-1820-5341
William SavageDisc Medicine, Watertown, MA, USA.
Cynthia E DunbarTranslational Stem Cell Biology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD, USA.ORCID 0000-0002-7645-838X

Funding

Intramural NIH HHS Z99 HL999999
6 · The paper itself

Abstract

Diamond-Blackfan anemia (DBA) is an inherited anemia characterized by ribosome defects that reduce globin chain production, producing an excess of toxic heme. Bitopertin selectively reduces heme synthesis, rebalances globin-heme production, and improves anemia in model systems. We report the first clinical trial of bitopertin for the treatment of steroid-relapsed/refractory DBA. This was an open-label, intrapatient dose-escalation study in 15 heavily pretreated, transfusion-dependent adult DBA patients (ClinicalTrials.gov NCT05828108). Bitopertin dosing increased monthly (5 mg escalating to 60 mg, daily), followed by 3-months maintenance. Primary endpoint was response (hemoglobin level, erythrocyte transfusions). Secondary endpoints were safety (adverse events, AE) and tolerability. Twelve patients completed the full 8-month course (2 discontinued early for non-bitopertin-related serious AE (SAE), 1 lost-to-follow-up). All 12 patients reached the planned 60-mg maximum dose. Two de-escalated to 40 mg (1 grade-2 dizziness, 1 concern for loss of early effect). Seven SAEs were reported, all unlikely/unrelated to bitopertin. Plasma drug levels were consistent with prior studies. At 10 mg daily and greater, all patients reached levels consistent with EC

Indexed as

bitopertinDiamond-Blackfan anemiaglycine uptake inhibitorheme synthesisribosomopathy

Identifiers

PMID42583025
PMCPMC13459865

What Socratic holds

Textmetadata
LicenceTDM
Read underepoch 390

Registered trials

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 Socratic graph.