Evidence map›Paper›PMID 41135032›Full record

Trial reportJournal of clinical oncology : official journal of the American Society of Clinical Oncology2025

US Food and Drug Administration Approval Summary: Imetelstat for Selected Patients With Low- to Intermediate-1 Risk Myelodysplastic Syndromes With Transfusion-Dependent Anemia.

Nina Kim, E Dianne Pulte, Lori A Ehrlich, Alexei C Ionan, Spencer Haupert, Jonathon Vallejo, Francis Green, Nan Zheng, Yun Wang, Jiang Liu and 10 more

Abstract readRandomized Controlled Trial
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Journal of clinical oncology : official journal of the American Society of Clinical Oncology, 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. Review
  2. How telomere attrition protects against cancer.Nature reviews. Genetics · 2026
    Review
  3. Review
  4. Review
  5. Article
  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

20 authors.

Nina KimCenter for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.ORCID 0000-0002-1809-0417
E Dianne PulteCenter for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.
Lori A EhrlichCenter for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.ORCID 0000-0001-5981-1332
Alexei C IonanCenter for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.ORCID 0000-0001-5892-8141
Spencer HaupertCenter for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.ORCID 0000-0001-5959-2301
Jonathon VallejoCenter for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.ORCID 0000-0002-9700-4037
Francis GreenCenter for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.
Nan ZhengCenter for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.
Yun WangCenter for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.
Jiang LiuCenter for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.
Javier G BlancoCenter for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.
Sarah E DorffCenter for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.ORCID 0009-0004-0762-5844
Brian BoothCenter for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.
Moran ChoeCenter for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.ORCID 0009-0009-4488-1616
Brenda GehrkeCenter for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.
Vishal BhatnagarOncology Center of Excellence, US Food and Drug Administration, Silver Spring, MD.ORCID 0000-0003-4650-0513
Marc TheoretOncology Center of Excellence, US Food and Drug Administration, Silver Spring, MD.ORCID 0000-0003-4973-5297
Richard PazdurOncology Center of Excellence, US Food and Drug Administration, Silver Spring, MD.ORCID 0000-0002-4771-9923
R Angelo De ClaroCenter for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.ORCID 0000-0001-8362-1977
Kelly J NorsworthyCenter for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD.ORCID 0000-0002-8462-9994

Funding

Intramural FDA HHS FD999999
6 · The paper itself

Abstract

On June 6, 2024, the US Food and Drug Administration (FDA) approved imetelstat (RYTELO, Geron) for adults with low- to intermediate-1 risk myelodysplastic syndromes (MDS) with transfusion-dependent anemia requiring ≥4 red blood cell units over 8 weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents. The approval was based on a randomized (2:1), double-blind, placebo-controlled multicenter trial, Study MDS3001. In the protocol-specified primary analysis, the ≥8-week RBC transfusion independence (RBC-TI) rate was 39.8% (95% CI, 30.9 to 49.3) in the imetelstat group versus 15% (95% CI, 7.1 to 26.6) in the placebo group (

Indexed as

AnemiaMyelodysplastic SyndromesAdultAgedAged, 80 and overDouble-Blind MethodDrug ApprovalErythrocyte TransfusionFemaleHumansMaleMiddle AgedOligonucleotidesUnited StatesUnited States Food and Drug AdministrationimetelstatOligonucleotides

Identifiers

PMID41135032
PMCPMC12614338

What OpenQuestion holds

Textmetadata
LicenceTDM
Read underepoch 390

Registered trials

None linked

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.