Evidence map›Paper›PMID 36935283›Full record

ReviewTrends in molecular medicine2023

Updates and advances in pyruvate kinase deficiency.

Neeti Luke, Kirsty Hillier, Hanny Al-Samkari, Rachael F Grace

Open access · greenAbstract readReview
In one paragraph

Review in Trends in molecular medicine, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
13citing papers in PubMed, 1 pooled it
4.4field-weighted citation impact, top 5% 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

13 citing papers in PubMed, 1 synthesis or guideline pooled it, 25 citations in OpenAlex.

  1. Pooled it
  2. Trial
  3. Article
  4. Article
  5. Glycolysis enzymes and cellular lactylation in tumour.Clinical and translational medicine · 2026
    Review
  6. Review
  7. Article
  8. Article
  9. Review
  10. Article
  11. Article
  12. Review
  13. Metabolomic analysis ofbioRxiv : the preprint server for biology · 2023
    Article
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

4 authors at 1 institution in 1 country.

Neeti LukeDepartment of Pediatrics, Division of Pediatric Hematology-Oncology, Hassenfeld Children's Hospital at NYU Langone Health, NYU Grossman School of Medicine, New York, NY, USA.
Kirsty HillierDepartment of Pediatrics, Division of Pediatric Hematology-Oncology, Hassenfeld Children's Hospital at NYU Langone Health, NYU Grossman School of Medicine, New York, NY, USA.
Hanny Al-SamkariDivision of Hematology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.
Rachael F GraceDana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Boston, MA, USA. Electronic address: Rachael.Grace@childrens.harvard.edu.
Harvard University · US

Funding

Antiangiogenic Therapy to Reduce Bleeding and Improve Health-Related Quality of Life in Hereditary Hemorrhagic TelangiectasiaK23HL159313 · NHLBI · MASSACHUSETTS GENERAL HOSPITAL · PI Hanny T Al-Samkari · 2022 to 2026
$999k
NHLBI NIH HHS K23 HL159313
6 · The paper itself

Abstract

Mutations in the PKLR gene lead to pyruvate kinase (PK) deficiency, causing chronic hemolytic anemia secondary to reduced red cell energy, which is crucial for maintenance of the red cell membrane and function. Heterogeneous clinical manifestations can result in significant morbidity and reduced health-related quality of life. Treatment options have historically been limited to supportive care, including red cell transfusions and splenectomy. Current disease-modifying treatment considerations include an oral allosteric PK activator, mitapivat, which was recently approved for adults with PK deficiency, and gene therapy, which is currently undergoing clinical trials. Studies evaluating the role of PK activators in other congenital hemolytic anemias are ongoing. The long-term effect of treatment with disease-modifying therapy in PK deficiency will require continued evaluation.

Indexed as

Anemia, Hemolytic, Congenital NonspherocyticPyruvate Metabolism, Inborn ErrorsAdultHumansPyruvate KinaseQuality of LifePyruvate Kinasecongenital hemolytic anemiagene therapymitapivatpyruvate kinase deficiencytransfusions

Identifiers

PMID36935283
PMCPMC11088755
OpenAlexW4327725905

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.