Evidence map›Paper›PMID 33401150›Full record

Trial reportBlood cells, molecules & diseases2021

Congenital dyserythropoietic anemia type I: First report from the Congenital Dyserythropoietic Anemia Registry of North America (CDAR).

Omar Niss, Robert B Lorsbach, Mikaela Berger, Satheesh Chonat, Morgan McLemore, David Buchbinder, Timothy McCavit, Linda G Shaffer, Jessica Simpson, Jeffrey H Schwartz and 6 more

Registry-linked trialOpen access · greenAbstract readClinical Trial
In one paragraph

Trial report in Blood cells, molecules & diseases, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02964494 (The Congenital Dyserythropoietic Anemia Registry), which is not on this map. Cited by 8 papers.

0numbers the graph read from it
0cells of the map it votes in
8citing papers in PubMed
0.6field-weighted citation impact, top 31% 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.

NCT02964494 recruitingnot on this map

The Congenital Dyserythropoietic Anemia Registry (CDAR)

Typeobservational_patient_registrySponsorChildren's Hospital Medical Center, CincinnatiRan2016 to 2031Enrolled10,000ConditionsCongenital Dyserythropoietic Anemia (CDA)
3 · Its place in the literature

Who cites it

8 citing papers in PubMed, 12 citations in OpenAlex.

  1. Article
  2. Article
  3. Molecular surprises in evaluations of red cell disorders.Hematology. American Society of Hematology. Education Program · 2025
    Review
  4. Mechanism of ASF1 engagement by CDAN1.Nature communications · 2025
    Article
  5. Article
  6. Mechanism of ASF1 Inhibition by CDAN1.bioRxiv : the preprint server for biology · 2024
    Article
  7. Mutations in theHaematologica · 2023
    Article
  8. Frontiers in physiology · 2021
    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

16 authors at 8 institutions in 1 country.

Omar NissDivision of Hematology, Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA; Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, USA. Electronic address: Omar.niss@cchmc.org.
Robert B LorsbachDepartment of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, USA; Division of Pathology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
Mikaela BergerDivision of Hematology, Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA; Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, USA.
Satheesh ChonatDepartment of Pediatrics, Emory University School of Medicine, Atlanta, GA, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Atlanta, GA, USA.
Morgan McLemoreDepartment of Hematology and Medical Oncology, Winship Cancer Institute of Emory University School of Medicine, Atlanta, GA, USA.
David BuchbinderDepartment of Hematology, CHOC Children's Hospital, Orange, CA, USA.
Timothy McCavitCook Children's Hospital, Fort Worth, TX, USA.
Linda G ShafferDell Children's Medical Center, Austin, TX, USA.
Jessica SimpsonUF Health Pediatric Subspecialty Program in Pensacola, Pensacola, FL, USA.
Jeffrey H SchwartzUF Health Pediatric Subspecialty Program in Pensacola, Pensacola, FL, USA.
Jessica MeznarichDivision of Hematology-Oncology, Department of Pediatrics, University of Utah, Salt Lake City, UT, USA; Primary Children's Hospital, Intermountain Healthcare, Salt Lake City, UT, USA.
Myesa EmbereshDivision of Hematology, Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
Katie G SeuDivision of Hematology, Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
Wenying ZhangDepartment of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, USA; Laboratory of Genetics and Genomics, Division of Human Genetics, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
Theodosia A KalfaDivision of Hematology, Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA; Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, USA. Electronic address: theodosia.kalfa@cchmc.org.
CDAR consortium
Cincinnati Children's Hospital Medical Center · USUniversity of Florida Health · USEmory University · USChildren's Hospital of Orange County · USCook Children's Medical Center · USDell Children's Medical Center of Central Texas · USIntermountain Healthcare · USPrimary Children's Hospital · US

Funding

LCenter for Clinical and Translational Science and TrainingUL1TR001425 · NCATS · UNIVERSITY OF CINCINNATI · PI HEUBI, JAMES E., KISSELA, BRETT M · 2015 to 2024
$37.5M
The Role of Erythroblastic Islands in Anemia of InflammationR01HL152099 · NHLBI · CINCINNATI CHILDRENS HOSP MED CTR · PI BLANC, LIONEL, KALFA, THEODOSIA ANASTASIOS · 2020 to 2023
$2.5M
NCATS NIH HHS UL1 TR001425NHLBI NIH HHS R01 HL152099
6 · The paper itself

Abstract

Congenital dyserythropoietic anemias (CDAs) are characterized by ineffective erythropoiesis and distinctive erythroblast abnormalities; the diagnosis is often missed or delayed due to significant phenotypic heterogeneity. We established the CDA Registry of North America (CDAR) to study the natural history of CDA and create a biorepository to investigate the pathobiology of this heterogeneous disease. Seven of 47 patients enrolled so far in CDAR have CDA-I due to biallelic CDAN1 mutations. They all presented with perinatal anemia and required transfusions during infancy. Anemia spontaneously improved during infancy in three patients; two became transfusion-independent rapidly after starting interferon-α

Indexed as

AdolescentAdultAnemia, Dyserythropoietic, CongenitalBlood TransfusionBone MarrowChildChild, PreschoolFemaleGenetic TestingGlycoproteinsHumansMaleMutationNorth AmericaNuclear ProteinsRegistriesCDAN1 protein, humanGlycoproteinsNuclear ProteinsAnemiaCDAN1Congenital dyserythropoietic anemiaErythropoiesisRare disease registry

Identifiers

PMID33401150
PMCPMC8809105
OpenAlexW3116803674

What OpenQuestion 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 OpenQuestion graph.