Evidence map›Paper›PMID 30792199›Full record

Trial reportHaematologica2019

The International Hereditary Thrombotic Thrombocytopenic Purpura Registry: key findings at enrollment until 2017.

Hendrika A van Dorland, Magnus Mansouri Taleghani, Kazuya Sakai, Kenneth D Friedman, James N George, Ingrid Hrachovinova, Paul N Knöbl, Anne Sophie von Krogh, Reinhard Schneppenheim, Isabella Aebi-Huber and 13 more

Abstract readClinical TrialMulticenter Study
In one paragraph

Trial report in Haematologica, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 88 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
88citing papers in PubMed, 2 pooled it
–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

88 citing papers in PubMed, 2 syntheses or guidelines pooled it.

  1. Guideline
  2. Pooled it
  3. Trial
  4. Review
  5. Mutation Spectrum ofInternational journal of molecular sciences · 2026
    Article
  6. Article
  7. Article
  8. Article
  9. Article
  10. Review
  11. Article
  12. Platelet Disorders and Medication Strategies.Thrombosis and haemostasis · 2026
    Review
  13. Article
  14. Review
  15. Review
  16. Review
  17. Article
  18. Modern management of congenital thrombotic thrombocytopenic purpura (cTTP).Hematology. American Society of Hematology. Education Program · 2025
    Article
  19. Optimizing the management of congenital thrombotic thrombocytopenic purpura.Research and practice in thrombosis and haemostasis · 2025
    Article
  20. Article

28 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

23 authors.

Hendrika A van DorlandDepartment of Hematology and Central Hematology Laboratory, Inselspital, Bern University Hospital, Bern, Switzerland.
Magnus Mansouri TaleghaniDepartment of Hematology and Central Hematology Laboratory, Inselspital, Bern University Hospital, Bern, Switzerland.
Kazuya SakaiDepartment of Blood Transfusion Medicine, Nara Medical University, Kashihara, Japan.
Kenneth D FriedmanDivision of Hematology and Oncology, Medical College of Wisconsin, Milwaukee, WI, USA.
James N GeorgeDepartment of Biostatistics Epidemiology, University of Oklahoma Health Sciences Center, Oklahoma City, OK, USA.
Ingrid HrachovinovaNRL for Hemostasis, Institute of Hematology and Blood Transfusion, Prague, Czech Republic.
Paul N KnöblDivision of Hematology and Hemostasis, Department of Medicine 1, Medical University of Vienna, Austria.
Anne Sophie von KroghDepartment of Hematology, St Olavs Hospital, Trondheim University Hospital, Trondheim, Norway.
Reinhard SchneppenheimDepartment of Paediatric Haematology and Oncology, University Medical Centre Hamburg-Eppendorf, Hamburg, Germany.
Isabella Aebi-HuberDepartment of Hematology and Central Hematology Laboratory, Inselspital, Bern University Hospital, Bern, Switzerland.
Lukas BütikoferCTU Bern, University of Bern, Bern, Switzerland.
Carlo R LargiadèrUniversity Institute of Clinical Chemistry, Inselspital, Bern University Hospital, Bern, Switzerland.
Zuzana CermakovaBlood Center, University Hospital Ostrava, Ostrava, Czech Republic.
Koichi KokameDepartment of Molecular Pathogenesis, National Cerebral and Cardiovascular Center, Suita, Japan.
Toshiyuki MiyataDepartment of Molecular Pathogenesis, National Cerebral and Cardiovascular Center, Suita, Japan.
Hideo YagiDepartment of Blood Transfusion Medicine, Nara Medical University, Kashihara, Japan.
Deirdra R TerrellDepartment of Biostatistics Epidemiology, University of Oklahoma Health Sciences Center, Oklahoma City, OK, USA.
Sara K VeselyDepartment of Biostatistics Epidemiology, University of Oklahoma Health Sciences Center, Oklahoma City, OK, USA.
Masanori MatsumotoDepartment of Blood Transfusion Medicine, Nara Medical University, Kashihara, Japan.
Bernhard LämmleDepartment of Hematology and Central Hematology Laboratory, Inselspital, Bern University Hospital, Bern, Switzerland.
Yoshihiro FujimuraDepartment of Blood Transfusion Medicine, Nara Medical University, Kashihara, Japan.
Johanna A Kremer HovingaDepartment of Hematology and Central Hematology Laboratory, Inselspital, Bern University Hospital, Bern, Switzerland johanna.kremer@msel.ch.
Hereditary TTP Registry

Funding

Life after Recovery in Adult Thrombotic Thrombocytopenic Purpura (TTP): Let's Hear It from the SurvivorsK01HL135466 · NHLBI · UNIVERSITY OF OKLAHOMA HLTH SCIENCES CTR · PI TERRELL, DEIRDRA R · 2017 to 2021
$687k
NHLBI NIH HHS K01 HL135466
6 · The paper itself

Abstract

Congenital thrombotic thrombocytopenic purpura is an autosomal recessive inherited disease with a clinically heterogeneous course and an incompletely understood genotype-phenotype correlation. In 2006, the Hereditary TTP Registry started recruitment for a study which aimed to improve the understanding of this ultra-rare disease. The objective of this study is to present characteristics of the cohort until the end of 2017 and to explore the relationship between overt disease onset and ADAMTS13 activity with emphasis on the recurring

Indexed as

ADAMTS13 ProteinAllelesHeterozygoteHomozygoteMutationPurpura, Thrombotic ThrombocytopenicAdolescentAdultAgedAge of OnsetChildChild, PreschoolFemaleHumansInfantInfant, NewbornADAMTS13 ProteinADAMTS13 protein, human

Identifiers

PMID30792199
PMCPMC6886414

What OpenQuestion holds

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LicenceCC BY-NC
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.