Literature DB >> 31764167

Registries for study of nonmalignant hematological diseases: the example of the Severe Chronic Neutropenia International Registry.

David C Dale1, Audrey Anna Bolyard2, Laurie A Steele1, Cornelia Zeidler3, Karl Welte4.   

Abstract

PURPOSE OF REVIEW: Registries provide 'real world' perspectives on the natural history and outcomes for many clinical conditions. The purpose of this review is to identify registries for nonmalignant hematological disease and to describe the operation of a successful long-term registry for patients with severe chronic neutropenia. RECENT
FINDINGS: There was an upswing in registries about 20 years ago, based on optimism about their utility to improve patient care. To show value, registries must define outcomes for populations of patients with specific medical conditions and the effects of treatment. This is challenging for many reasons. The Severe Chronic Neutropenia International Registry is an example of a successful registry. This report describes underlying reasons for its success.
SUMMARY: Registries are important to organize and analyze clinical information across geographic, ethnic and social boundaries. They are also challenging to organize, administer and support.

Entities:  

Year:  2020        PMID: 31764167      PMCID: PMC7236759          DOI: 10.1097/MOH.0000000000000558

Source DB:  PubMed          Journal:  Curr Opin Hematol        ISSN: 1065-6251            Impact factor:   3.284


  51 in total

1.  The European internet-based patient and research database for primary immunodeficiencies: update 2011.

Authors:  B Gathmann; N Binder; S Ehl; G Kindle
Journal:  Clin Exp Immunol       Date:  2012-03       Impact factor: 4.330

2.  Digenic mutations in severe congenital neutropenia.

Authors:  Manuela Germeshausen; Cornelia Zeidler; Manfred Stuhrmann; Marina Lanciotti; Matthias Ballmaier; Karl Welte
Journal:  Haematologica       Date:  2010-03-10       Impact factor: 9.941

3.  Elastase inhibitors as potential therapies for ELANE-associated neutropenia.

Authors:  Vahagn Makaryan; Merideth L Kelley; Breanna Fletcher; Audrey Anna Bolyard; A Andrew Aprikyan; David C Dale
Journal:  J Leukoc Biol       Date:  2017-07-28       Impact factor: 4.962

4.  Extended spectrum of human glucose-6-phosphatase catalytic subunit 3 deficiency: novel genotypes and phenotypic variability in severe congenital neutropenia.

Authors:  Kaan Boztug; Philip S Rosenberg; Marie Dorda; Siddharth Banka; Thomas Moulton; Julie Curtin; Nima Rezaei; John Corns; Jeffrey W Innis; Zekai Avci; Hung Chi Tran; Isabelle Pellier; Paolo Pierani; Rachel Fruge; Nima Parvaneh; Setareh Mamishi; Rajen Mody; Phil Darbyshire; Jayashree Motwani; Jennie Murray; George R Buchanan; William G Newman; Blanche P Alter; Laurence A Boxer; Jean Donadieu; Karl Welte; Christoph Klein
Journal:  J Pediatr       Date:  2011-11-01       Impact factor: 4.406

5.  Mutations in ELA2, encoding neutrophil elastase, define a 21-day biological clock in cyclic haematopoiesis.

Authors:  M Horwitz; K F Benson; R E Person; A G Aprikyan; D C Dale
Journal:  Nat Genet       Date:  1999-12       Impact factor: 38.330

6.  The cellular and molecular mechanisms for neutropenia in Barth syndrome.

Authors:  Vahagn Makaryan; Willem Kulik; Frederic M Vaz; Christopher Allen; Yigal Dror; David C Dale; Andrew A Aprikyan
Journal:  Eur J Haematol       Date:  2011-12-04       Impact factor: 2.997

Review 7.  Clinical and molecular pathophysiology of Shwachman-Diamond syndrome: an update.

Authors:  Kasiani C Myers; Stella M Davies; Akiko Shimamura
Journal:  Hematol Oncol Clin North Am       Date:  2012-11-03       Impact factor: 3.722

8.  Purification and biochemical characterization of human pluripotent hematopoietic colony-stimulating factor.

Authors:  K Welte; E Platzer; L Lu; J L Gabrilove; E Levi; R Mertelsmann; M A Moore
Journal:  Proc Natl Acad Sci U S A       Date:  1985-03       Impact factor: 11.205

9.  The United Kingdom Primary Immune Deficiency (UKPID) Registry: report of the first 4 years' activity 2008-2012.

Authors:  J D M Edgar; M Buckland; D Guzman; N P Conlon; V Knerr; C Bangs; V Reiser; Z Panahloo; S Workman; M Slatter; A R Gennery; E G Davies; Z Allwood; P D Arkwright; M Helbert; H J Longhurst; S Grigoriadou; L A Devlin; A Huissoon; M T Krishna; S Hackett; D S Kumararatne; A M Condliffe; H Baxendale; K Henderson; C Bethune; C Symons; P Wood; K Ford; S Patel; R Jain; S Jolles; T El-Shanawany; H Alachkar; A Herwadkar; R Sargur; A Shrimpton; G Hayman; M Abuzakouk; G Spickett; C J Darroch; S Paulus; S E Marshall; E M McDermott; P T Heath; R Herriot; S Noorani; M Turner; S Khan; B Grimbacher
Journal:  Clin Exp Immunol       Date:  2014-01       Impact factor: 4.330

10.  Characteristics and temporal trends in patient registries: focus on the life sciences industry, 1981-2012.

Authors:  Karin Travers; Rachel H Sallum; Meghan D Burns; Charles E Barr; Mary S Beattie; Chris L Pashos; Bryan R Luce
Journal:  Pharmacoepidemiol Drug Saf       Date:  2014-07-31       Impact factor: 2.890

View more
  5 in total

1.  Chronic neutropenic colitis with complete colonic obstruction in a patient with severe congenital neutropenia associated with G6PC3 mutations.

Authors:  Taxiarchis Konstantinos Nikolouzakis; Konstantinos Spyridakis; Maria Tzardi; Grigorios Tsaknakis; Maria Ximeri; Maksim Klimiankou; Emmanuel Chrysos; Julia Skokowa; Helen A Papadaki
Journal:  Ann Hematol       Date:  2022-01-27       Impact factor: 3.673

2.  Identification of GFI1 mutations in adult patients with congenital neutropenia.

Authors:  Erasmia Boutakoglou; Maksim Klimiankou; Grigorios Tsaknakis; Elias Drakos; Julia Skokowa; Helen A Papadaki
Journal:  Ann Hematol       Date:  2022-09-16       Impact factor: 4.030

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

Authors:  Omar Niss; Robert B Lorsbach; Mikaela Berger; Satheesh Chonat; Morgan McLemore; David Buchbinder; Timothy McCavit; Linda G Shaffer; Jessica Simpson; Jeffrey H Schwartz; Jessica Meznarich; Myesa Emberesh; Katie G Seu; Wenying Zhang; Theodosia A Kalfa
Journal:  Blood Cells Mol Dis       Date:  2020-12-24       Impact factor: 3.039

4.  Outcomes for patients with severe chronic neutropenia treated with granulocyte colony-stimulating factor.

Authors:  David C Dale; Audrey Anna Bolyard; James A Shannon; James A Connelly; Daniel C Link; Mary Ann Bonilla; Peter E Newburger
Journal:  Blood Adv       Date:  2022-07-12

5.  Congenital and Acquired Chronic Neutropenias: Challenges, Perspectives and Implementation of the EuNet-INNOCHRON Action.

Authors:  Helen A Papadaki; Irene Mavroudi; Antonio Almeida; Juergen Bux; Joanna Cichy; David C Dale; Jean Donadieu; Petter Höglund; Oliver Karanfilski; Cristina Mecucci; Jan Palmblad; Julia Skokowa; Kostas Stamatopoulos; Ivo Touw; Alan J Warren; Karl Welte; Cornelia Zeidler; Carlo Dufour
Journal:  Hemasphere       Date:  2020-06-08
  5 in total

北京卡尤迪生物科技股份有限公司 © 2022-2023.