Literature DB >> 33275748

A patient with Gaucher disease and plasma cell dyscrasia: bidirectional impact.

Ari Zimran1,2, Rosa Ruchlemer3,2, Shoshana Revel-Vilk1,2.   

Abstract

Patients with Gaucher disease (GD), a rare autosomal recessive glycosphingolipid storage disease, commonly present to hematologists with unexplained splenomegaly, thrombocytopenia, anemia, and bone symptoms. Patients with GD may develop other manifestations, such as autoimmune thrombocytopenia, monoclonal gammopathy, multiple myeloma, or, even more rarely, other hematological malignancies; sometimes they are first diagnosed during an assessment of those disorders. Although the diagnosis and management of patients with GD have significantly evolved over the last 30 years, some patients remain poor responders to GD-specific therapy, needing novel and investigational therapies. Ideally, patients with GD, like patients with other rare diseases, should be managed by a multidisciplinary team expert with the diverse clinical manifestations and potential GD-related or -unrelated comorbidities. The hematology community should be knowledgeable regarding the presentation and the variety of hematologic complications and comorbidities associated with Gaucher disease.
© 2020 by The American Society of Hematology.

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Mesh:

Year:  2020        PMID: 33275748      PMCID: PMC7727517          DOI: 10.1182/hematology.2020000123

Source DB:  PubMed          Journal:  Hematology Am Soc Hematol Educ Program        ISSN: 1520-4383


  30 in total

1.  Gaucher disease and cancer incidence: a study from the Gaucher Registry.

Authors:  Barry E Rosenbloom; Neal J Weinreb; Ari Zimran; Katherine A Kacena; Joel Charrow; Elizabeth Ward
Journal:  Blood       Date:  2005-02-17       Impact factor: 22.113

2.  Exploring the patient journey to diagnosis of Gaucher disease from the perspective of 212 patients with Gaucher disease and 16 Gaucher expert physicians.

Authors:  Atul Mehta; Nadia Belmatoug; Bruno Bembi; Patrick Deegan; Deborah Elstein; Özlem Göker-Alpan; Elena Lukina; Eugen Mengel; Kimitoshi Nakamura; Gregory M Pastores; Jordi Pérez-López; Ida Schwartz; Christine Serratrice; Jeffrey Szer; Ari Zimran; Maja Di Rocco; Zoya Panahloo; David J Kuter; Derralynn Hughes
Journal:  Mol Genet Metab       Date:  2017-08-04       Impact factor: 4.797

3.  MGUS, lymphoplasmacytic malignancies, and Gaucher disease: the significance of the clinical association.

Authors:  Neal J Weinreb; Pramod K Mistry; Barry E Rosenbloom; Madhav V Dhodapkar
Journal:  Blood       Date:  2018-04-12       Impact factor: 22.113

Review 4.  Substrate reduction therapy for GBA1-associated Parkinsonism: Are we betting on the wrong mouse?

Authors:  Ellen Sidransky; David Arkadir; Peter Bauer; Tama Dinur; Grisel Lopez; Arndt Rolfs; Ari Zimran
Journal:  Mov Disord       Date:  2019-11-11       Impact factor: 10.338

5.  Saposin C is a frequent target of paraproteins in Gaucher disease-associated MGUS/multiple myeloma.

Authors:  Klaus-Dieter Preuss; Carla E M Hollak; Natalie Fadle; Marinus van Oers; Evi Regitz; Michael Pfreundschuh
Journal:  Br J Haematol       Date:  2018-11-18       Impact factor: 6.998

6.  Low-dose low-frequency imiglucerase as a starting regimen of enzyme replacement therapy for patients with type I Gaucher disease.

Authors:  D Elstein; A Abrahamov; I Hadas-Halpern; A Meyer; A Zimran
Journal:  QJM       Date:  1998-07

7.  Phenotypic heterogeneity of N370S homozygotes with type I Gaucher disease: an analysis of 798 patients from the ICGG Gaucher Registry.

Authors:  C Fairley; A Zimran; M Phillips; M Cizmarik; J Yee; N Weinreb; S Packman
Journal:  J Inherit Metab Dis       Date:  2008-11-03       Impact factor: 4.982

Review 8.  Patient reported outcome measures in rare diseases: a narrative review.

Authors:  Anita Slade; Fatima Isa; Derek Kyte; Tanya Pankhurst; Larissa Kerecuk; James Ferguson; Graham Lipkin; Melanie Calvert
Journal:  Orphanet J Rare Dis       Date:  2018-04-23       Impact factor: 4.123

9.  Immunoglobulin Abnormalities in Gaucher Disease: an Analysis of 278 Patients Included in the French Gaucher Disease Registry.

Authors:  Yann Nguyen; Jérôme Stirnemann; Florent Lautredoux; Bérengère Cador; Monia Bengherbia; Karima Yousfi; Dalil Hamroun; Leonardo Astudillo; Thierry Billette de Villemeur; Anaïs Brassier; Fabrice Camou; Florence Dalbies; Dries Dobbelaere; Francis Gaches; Vanessa Leguy-Seguin; Agathe Masseau; Yves-Marie Pers; Samia Pichard; Christine Serratrice; Marc G Berger; Bruno Fantin; Nadia Belmatoug
Journal:  Int J Mol Sci       Date:  2020-02-13       Impact factor: 5.923

10.  Long Term Follow-Up of 103 Untreated Adult Patients with Type 1 Gaucher Disease.

Authors:  Tama Dinur; Ari Zimran; Michal Becker-Cohen; David Arkadir; Claudia Cozma; Marina Hovakimyan; Sebastian Oppermann; Laura Demuth; Arndt Rolfs; Shoshana Revel-Vilk
Journal:  J Clin Med       Date:  2019-10-11       Impact factor: 4.241

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  1 in total

Review 1.  Novel Management and Screening Approaches for Haematological Complications of Gaucher's Disease.

Authors:  Pilar Giraldo; Marcio Andrade-Campos
Journal:  J Blood Med       Date:  2021-12-07
  1 in total

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