Long-term follow-up of mTOR inhibition for Erdheim-Chester disease-Reference-Cited by-同舟云学术

Long-term follow-up of mTOR inhibition for Erdheim-Chester disease

Published:2020-05-28 Issue:22 Volume:135 Page:1994-1997
ISSN:0006-4971
Container-title:Blood
language:en
Short-container-title:

Author:

Pegoraro Francesco¹^ORCID,Maniscalco Valerio¹,Peyronel Francesco²^ORCID,Westenend Pieter J.³^ORCID,Hendriksz Tadek R.⁴,Roperto Rosa M.⁵,Palumbo Alessandro A.⁶,Sieni Elena⁷^ORCID,Romagnani Paola⁵⁸^ORCID,van Bommel Eric F. H.⁹,Vaglio Augusto⁵⁸

Affiliation:

1. Department of Health Sciences, University of Firenze, Florence, Italy;

2. Nephrology Unit, University Hospital, Parma, Italy;

3. Department of Pathology and

4. Department of Radiology, Albert Schweitzer Hospital, Dutch National Center of expertise for retroperitoneal fibrosis, Dordrecht, The Netherlands;

5. Nephrology and Dialysis Unit, Meyer Children’s Hospital, Florence, Italy;

6. Department of Radiology, University Hospital, Parma, Italy;

7. Department of Pediatric Hematology and Oncology, Meyer Children’s Hospital, Florence, Italy;

8. Department of Biomedical, Experimental and Clinical Sciences “Mario Serio," University of Firenze, Florence, Italy; and

9. Department of Nephrology, Albert Schweitzer Hospital, Dutch National Center of Expertise for Retroperitoneal Fibrosis, Dordrecht, The Netherlands

Abstract

Two articles this week focus on Erdheim-Chester disease (ECD), a rare histiocytosis that mainly affects adults. Clonal somatic mutations primarily involving proteins in the BRAF and MPAK pathways have established ECD as a myeloid neoplasm, with targeted therapies now available for patients. In the first paper, an international panel presents new consensus recommendations for evaluation and treatment of ECD. In the second paper, Pegoraro and colleagues present long-term outcomes of patients with ECD treated with sirolimus, with responses in patients both with and without BRAF mutations.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

Link

http://ashpublications.org/blood/article-pdf/135/22/1994/1736017/bloodbld2019004478r1.pdf

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