Long-term follow-up of mTOR inhibition for Erdheim-Chester disease
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 f...
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| Veröffentlicht in: | Blood 2020-05, Vol.135 (22), p.1994-1997 |
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| Hauptverfasser: | , , , , , , , , , , |
| Format: | Artikel |
| Sprache: | eng |
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| container_end_page | 1997 |
|---|---|
| container_issue | 22 |
| container_start_page | 1994 |
| container_title | Blood |
| container_volume | 135 |
| creator | Pegoraro, Francesco Maniscalco, Valerio Peyronel, Francesco Westenend, Pieter J. Hendriksz, Tadek R. Roperto, Rosa M. Palumbo, Alessandro A. Sieni, Elena Romagnani, Paola van Bommel, Eric F.H. Vaglio, Augusto |
| description | 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. |
| doi_str_mv | 10.1182/blood.2019004478 |
| format | Article |
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| language | eng |
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| source | MEDLINE; Elektronische Zeitschriftenbibliothek - Frei zugängliche E-Journals; Alma/SFX Local Collection |
| subjects | Erdheim-Chester Disease - diagnostic imaging Erdheim-Chester Disease - drug therapy Erdheim-Chester Disease - genetics Follow-Up Studies Humans Mutation Prednisolone - therapeutic use Protein Kinase Inhibitors - therapeutic use Proto-Oncogene Proteins B-raf - genetics Time Factors Tomography, X-Ray Computed TOR Serine-Threonine Kinases - antagonists & inhibitors Treatment Outcome |
| title | Long-term follow-up of mTOR inhibition for Erdheim-Chester disease |
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