Single‐agent cladribine as an effective front‐line therapy for adults with Langerhans cell histiocytosis

Abstract

ID number selected at random. In addition, we will strongly recommend that all recipient groups ensure that every member of the staff sign a Confidentiality Agreement. We will develop a data and resource sharing agreement with the legal advice from the Compliance Office of the University of Alabama at Birmingham, and ensure that we follow NIH guidelines with respect to data sharing. Before data are shared with researchers, we will require that researchers enter into a data-sharing and resource-sharing agreement. This agreement will ensure that: (a) data are used only for research purposes; (b) data do not identify any individual participant; (c) data are secure, using appropriate computer technology; and (d) there is a commitment to destroy or return data after analyses are completed.

ALK-positive histiocytosis: a new clinicopathologic spectrum highlighting neurologic involvement and responses to ALK inhibition

“…cytotoxic chemotherapy, corticosteroids, immunosuppression and/or interferon-alpha), ALK inhibition, or a combination of these. As previously published for Erdheim-Chester disease (ECD) and Langerhans cell histiocytosis (LCH) 27,52,53 , responses to treatment as documented in the medical record were classified as complete response (complete resolution of disease), partial response (partial resolution of disease), stable disease (no significant change in lesions), or progressive disease (growth of known lesions or appearance of new lesions). Responses were extracted from the clinical medical records and based on formal clinical interpretations of ultrasound, computed tomography (CT), magnetic resonance imaging (MRI) and/or positron emission tomography (PET)-CT when available.…”

Histiocytosis and the nervous system: from diagnosis to targeted therapies

“…Purine-analog chemotherapies cytarabine and cladribine given as monotherapies are effective for neurologic LCH; some specialists use these agents as first-line therapy while others reserve them for relapsed or refractory disease. 27 , 36 , 37 The implications of BRAF and other MAPK pathway mutational status upon LCH treatment remain largely unclear. BRAF or MEK inhibitors have demonstrated efficacy in prospective trials of histiocytosis patients, although with a small proportion of LCH participants.…”

Histiocytic Neoplasms, Version 2.2021, NCCN Clinical Practice Guidelines in Oncology

“…63 A more recent retrospective study conducted at an NCCN Member Institution also showed a high ORR (79%) in adults with multifocal LCH (n=38; 82% multisystem) who were treated with cladribine, with complete response (CR) and partial response (PR) observed in 26% and 53% of patients, respectively. 64 Fiveyear overall survival (OS), progression-free survival (PFS), and duration of response (DOR) for the sample was 75%, 58%, and 70%, respectively. There is also evidence supporting clofarabine for relapsed/refractory LCH in the pediatric setting, with disease improvement observed in 73% of 11 patients.…”