The combination of methotrexate and cytosine arabinoside in newly diagnosed adult Langerhans cell histiocytosis: a prospective phase II interventional clinical trial

Abstract

Background: Langerhans Cell Histiocytosis (LCH) is a rare disease puzzling both children and adults, however outcome of adult patients is unfavorable. This prospective interventional trial aims to test the efficacy and safety of the combination of methotrexate and cytosine arabinoside in adult LCH patients. Method: A total of 36 patients enrolled diagnosed with LCH and treated in our center from 1st Jan, 2014 to 30th Jun, 2016. Result: Nineteen patients underwent the detection of BRAF mutation, with a positive rate of 21.1%. The overall response rate was 100%, only 16.7% achieved complete response. The overall regression rate of osseous lesions was 100%. Regression of central nervous system involvement was also favorable. After a median follow-up of 44 months, the estimated event-free survival was 48.9 months, the overall survival rate was 97.2%. The risk organ involvement showed strong prognostic value, EFS was 34.1 or 54.6 months (p = 0.001) in groups with/without risk organ involvement respectively. Neutropenia and thrombocytopenia were the most common adverse effects. Conclusion: The regimen of methotrexate and cytosine arabinoside (MA) is effective and safe in treating adult LCH patients, and timely preventions may be considered for the high incidence of hematological adverse effects. Trial registration: Trial No. NCT02389400 on Clinicaltrials.gov, registered on 10th Mar. 2015.

Spontaneous Thyroid Hemorrhage Caused by Langerhans Cell Histiocytosis: A Case Report and Literature Review

“…The bones, skin, pituitary gland, liver, spleen, hematopoietic system, and lungs are most frequently affected by LCH. Thyroid LCH involvement is not frequent, affecting 9%-25% of individuals with LCH ( 32 – 35 ). To our knowledge, this is the first reported case of spontaneous hemorrhage caused by thyroid gland LCH in the literature.…”

Management der Langerhans-Zell-Histiozytose bei erwachsenen Patienten

“…In einer prospektiven Studie haben Patienten mit multifokaler SS-oder MS-LCH in hohem Maß auf eine Chemotherapie mit hochdosiertem Methotrexat und Cytarabin angesprochen [24]. Dies betraf vor allem Krankheitsmanifestationen in Skelett und ZNS [27], was insbesondere auf die gute Passage der Blut-Hirn-Schranke durch beide Zytostatika zurückgeführt wurde bei jedoch insgesamt ausgeprägter hämatologischer Toxizität.…”