克拉屈滨-成人朗格汉斯综合征EPI14070712.pdf

CLINICAL AND LABORATORY OBSERVATIONS Treatment of Refractory Langerhans Cell Histiocytosis (LCH) With a Combination of 2-Chlorodeoxyadenosine and Cytosine Arabinoside Nataly Apollonsky, MD* and Jeffrey M. Lipton, MD, PhDw cytosine arabinoside (Ara-C) combination chemotherapy Summary: The combination of 2-chlorodeoxyadenosine (2-CDA) demonstrated its efficacy in children with refractory LCH.8 and cytosine arabinoside (Ara-C) has been shown to be effective in Encouraging outcomes however were associated with children with refractory Langerhans cell histiocytosis (LCH). We significant toxicity. In that study, 2 patients expired after have treated 5 patients with recurrent LCH with 2-CDA/Ara-C chemotherapy and closely followed immune and hematopoietic the first course of 2-CDA and Ara-C and a third patient function. These patients display a decline in the absolute CD4, was withdrawn from the study and expired after the CD8, and natural killer cell number, decrease in the CD4/CD8 hematopoietic stem cell transplantation. All of these ratio. Septic events, including pneumocystis infection were present patients were heavily pretreated with vinblastine, steroids, after most of the treatment courses (15/21). These data suggest that etoposide, and Ara-C. The toxic deaths in this study were 2-CDA /Ara-C, should be considered in resistant and relapsed attributed not only to the toxicity of the 2-CDA and Ara-C, pediatric patients with LCH with high-risk multiorgan involve- but also to the intensive chemotherapy before enrolment. In ment. Consequent profound prolonged