Stem cell transplantation: A promising approach for pediatric patients with refractory multistage langerhans cell histiocytosis

Journal of Applied Hematology

article

Ibrahim AlFawaz¹  Amal Alhejaili¹  Khawar Siddiqui¹  Mouhab Ayas¹ 

[1]Department of Pediatric Hematology/Oncology, King Faisal Specialist Hospital and Research Center

[2]College of Medicine, Taibah University

关键词: Langerhans cell histiocytosis;    pediatric;    stem cell transplantation;   

DOI  :  10.4103/joah.joah_223_20

学科分类：社会科学、人文和艺术（综合）

来源: Medknow

【 摘 要 】

BACKGROUND: Langerhans cell histiocytosis is a rare disorder leading to destruction of tissues as well as causing lesions in the body. LCH can be self-limiting to disseminated life threatening. OBJECTIVES: We wanted to review data on presentation and outcome of our treatment efforts on patients with LCH diagnosed and treated at our institution. MATERIAL AND METHODS: In this retrospective study involving 75 pediatric patients with LCH diagnosed between 1998 and 2018, we evaluated clinicopathological features, laboratory findings, treatment modalities and outcome. RESULTS: In males median age at the time of diagnosis was 2.4 years. The median follow up was 63+/- 7.9 months. The most common presenting symptom was a bone lesion-related symptom. 57.3% were above 2 years of age at presentation while 38.7% below 2 years of age. 53% of cases had multisystem disease at diagnosis. Liver found to be most common organ involved. Treatment consisted of observation, surgery or chemotherapy. Vinblastine with steroid was the most common used chemotherapy regimen. 3 patients with progressive refractory disease underwent stem cell transplantation. CONCLUSION: Overall survival was significantly lower in patients with liver involvement.

【 授权许可】

CC BY-NC-SA