Nationwide retrospective review of hematopoietic stem cell transplantation in children with refractory Langerhans cell histiocytosis

2019 ◽  
Vol 111 (1) ◽  
pp. 137-148 ◽  
Author(s):  
Kazuko Kudo ◽  
◽  
Miho Maeda ◽  
Nobuhiro Suzuki ◽  
Hirokazu Kanegane ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Retrospective Review ◽  
Langerhans Cell Histiocytosis ◽  
Langerhans Cell ◽  
Hematopoietic Stem

scholarly journals Autologous hematopoietic stem cell transplantation for efficient treatment of multisystem, high-risk, BRAF V600E-negative Langerhans cell histiocytosis

2019 ◽  
Vol 47 (9) ◽  
pp. 4522-4529
Author(s):  
Yaozhu Pan ◽  
Rui Xi ◽  
Cunbang Wang ◽  
Lei Fang ◽  
Jiaofeng Bai ◽  
...  
Keyword(s):  
Stem Cell ◽  
High Risk ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Lymph Nodes ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Langerhans Cell Histiocytosis ◽  
Braf V600e ◽  
Hematopoietic Stem

Langerhans cell histiocytosis (LCH) is a disorder caused by clonal proliferation of CD1a+/CD207+ cells and characterized by varying degrees of organ involvement. Treatment of LCH is risk adapted; patients with multisystem disease and risk-organ involvement require more intensive therapy. Optimal therapies for multisystem, high-risk LCH remain uncertain. Recently, targeted therapy using inhibitors of mutated BRAF (the gene encoding serine/threonine-protein kinase B-Raf) has proven very effective in patients with multisystem refractory LCH. Herein, we report a case of LCH with involvement of the bones, liver, and lymph nodes. Using next-generation sequencing of the patient’s pathological sample, we identified a mutation in MAP2K1 in exon 3 (c.362G>C, p.Cys121Ser) and no mutation in BRAF; thus, high-risk, multisystem LCH with MAP2K1 mutation and wild-type BRAF was diagnosed. After four chemotherapy treatments (COEP regimen), the patient received autologous hematopoietic stem cell transplantation (auto-HSCT). Complete remission was confirmed by follow-up positron emission tomography–computed tomography, which showed no lesions in liver, lymph nodes, or bones compared with the pretreatment period. To date, the patient has sustained good health for 24 months. In conclusion, auto-HSCT may be an effective treatment option for high-risk, multisystem BRAF V600E-negative LCH.

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