Excellent remission rates with limited toxicity in relapsed/refractory Langerhans cell histiocytosis with pulse dexamethasone and lenalidomide in children

Uppuluri, Ramya; Ramachandrakurup, Sreejith; Subburaj, Divya; Bakane, Atish; Raj, Revathi

doi:10.1002/pbc.26199

Cited by 7 publications

(4 citation statements)

References 19 publications

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“…After the description of recurrent oncogenic mutations affecting the MAPK pathway in LCH patients, targeted therapies such as BRAF, MEK or BRAF/MEK inhibitors were reported to be useful for patients with these mutations who even were unresponsive to salvage treatments 22,31,32. However, further studies are warranted to reveal the efficacy, safety, and long term outcome in the pediatric population for targeted therapies.…”

Section: Discussionmentioning

confidence: 99%

Langerans Cell Histiocytosis: Single Center Experience of 25 Years

Tüysüz¹,

Yıldız²,

Özdemir³

et al. 2019

Mediterr J Hematol Infect Dis

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Objectives: To review a single center outcome of patients with Langerhans Cell Histiocytosis diagnosed at a referral tertiary hospital from Turkey.Methods: The files between 1989 and 2014 of 80 patients with LCH were retrospectively analyzed. Results: During the 25 year period, 80 patients were diagnosed with LCH. The median age at diagnosis was 53 months (2-180 months) and median follow-up time of patients was 10 years and 9 months (24 months-25 years). Bone was the most frequently affected organ (n:60, 75%). Initially, 43 patients (54%) had single system (SS) disease, 20 patients (25%) had multisystem (MS) disease without risk organ involvement (MS-RO-), and 17 patients (21%) had multisystem disease with risk organ involvement (MS-RO+). The overall survival (OS) rate was 91% and event-free survival (EFS) rate was 67% at 10 years. The overall survival rate was lower in patients with RO involvement (p=<0.001), lack of response to systemic chemotherapy on 12th week (p=<0.001), younger age (<2 years) at presentation (p=<0.02), and skin involvement (<0.001). Discussion: Besides the known risk factors like younger age (<2 years) at presentation, multi system risk organ involvement, and lack of response to initial chemotherapy, skin involvement was also associated with unfavorable outcome in our cohort. Evaluation of large cohorts of patients is needed to investigate this further.

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Section: Discussionmentioning

confidence: 99%

Langerans Cell Histiocytosis: Single Center Experience of 25 Years

Tüysüz¹,

Yıldız²,

Özdemir³

et al. 2019

Mediterr J Hematol Infect Dis

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“…The ND-CNS-LCH is commonly treated with intravenous immunoglobulin alone or in combination with chemotherapy regimens [ 40 , 62 , 64 , 96 ]. Moreover, retinoic acid [ 97 ], thalidomide and its analogues [ 98 – 100 ], as well as high doses of steroids [ 55 , 100 ], have been proposed for the treatment of ND-CNS-LCH. Cytotoxic-immunosuppressive approaches have included cladribine [ 101 , 102 ] and cytarabine because of good CNS penetration [ 9 , 17 , 56 ].…”

Section: Treatmentmentioning

confidence: 99%

Langerhans cell histiocytosis in children – a disease with many faces. Recent advances in pathogenesis, diagnostic examinations and treatment

Jezierska¹,

Stefanowicz²,

Romanowicz³

et al. 2018

pdia

114

143

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Langerhans cell histiocytosis is a rare clonal disease characterized by the proliferation of CD1a-positive immature dendritic cells. The purpose of this article was to present an updated review of recent advances in the pathogenesis, clinical features, imaging and treatment of this disease. The discovery of oncogenic BRAF mutations and the presence of proinflammatory cytokines and chemokines confirmed the unusual characteristics of this disease. Currently, children with organ involvement who do not have a good response to chemotherapy and have neurodegeneration or diabetes insipidus are the most problematic patients. Further research is needed to improve the results of treatment.

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“…However, patients with refractory/progressive disease in our series had adverse outcome. [10][11][12]…”

Section: Discussionmentioning

confidence: 99%

Langerhans cell histiocytosis: An enigmatic disease

Jain

Kumar

Aggarwal

et al. 2019

South Asian J Cancer

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Background: Langerhans cell histiocytosis (LCH) is a poorly understood disease with heterogeneous clinical presentation ranging from unifocal bony involvement to disseminated disease with life-threatening complications. Materials and Methods: The clinical profile, laboratory findings, treatment, and long-term outcome were retrieved from maintained medical records from January 2006 to January 2016 and were retrospectively analyzed. The extent of the disease was classified as per the LCH-III trial of “The Histiocyte Society.” The assessment and categorization of treatment response followed LCH III trial definitions. Results: A total of 28 children with LCH were diagnosed. The age ranged between 5 months and 9 years, with a mean of 3½ years. The M: F ratio was 3:1. Single system, unifocal and multifocal bone diseases were seen in nine (32.1%) and two (7.1%) cases, respectively. Disseminated disease without risk organ involvement was seen in six (21.1%), whereas disseminated disease with risk organ involvement was seen in 11 (39.3%) cases. The most common presentation was bony involvement (19 [67.8%]), out of which 16 (88.8%) had skull involvement. During follow-up, 17 (60.7%) were in complete remission though five (17.8%) of them relapsed, but achieved second remission. Two (7.1%) were lost to follow-up. Six (21.4%) had progressive disease of which four expired and two abandoned treatment. Two (10.7%) refused the initiation of treatment. Conclusion: A better understanding of the disease, early suspicion, and diagnosis can improve the outcome of patients with LCH.

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Excellent remission rates with limited toxicity in relapsed/refractory Langerhans cell histiocytosis with pulse dexamethasone and lenalidomide in children

Cited by 7 publications

References 19 publications

Langerans Cell Histiocytosis: Single Center Experience of 25 Years

Langerans Cell Histiocytosis: Single Center Experience of 25 Years

Langerhans cell histiocytosis in children – a disease with many faces. Recent advances in pathogenesis, diagnostic examinations and treatment

Langerhans cell histiocytosis: An enigmatic disease

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