Characteristics and Treatment Outcomes of Pediatric Langerhans Cell Histiocytosis with Thymic Involvement - 06/05/22

Doi : 10.1016/j.jpeds.2022.01.007

Ja-Feng Yao, MD ^1,^2,^3,⁴, Dong Wang, MD ^1,^2,^3,⁴, Hong-Hao Ma, MD ^1,^2,^3,⁴, Hong-Yun Lian, MD ^1,^2,^3,⁴, Li Zhang, MD ^1,^2,^3,⁴, Tian-You Wang, MD ^1,^2,^3,⁴, Zhi-Gang Li, PhD ^2,^3,^4,⁵, Jin Jiang, MD ^1,^2,^3,⁴, Lei Cui, PhD ^2,^3,^4,^5,^⁎^{^{∗
Contributed equally.}} , Rui Zhang, MD ^1,^2,^3,^4,^{^{∗
Contributed equally.}}
¹ Hematology Center, Beijing Children’s Hospital, Capital Medical University, National Center for Children’s Health, Beijing, China
² Beijing Key Laboratory of Pediatric Hematology Oncology, Beijing Children’s Hospital, Capital Medical University, National Center for Children’s Health, Beijing, China
³ National Key Discipline of Pediatrics, Capital Medical University, Beijing, China
⁴ Key Laboratory of Major Diseases in Children, Ministry of Education, Beijing, China
⁵ Hematologic Diseases Laboratory, Beijing Pediatric Research Institute, Beijing Children’s Hospital, Capital Medical University, National Center for Children’s Health, Beijing, China

^∗Reprint requests: Lei Cui, PhD, Hematologic Diseases Laboratory, Beijing Pediatric Research Institute, Beijing Children’s Hospital, Capital Medical University, National Center for Children’s Health, Nanlishi Rd, No. 56, Xicheng District, Beijing 100045, ChinaHematologic Diseases LaboratoryBeijing Pediatric Research InstituteBeijing Children’s HospitalCapital Medical UniversityNational Center for Children’s HealthNanlishi RdNo. 56Xicheng DistrictBeijing100045China

Abstract

Objective

To evaluate the characteristics and treatment outcomes of patients with pediatric Langerhans cell histiocytosis (LCH) with thymic involvement.

Study design

We retrospectively described the clinical, biological, and imaging characteristics of a series of 19 patients with pediatric LCH with thymic involvement in our center between September 2016 and December 2019. We further analyzed the treatment response and outcomes of patients treated with chemotherapy or targeted therapy.

Results

Thymic involvement was found in 4.4% of a 433-consecutive pediatric LCH cohort; all LCH–thymic involvement presented with multisystem disease. Patients with thymic involvement were typically younger, harboring more lung and thyroid involvement and less bone involvement than those without thymic involvement. Most patients with thymic involvement had alteration of immunocompetence with decreased numbers of T-lymphocyte subsets and immunoglobulin G levels. Overall, 47.1% of patients demonstrated a response after 6 weeks of induction therapy, and 92.3% of the patients who did not respond to the first-line treatment had resolution of thymus after the second-line and/or targeted therapy. The progression/relapse rate showed no difference between patients who shifted to second-line therapy and those to dabrafenib (33.3% vs 25%, P = 1.000). The survival for patients with thymic involvement did not differ from those without thymic involvement. More patients treated with second-line chemotherapy had severe adverse events than those given dabrafenib (88.9% vs 0, P < .001).

Conclusions

Thymic involvement was observed rarely in LCH and had specific clinical characteristics. Chemotherapy could resolve most thymic lesions, and BRAF inhibitors might provide a promising treatment option with less toxicity for infants with BRAF-V600E mutation.

Trial registration

www.chictr.org.cn, identifier: ChiCTR2000030457 (BCH-LCH 2014 study); ChiCTR2000032844 (dabrafenib study).

Le texte complet de cet article est disponible en PDF.

Keywords : LCH, thymic involvement, chemotherapy, dabrafenib, treatment outcome

Abbreviations : AE, BRAF, CD, CT, IgG, LCH

Plan

Characteristics of Patients with LCH and Thymic Involvement

Clinical Manifestation and Biologic Measures

Imaging Presentation of Thymus

Treatment Response and Survival

Discussion

Supported by the National Natural Science Foundation of China, China (No. 82070202 [to R.Z.] and No. 82141119 [to Z.-G.L.]), the Capital’s Funds for Health Improvement and Research (No. 2020-2-2093 [to R.Z.]), the Special Fund of the Pediatric Medical Coordinated Development Center of Beijing Hospitals Authority (No. XTZD20180201 [to T.-Y.W.]), and Funding for Reform and Development of Beijing Municipal Health Commission (to Z.-G.L.). The authors declare no conflicts of interest.

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Characteristics and Treatment Outcomes of Pediatric Langerhans Cell Histiocytosis with Thymic Involvement - 06/05/22

Abstract

Objective

Study design

Results

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Trial registration

Plan

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