Successful Salvage Treosulfan-Based Megachemotherapy With Allogeneic Stem Cell Transplantation in Nonsyndromic, Therapy-Resistant Disseminated Juvenile Xanthogranuloma: A Case Report

Janeczko-Czarnecka, Małgorzata; Raciborska, Anna; Gos, Monika; Kordowska, Olga; Gajek, Kornelia; Rybka, Blanka; Ryczan‐Krawczyk, Renata; Kałwak, Krzysztof; Ussowicz, Marek

doi:10.1016/j.transproceed.2020.05.025

Cited by 4 publications

(6 citation statements)

References 28 publications

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“…experienced three allogeneic HSCT (allo‐HSCT) from a fully matched unrelated donor (MUD) because of systemic chemotherapy resistance. The patient experienced acute graft‐versus‐host disease (GvHD) and lymphoproliferative disease (LPD) caused by Epstein–Barr virus (EBV) infection and was in complete remission with full donor chimerism and without symptoms of JXG 7.5 years after the first allo‐HSCT 4 . Neska‐Matuszewska et al.…”

Section: Resultsmentioning

confidence: 99%

“…The data about HSCT for the refractory or recurrent SJXG were rare. In our review, four patients received HSCT because of unavailable surgery, therapy resistance, or disease recurrence, all remaining in remission by the end of the follow‐up 4,5,7 . However, more research on its efficacy and feasibility for refractory or recurrent SJXG is warranted.…”

Section: Discussionmentioning

confidence: 99%

“…The patient experienced acute graft-versus-host disease (GvHD) and lymphoproliferative disease (LPD) caused by Epstein-Barr virus (EBV) infection and was in complete remission with full donor chimerism and without symptoms of JXG 7.5 years after the first allo-HSCT. 4 Neska-Matuszewska et al also reported that a 3-year-old male received two allo-HSCT, and the patient experienced CNS EBV posttransplant lymphoproliferative disorder (CNS-EBV-PTLD). 5 Maeda et al reported that a patient with intracranial lesions underwent allo-HSCT because of no response to systemic chemotherapy and the patient was alive disease-free 10 years after HSCT.…”

Section: Treatment and Outcomementioning

confidence: 99%

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Systemic juvenile xanthogranuloma: A systematic review

Zou

Wei

et al. 2023

Pediatric Blood & Cancer

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Objective: To perform a systematic review to investigate the available literature regarding systemic juvenile xanthogranuloma (SJXG) and report the population characteristics, clinical manifestation, therapy, and outcome. Review methods:A search of PubMed, Embase, and Cochrane Library for all articles published between 1981 and 2022 was performed with variations and combinations of the following search terms: extracutaneous, visceral, systemic, and juvenile xanthogranuloma (JXG). Data extracted included demographics, organ involvement, treatment, outcome, and permanent sequelae.Results: A total of 103 articles encompassing 159 patients met the inclusion criteria.The median onset age was 9 months, with a male predominance (61%). The distribution of major involved organs varied by age, and younger onset age was associated with more organ involvement. The most commonly involved site was the central nervous system (CNS) (40.9%), followed by the liver (31.4%), the lung (18.9%), and the eye (18.2%). At the termination of follow-up, 93 patients (58.5%) were alive with no disease, 56 (35.2%) were alive with disease, and 10 (6.3%) were dead of disease. There was a significant difference in outcome between patients with and without spleen involvement (p = .0003), and patients with spleen involvement suffered a higher risk of death.Permanent sequelae mainly comprised CNS symptoms and ocular manifestations. Conclusions: SJXG can involve varying numbers and combinations of extracutaneoussites. There is no standard therapy for SJXG and clinicians should choose individualized therapy modalities.

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Section: Resultsmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Section: Treatment and Outcomementioning

confidence: 99%

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Systemic juvenile xanthogranuloma: A systematic review

Zou

Wei

et al. 2023

Pediatric Blood & Cancer

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“…In refractory SJXG, hematopoietic stem cell transplantation (SCT) may be another treatment option. Only a few cases are reported in the literature treated with hematopoietic SCT 11‐13 . Ito et al reported a case of SJXG with bone involvement and multiple pathologic fractures who were refractory to chemotherapy and ultimately treated with haploidentical STC 11 …”

Section: Discussionmentioning

confidence: 99%

Successful treatment of congenital systemic juvenile xanthogranulomatosis with pulmonary involvement

Ataseven

Kızmazoğlu

Öniz

2021

Pediatric Dermatology

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Juvenile xanthogranuloma (JXG) is a common form of non-Langerhans cell histiocytosis, which usually presents with spontaneously regressing skin lesions. Systemic involvement is rare and mostly seen in patients with multiple skin nodules. It can spontaneously regress, but sometimes systemic involvement can cause life-threatening symptoms and can be fatal. Herein, we report a case of congenital systemic JXG with multiple skin nodules, soft tissue and pulmonary involvement. She was successfully treated with chemotherapy according to Langerhans cell histiocytosis treatment protocol IV of the Histiocyte Society (LCH-IV).

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“…The only side effects observed were mild eczema and transient fever 24 . Hematopoietic stem cell transplantation may be another treatment option for refractory SJXG, in which the reconstruction of hematopoietic stem cells helps normalize histiocyte differentiation 25 . Ito et al 26 .…”

Section: The Treatment Modalities In Sjxgmentioning

confidence: 99%

Frequent detection of genetic aberrations reveals novel pathogenesis and treatment modalities in systemic juvenile xanthogranuloma

Xu,

Ma,

Yao

et al. 2023

Pediatric Investigation

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Frequent detection of genetic aberrations reveals novel pathogenesis and treatment modalities in systemic juvenile xanthogranulomaJuvenile xanthogranuloma (JXG), the most common form of non-Langerhans cell histiocytosis (non-LCH), is generally confined to the skin during infancy and early childhood. 1 JXG rarely involves extracutaneous tissues or systemic organs, resulting in high morbidity and mortality rates. Clonality in JXG has been verified by histopathological and genetic analyses, which have shown it to be tumorous rather than a reactive disorder of JXG. 2 However, the exact etiopathogenesis of JXG remains unclear.Diverse organs and systems can be involved in systemic JXG (SJXG). The most common extracutaneous sites include the eyes, central nervous system (CNS), liver, and lungs. Compared with cutaneous JXG, SJXG usually requires more aggressive treatment. Conventionally, the first-line treatment for SJXG is based on chemotherapy used in Langerhans cell histiocytosis (LCH), such as corticosteroids and vinblastine, with various responses. 3 Alternative regimens include methotrexate, cladribine, and cytarabine when initial chemotherapy fails.In the past decade, genomic studies have been conducted on histiocytosis, including JXG, and distinctive genetic abnormalities have been discovered (e.g., BRAFV600E mutations and ALK translocations). 4,5 These novel genetic discoveries further confirmed the tumorigenesis of JXG and highlighted the possibility of therapeutic targets in refractory or severe cases. GENETIC ABERRATIONS IN THE PATHOGENESIS OF SJXGJXG is involved in the clonal proliferation of histiocytes by human androgen receptor assay (HUMARA). 2 However, the drivers of cell proliferation in JXG remain to be elucidated. Several cases of JXG have been reported

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Successful Salvage Treosulfan-Based Megachemotherapy With Allogeneic Stem Cell Transplantation in Nonsyndromic, Therapy-Resistant Disseminated Juvenile Xanthogranuloma: A Case Report

Cited by 4 publications

References 28 publications

Systemic juvenile xanthogranuloma: A systematic review

Systemic juvenile xanthogranuloma: A systematic review

Successful treatment of congenital systemic juvenile xanthogranulomatosis with pulmonary involvement

Frequent detection of genetic aberrations reveals novel pathogenesis and treatment modalities in systemic juvenile xanthogranuloma

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