Langerhans Cell Histiocytosis of the Gastrointestinal Tract: Evidence for Risk Organ Status

Yoon, Hoi Soo; Lee, Jae Hee; Michlitsch, Jennifer; Garcia-Carega, Manuel; Jeng, Michael

doi:10.1016/j.jpeds.2019.05.003

Cited by 11 publications

(13 citation statements)

References 33 publications

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“…A recent small retrospective study found an unfavorable impact of GI-LCH on disease prognosis that appeared to be independent of risk organ involvement. 10 Our analysis validates the unfavorable impact of GI-LCH on disease outcome; however, we were able to find an additive negative prognostic effect in patients with risk organ involvement only.…”

Section: Discussionsupporting

confidence: 81%

“…24 Documented manifestations encompass vomiting, abdominal pain, failure to thrive, protein-losing enteropathy, anasarca, bloody or nonbloody diarrhea, hematochezia, and intestinal perforation. 9,10,12,25 A postmortem study of 12 deceased children with LCH suggested that clinically silent gut involvement may be more frequent than manifested GI-LCH. 24 As shown by the few available case series and the numerous case reports and literature reviews, GI-LCH typically manifests in the setting of MS-LCH and affects predominantly infants and toddlers.…”

Section: Discussionmentioning

confidence: 99%

“…GI-LCH in children is almost invariably associated with skin involvement. 9,10,12 The preceding or concomitant characteristic skin rash provides a clue as to the underlying LCH.…”

Section: Discussionmentioning

confidence: 99%

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Additive Prognostic Impact of Gastrointestinal Involvement in Severe Multisystem Langerhans Cell Histiocytosis

Minkov

Pötschger

Thacker

et al. 2021

The Journal of Pediatrics

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for the LCH Study Group of the Histiocyte Society Objective To evaluate the prognostic impact of gastrointestinal involvement on the survival of children with Langerhans cell histiocytosis (GI-LCH) registered with the international clinical trials of the Histiocyte Society. Study designThis was a retrospective analysis of 2414 pediatric patients registered onto the consecutive trials DAL-HX 83, DAL-HX 90, LCH-I, LCH-II, and LCH-III.Results Among the 1289 patients with single-system LCH, there was no single case confined to the GI tract; 114 of 1125 (10%) patients with multisystem LCH (MS-LCH) had GI-LCH at initial presentation. GI-LCH was significantly more common in children aged <2 years at diagnosis (13% vs 6% in those aged >2 years; P < .001) and in those with risk organ involvement (15% vs 6% in those without risk organ involvement; P < .001). The 5-year overall survival (OS) in patients without risk organ involvement was excellent irrespective of GI disease (98% vs 97% in patients with GI-LCH; P = .789). In patients with risk organ involvement, the 5-year OS was 51% in 70 patients with GI-LCH vs 72% in 394 patients without GI-LCH (P < .001).Conclusions GI-LCH has an additive unfavorable prognostic impact in children with MS-LCH and risk organ involvement. The emerding need for more intensive or alternative treatments mandates prospective evaluation.

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Section: Discussionsupporting

confidence: 81%

Section: Discussionmentioning

confidence: 99%

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Additive Prognostic Impact of Gastrointestinal Involvement in Severe Multisystem Langerhans Cell Histiocytosis

Minkov

Pötschger

Thacker

et al. 2021

The Journal of Pediatrics

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“…It usually occurs in the setting of a multisystem LCH, and depending of the affected gut segment, clinically presents with vomiting, abdominal pain, protein-losing enteropathy, bloody and non-bloody diarrhea, malabsorption, and failure to thrive. The prognostic value of gut involvement remains controversial but currently published paper suggests unfavorable impact on survival [30].…”

Section: Gastrointestinal Tractmentioning

confidence: 99%

Childhood Langerhans Cell Histiocytosis: Epidemiology, Clinical Presentations, Prognostic Factors, and Therapeutic Approaches

Sterlich¹,

Minkov²

2021

Rare Diseases - Diagnostic and Therapeutic Odyssey

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Childhood LCH is a rare disease, affecting 4–9 per 1,000,000 children below the age of 15 years. It is driven by somatic mutations in the MAPK pathway, arising in myeloid marrow progenitors. Both genders are affected by a slight male preponderance. The clinical spectrum of LCH varies from a single lesion affecting one organ system to severe multisystem disease with dysfunction of vital organs. Likewise, variable and unpredictable is its course, spanning from self-limiting course to progression with lethal outcome. Recognized unfavorable prognostic factors are the involvement of hematopoiesis, liver, and spleen, as well as non-response to systemic treatment. Recent studies suggest that patients carrying the BRAFV600E mutation may have a more severe clinical phenotype and less favorable prognosis. The combination of prednisolone and vinblastine is the standard first-line treatment for disseminated disease. Second-line options used in clinical practice are not well evidenced. Inhibitors of the MAPK pathway are a promising alternative option.

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“…children is rare but is associated with a poor prognosis, namely MS disease with high-risk organ involvement, resistance to conventional therapy, and high-risk of death [2,10]. Associated symptoms typically include vomiting, abdominal pain, diarrhea and bloody stools.…”

Section: Q1mentioning

confidence: 99%

Effective rescue treatment with vemurafenib of an infant with high-risk Langerhans cell histiocytosis

Gerbaux

Diallo

Dedeken

et al. 2020

Annales de Dermatologie et de Vénéréologie

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Langerhans Cell Histiocytosis of the Gastrointestinal Tract: Evidence for Risk Organ Status

Cited by 11 publications

References 33 publications

Additive Prognostic Impact of Gastrointestinal Involvement in Severe Multisystem Langerhans Cell Histiocytosis

Additive Prognostic Impact of Gastrointestinal Involvement in Severe Multisystem Langerhans Cell Histiocytosis

Childhood Langerhans Cell Histiocytosis: Epidemiology, Clinical Presentations, Prognostic Factors, and Therapeutic Approaches

Effective rescue treatment with vemurafenib of an infant with high-risk Langerhans cell histiocytosis

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