Early failure of eculizumab in a patient with malignant atrophic papulosis: Is it time for initial combination therapy of eculizumab and treprostinil?

Razanamahery, J.; Payet-Revest, C.; Mareschal, Adrien; Saizonou, Ines; Bonnet, Louise; Gil, H.; Humbert, S.; Magy-Bertrand, Nadine

doi:10.1111/1346-8138.15120

Cited by 7 publications

(8 citation statements)

References 5 publications

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“…It has been reported that eculizumab cannot provide sustained remission of gastrointestinal symptoms ( 32 , 33 ), but the combination of treprostinil and eculizumab is encouraging ( 33 , 34 ). The mechanism of treprostinil in this condition is unknown, possibly increasing the number of circulating endothelial cells and allowing angiogenesis ( 9 ).…”

Section: Discussionmentioning

confidence: 99%

Malignant atrophic papulosis treated with eculizumab and hirudin: a fatal case report and literature review

Yu,

Wang,

Tang

et al. 2024

Front. Cardiovasc. Med.

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BackgroundMalignant atrophic papulosis (MAP) is a rare obliterative vasculopathy whose etiology and pathophysiological mechanisms remain unknown, and the treatment is still empirical. It can involve multiple systems, especially the gastrointestinal tract and central nervous system, and has a poor prognosis.Case presentationA 20-year-old Chinese male appeared to have Widespread atrophic papules and plaques, intermittent abdominal pain, recurrent bowel perforation, and psoas abscess. The clinical diagnosis of MAP was supported by skin biopsy. He was then treated with anticoagulants, antiplatelets, glucocorticoids, and immunosuppressants and started on eculizumab and hirudin after the first surgical interventions. Despite the aggressive immunosuppression, anticoagulant, antiplatelet, humanized monoclonal antibodies, and surgery therapy, he died five months after presentation.ConclusionsMAP is an extremely rare obliterative vasculopathy manifesting as benign cutaneous involvement or potentially malignant systemic involvement. MAP patients who exhibit any abdominal symptoms should undergo laparoscopy and evaluation in time and start on eculizumab and treprostinil as soon as possible, as the combination of them is presently the most effective treatment option for gastrointestinal MAP and hopefully reduce mortality.

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Section: Discussionmentioning

confidence: 99%

Malignant atrophic papulosis treated with eculizumab and hirudin: a fatal case report and literature review

Yu,

Wang,

Tang

et al. 2024

Front. Cardiovasc. Med.

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“…Regarding treatment, there have been reports of the efficacy of eculizumab (monoclonal antibody targeted against complement C5) and treprostinil (synthetic prostaglandin agonist), though additional work in this area is greatly needed. [ 6 7 ]…”

Section: Discussionmentioning

confidence: 99%

An Elderly Female with Progressive Inflammatory Scar-Like Papules

Adelman

Spratt

Friedman

2021

Indian Journal of Dermatology

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“…Within months of initiation of treatment, those so treated have noted recurrent abdominal pain. Perforation while on long-term eculizumab monotherapy has been seen [ 37 ].…”

Section: Treatmentmentioning

confidence: 99%

Gastrointestinal Kohlmeier–Degos disease: a narrative review

Sattler

Magro

Shapiro

et al. 2022

Orphanet J Rare Dis

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Introduction Kohlmeier-Degos (K-D) disease is a rare obliterative vasculopathy that can present as a benign cutaneous form or with potentially malignant systemic involvement. The gastrointestinal tract is most frequently involved in systemic disease and mortality is often related to bowel perforations. Herein, we provide information to providers and patients regarding gastrointestinal K-D symptomology, pathology, treatment, and diagnosis, with a focus on the importance of timely diagnostic laparoscopy. We present three new cases of gastrointestinal K-D to highlight varying disease presentations and outcomes. Body Based on reviewed reports, perforation is preceded by at least one gastrointestinal symptom: abdominal pain/cramping, anorexia/weight loss, vomiting, diarrhea, nausea, gastrointestinal bleeding, obstipation, constipation, and abdominal fullness. Perforation most commonly occurs in the small intestine and often results in sepsis and death. Although underutilized, laparoscopy is the most sensitive and specific diagnostic technique, demonstrating serosal porcelain plaques similar to those on the skin and characteristic for K–D. The combination of eculizumab and treprostinil is presently the most effective treatment option for gastrointestinal K–D. The pathology of gastrointestinal K-D is characterized by an obliterative intimal arteriopathy eventuating in occlusive acellular deposits of mucin and collagen along with an extravascular pauci-cellular sclerosing process resembling scleroderma confined to the subserosal fat. C5b-9 and interferon-alpha are both expressed in all caliber of vessels in the affected intestine. While C5b-9 blockade does not prevent the intimal expansion, enhanced type I interferon signaling is likely a key determinant to intimal expansion by, causing an influx of monocytes which transdifferentiate into procollagen-producing myofibroblast-like cells. Conclusion Prompt laparoscopic evaluation is necessary in any K–D patient with an abdominal symptom to facilitate diagnosis and treatment initiation, as well as to hopefully decrease mortality. Those with gastrointestinal K–D should start on eculizumab as soon as possible, as onset of action is immediate.

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Early failure of eculizumab in a patient with malignant atrophic papulosis: Is it time for initial combination therapy of eculizumab and treprostinil?

Cited by 7 publications

References 5 publications

Malignant atrophic papulosis treated with eculizumab and hirudin: a fatal case report and literature review

Malignant atrophic papulosis treated with eculizumab and hirudin: a fatal case report and literature review

An Elderly Female with Progressive Inflammatory Scar-Like Papules

Gastrointestinal Kohlmeier–Degos disease: a narrative review

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