Effective treatment of malignant atrophic papulosis (Köhlmeier-Degos disease) with treprostinil--early experience.

[malignant atrophic papulosis]

Malignant atrophic papulosis (Köhlmeier-Degos disease ; MAP ) is an uncommon endotheliopathy with pathological findings similar to the vascular lesions of systemic sclerosis . These two disorders can overlap . When associated with visceral lesions , MAP has been considered almost universally and rapidly fatal . A recent report described dramatic response to treatment with eculizumab , but disease progression after initial response to therapy has occurred .We describe the clinical and pathologic findings in two patients , one with MAP and the other with MAP like lesions , who received treatment with subcutaneous treprostinil . One patient had an overlap syndrome with features of systemic lupus erythematosus (SLE ) and scleroderma and severe pulmonary hypertension . She also had very extensive MAP like cutaneous lesions . There was no evidence of central nervous system (CNS ) disease and laparoscopy revealed no visible MAP lesions on the serosa of the small bowel . The second patient had experienced life-threatening disease progression despite ongoing eculizumab therapy . During this treatment , he had developed CNS and bladder involvement with neurologic symptoms and gross hematuria .Patient one was placed on therapy with treprostinil for her pulmonary hypertension , but in the months subsequent to initiation of treatment , dramatic and complete resolution of cutaneous MAP like lesions and disabling digital pain occurred . In patient two , therapy with treprostinil was temporally associated with clearing of hematuria , resolution of CNS symptoms and improvement in MRI findings .Treprostinil may offer a second effective treatment approach to individuals with MAP or "rescue therapy " to those in whom eculizumab treatment has failed to maintain suppression of disease activity .