Background: Neurofibromatosis type 1 (NF1) is a rare multisystem genetic disorder. During the course of the disease it can be rarely complicated with pulmonary hypertension (PH) which confers a dismal prognosis.
Case presentation: We describe the case of a 57-year-old female patient with NF1 complicated by severe precapillary PH despite dual disease-specific oral combination therapy. The patient was treated with initial atrial septostomy followed by administration of high-dose subcutaneous treprostinil with a favorable medium-term clinical and hemodynamic response.
Conclusions: PH secondary to NF1 may be successfully treated with the combination of atrial septostomy and PH targeted therapy in selected patients.
Keywords: Case report; Neurofibromatosis; Pulmonary hypertension; Septostomy.