15 years journey of idiopathic pulmonary arterial hypertension with BMPR2 mutation
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초록
Pulmonary arterial hypertension (PAH) is known as one of diseases with the worst prognosis. Recently, targeted PAH drugs have been developed and approved for use; therefore, the treatment strategy and goals have changed, and the prognosis has improved over two decades. We reviewed the case of a female who showed the natural disease course of heritable PAH in treatment with the targeted PAH drugs under the Korean Health Insurance policy. At the age of 15, she visited the outpatient clinic for dyspnea on exertion that occurred 3 years ago. At that time, severe pulmonary hypertension was revealed by an echocardiography and there was no evidence of significant shunt lesion or embolism. After 4 years of loss to follow-up, her performance was WHO functional class III and she still suffered from dyspnea. The initial monotherapy using an endothelin receptor antagonist was started in 2008. After 2 years, BMPR 2 mutation was detected. Her clinical symptoms gradually worsened because of poor compliance. To escalate therapy, combination therapy was given, and finally, triple maximal therapy was maintained. The next step is to consider intravenous prostanoids. Various combinations of targeted therapy have been tried, and several trials have been confirmed that improve the prognosis. Initial upfront combination therapy and a more enthusiastic approach make good a better prognosis. In this area, active support of the government insurance policy is indispensable in Korea.
Abstract
Pulmonary arterial hypertension (PAH) is known as one of diseases with the worst prognosis. Recently, targeted PAH drugs have been developed and approved for use; therefore, the treatment strategy and goals have changed, and the prognosis has improved over two decades. We reviewed the case of a female who showed the natural disease course of heritable PAH in treatment with the targeted PAH drugs under the Korean Health Insurance policy. At the age of 15, she visited the outpatient clinic for dyspnea on exertion that occurred 3 years ago. At that time, severe pulmonary hypertension was revealed by an echocardiography and there was no evidence of significant shunt lesion or embolism. After 4 years of loss to follow-up, her performance was WHO functional class III and she still suffered from dyspnea. The initial monotherapy using an endothelin receptor antagonist was started in 2008. After 2 years, BMPR 2 mutation was detected. Her clinical symptoms gradually worsened because of poor compliance. To escalate therapy, combination therapy was given, and finally, triple maximal therapy was maintained. The next step is to consider intravenous prostanoids. Various combinations of targeted therapy have been tried, and several trials have been confirmed that improve the prognosis. Initial upfront combination therapy and a more enthusiastic approach make good a better prognosis. In this area, active support of the government insurance policy is indispensable in Korea.
참고문헌 (19)
- D’Alonzo GE, "Survival in patients with primary pulmonary hypertension. Results from a national prospective registry", Ann Intern Med, 115(5), pp.343~349 (1991)
- 박예민, "Functional Class and Targeted Therapy Are Related to the Survival in Patients with Pulmonary Arterial Hypertension", Yonsei Medical Journal, 55(6), pp.1526~1532 (2014), 연세대학교의과대학[DOI]
- 정욱진, "Baseline Characteristics of the Korean Registry of Pulmonary Arterial Hypertension", Journal of Korean Medical Science, 30(10), pp.1429~1438 (2015), 대한의학회
- Barst RJ, "A comparison of continuous intravenous epoprostenol(prostacyclin)with conventional therapy for primary pulmonary hypertension", N Engl J Med, 334(5), pp.296~301 (1996)
- Simonneau G, "Continuous subcutaneous infusion of treprostinil, a prostacyclin analogue, in patients with pulmonary arterial hypertension : a double-blind, randomized, placebo-controlled trial", Am J Respir Crit Care Med, 165(6), pp.800~804 (2002)
- Olschewski H, "Inhaled iloprost for severe pulmonary hypertension", N Engl J Med, 347(5), pp.322~329 (2002)
- Barst RJ, "Beraprost therapy for pulmonary arterial hypertension", J Am Coll Cardiol, 41(12), pp.2119~2125 (2003)
- Channick RN, "Effects of the dual endothelin-receptor antagonist bosentan in patients with pulmonary hypertension : a randomised placebo-controlled study", Lancet, 358(9288), pp.1119~1123 (2001)
- Rubin LJ, "Bosentan therapy for pulmonary arterial hypertension", N Engl J Med, 346(12), pp.896~903 (2002)
- Humbert M, "Combination of bosentan with epoprostenol in pulmonary arterial hypertension : BREATHE-2", Eur Respir J, 24(3), pp.353~359 (2004)
- Sitbon O, "Initial dual oral combination therapy in pulmonary arterial hypertension", Eur Respir J, 47(6), pp.1727~1736 (2016)
- van de Veerdonk MC, "Upfront combination therapy reduces right ventricular volumes in pulmonary arterial hypertension", Eur Respir J, 49(6), pp.1700007~ (2017)
- Fox BD, "Combination therapy for pulmonary arterial hypertension : a systematic review and metaanalysis", Can J Cardiol, 32(12), pp.1520~1530 (2016)
- Tamura Y, "Effectiveness and outcome of pulmonary arterial hypertension-specific therapy in Japanese patients with pulmonary arterial hypertension", Circ J, 82(1), pp.275~282 (2017)
- Galie N, "2015 ESC", ERS guidelines for the diagnosis and treatment of pulmonary hypertension : the joint task force for the diagnosis and treatment of pulmonary hypertension of the European Society of Cardiology(ESC)and the European Respiratory Society(ERS) : endorsed by : Association for European Paediatric and Congenital Cardiology(AEPC), International Society for Heart and Lung Transplantation(ISHLT), 2015, pp.46(4), 903~975
- Tokunaga N, "Rapid and high-dose titration of epoprostenol improves pulmonary hemodynamics and clinical outcomes in patients with idiopathic and heritable pulmonary arterial hypertension", J Cardiol, 68(6), pp.542~547 (2016)
- Matsubara H, "Treatment of idiopathic", hereditary pulmonary arterial hypertension, 2014, pp.64(4), 243~249
- Ogawa A, "Long-term patient survival with idiopathic", heritable pulmonary arterial hypertension treated at a single center in Japan, 2014, pp.118(2), 414~419
- Grignola JC, "Up-front combination therapy in pulmonary arterial hypertension : from clinical trials to ‘real world’ observational studies", Int J Cardiol, 173(3), pp.349~350 (2014)