Association therapy with macitentan added to sildenafil in a thalassemic patient with pulmonary hypertension

Macitentan Administration for Pulmonary Hypertension Due to β-thalassemia with Multiple Organ Failure

“…In the aforementioned studies, PDE-5 inhibitors, including sildenafil and tadalafil, which enhance the nitric oxide (NO)-cyclic guanosine monophosphate biological pathway, have been frequently used because low NO bioavailability due to chronic hemolysis is considered the main cause of PH secondary to βthalassemia (6)(7)(8). Likewise, combination therapy with PDE-5 inhibitors and ERAs was also used (9,11,13), with all drugs showing improvement in pulmonary hemodynamics or subjective symptoms, including severe cases. However, because most evaluation methods employed in the aforementioned studies involved echocardiography and not RHC, further investigations are warranted to accurately assess the efficacy of SPVs in patients with PH secondary to βthalassemia, as SPVs may be a useful treatment option for this disease.…”

“…Several studies and case reports have suggested the efficacy of SPVs, including phosphodiesterase type 5 (PDE-5) inhibitors, endothelin receptor antagonists (ERAs), and prostacyclin analogs, in treating patients with PH secondary to β-thalassemia ( Table 2 ) ( 6 - 13 ). In the aforementioned studies, PDE-5 inhibitors, including sildenafil and tadalafil, which enhance the nitric oxide (NO)-cyclic guanosine monophosphate biological pathway, have been frequently used because low NO bioavailability due to chronic hemolysis is considered the main cause of PH secondary to β-thalassemia ( 6 - 8 ).…”

“…In addition, no previous reports exist on the administration of macitentan alone in patients with β-thalassemia and PH. Furthermore, only one case report described a patient with PH accompanied by β-thalassemia who received a combination therapy of macitentan and sildenafil ( 13 ). Based on this report, macitentan improved the patient's clinical condition, dyspnea, and performance in the six-minute walking test.…”