Recommendations for the clinical management of patients receiving macitentan for pulmonary arterial hypertension (PAH): A Delphi consensus document

Abstract

In patients treated with macitentan (Opsumit®, Actelion Pharmaceuticals Ltd., Basel, Switzerland) for pulmonary arterial hypertension (PAH), prevention and/or effective management of treatment-related adverse events may improve adherence. However, management of these adverse events can be challenging and the base of evidence and clinical experience for macitentan is limited. In the absence of evidence, consensus recommendations from physicians experienced in using macitentan to treat PAH may benefit patients and physicians who are using macitentan. Consensus recommendations were developed by a panel of physicians experienced with macitentan and PAH using a modified Delphi process. Over three iterations, panelists developed and refined a series of statements on the use of macitentan in PAH and rated their agreement with each statement on a Likert scale. The panel of 18 physicians participated and developed a total of 118 statements on special populations, add-on therapy, drug–drug interactions, warnings and precautions, hospitalization and functional class, and adverse event management. The resulting consensus recommendations are intended to provide practical guidance on real-world issues in using macitentan to treat patients with PAH.

The clinical experience of macitentan in pulmonary hypertension in Indian cohort: 12-month follow-up

“…Furthermore, of worth noting is the good safety and tolerability observed. [ 8 ] In patients with multiple medications used for the medical management of the acquired immunodeficiency syndrome, the stability of the viral load and CD4 levels and the lack of drug–drug interactions between macitentan and the antiretrovirals used in patients with HIV-related PAH indicate a safety advantage. Neither macitentan nor its active metabolite has clinically significant inducer or inhibitory effects on cytochrome enzymes P450, and macitentan has a favorable hepatic safety profile relative to other ERAs.…”

Management of repository corticotrophin injection therapy for pulmonary sarcoidosis: a Delphi study

“…Baughman) have extensive experience organising initial panels of experts and have previous experience conducting Delphi studies that have resulted in publications that are used by physicians in clinical practice. The prior studies produced consensus recommendations for the use of oral treprostinil for pulmonary arterial hypertension [22] and recommendations on the clinical management of pulmonary arterial hypertension patients receiving macitentan [23], in addition to the recommendations on a treatment algorithm for sarcoidosis in this series [13].…”

Expert consensus on the management of adverse events and prescribing practices associated with the treatment of patients taking pirfenidone for idiopathic pulmonary fibrosis: a Delphi consensus study

“…The Delphi process has several limitations [ 25 , 26 ], lack of a standardized consensus-defining criterion being one of the drawbacks [ 28 – 30 ]. Given that the study is designed to elicit guidance when no strong evidence is available, the process itself may not be statistically rigorous, and even if consensus is reached, there is no guarantee that the consensus answer is generalizable or appropriate [ 28 , 30 , 31 ].…”

“…Via a series of 3 questionnaires, our modified Delphi study established statements based on the prescribing information of pirfenidone, clinical trial evidence, and the panelists’ own experience and knowledge. The modified Delphi procedure has been previously described [ 25 , 26 ] and key steps in the process are outlined as follows (Fig. 1 ): Based on published literature, an initial questionnaire was developed by moderators (FFR, ZS and SDN) and distributed to the panel via an online survey platform [ 4 , 8 – 10 ].…”

“…Consensus was defined when the mean rating from the panelists was ≥2.5 or ≤ − 2.5, on the Likert scale used in the final questionnaire (Fig. 2 ), with a standard deviation that did not cross zero [ 25 ]. Recommendations with a mean rating absolute value ≥4.0 were defined as strong.…”