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Incremental Burden of Disease in Patients Diagnosed with Pulmonary Arterial Hypertension Receiving Monotherapy and Combination Vasodilator Therapy

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Abstract

Introduction

Pulmonary arterial hypertension (PAH) is a rare, severely debilitating disease with high mortality. There are limited data available on treatment patterns and burden of disease from conditions of actual care.

Methods

This analysis assesses the burden of disease for patients with PAH treated with monotherapy and combination therapies excluding and including intravenous (IV) prostacyclin analogues (PGI2). Data were drawn from the Adelphi PAH Disease Specific Programme, a cross sectional survey of consulting patients undertaken in the US, Germany, Italy and the UK in 2010. Outcomes included demographics, clinical characteristics, health-care resource utilization, and quality of life measured by the Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR).

Results

Data were analyzed from 446 patients receiving ≥1 of 3 PAH-specific treatment classes. Physicians comprised mainly pulmonologists and cardiologists. The symptoms, functioning and quality of life scales of the CAMPHOR instrument were completed by 218, 229, and 214 patients, respectively. Although 46.2% of patients were classified as World Health Organization (WHO) functional class III or IV, only 24.4% of the population received combination therapy. Combination therapy including IV PGI2 was used in 4.7% of all patients. Patients on monotherapy had the lowest pulmonary vascular resistance values, the highest recorded 6-min walk distance and the lowest recorded levels of dyspnea. Patients on combination therapy including IV PGI2 scored worse on all three variables and had more hospitalizations than patients on less aggressive combination therapy. With increasing therapeutic regimens, the CAMPHOR scores were higher, indicating worse states of health.

Conclusions

Combination treatment and particularly the use of prostacyclins remain underused in an unselected population of PAH patients surveyed under conditions of actual care. The disease burden is substantial and increases with greater severity of disease and more aggressive treatments. This necessitates improvement in optimizing current therapy, as well as novel and innovative combination options.

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Acknowledgments

Sponsorship for this study and article processing charges were funded by Novartis Pharma AG. Editorial assistance in the preparation of this manuscript was provided by Maren White of White Quill Ltd. Support for this assistance was funded by Novartis Pharma AG.

Dr. Small and Dr Piercy are the guarantors for this article, and take responsibility for the integrity of the work as a whole.

Conflict of interest

M Small is a full-time employee of Adelphi Real World.

J Piercy is a full-time employee of Adelphi Real World.

J Pike is a full-time employee of Adelphi Real World.

A Cerulli is a full-time employee of Novartis Pharma AG.

Compliance with ethics guidelines

The analysis in this article is based on previously conducted studies, and does not involve any new studies of human or animal subjects performed by any of the authors.

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Correspondence to Mark Small.

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Small, M., Piercy, J., Pike, J. et al. Incremental Burden of Disease in Patients Diagnosed with Pulmonary Arterial Hypertension Receiving Monotherapy and Combination Vasodilator Therapy. Adv Ther 31, 168–179 (2014). https://doi.org/10.1007/s12325-014-0094-z

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  • DOI: https://doi.org/10.1007/s12325-014-0094-z

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