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A 36-Month Survival Analysis of Patients Beginning Oral PAH Monotherapy: An Indication for Escalation of Therapy?

M. Tankersley

Lesley D'Albini

A. N. Ozanich

Alan Whitman


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Conference: 2008 International PHA Conference and Scientific Sessions

Release Date: 06.20.2008

Presentation Type: Abstracts

Tankersley M.A., D’Albini L.D., Ozanich A.N., Whitman A.J.

Accredo Therapeutics, Memphis, TN, USA

BACKGROUND: Pulmonary Arterial Hypertension (PAH) is a progressive disease requiring comprehensive patient management to optimize outcomes.  Specialty Pharmacy Service providers are an integral component of health care teams which provide medication, monitoring and support to patients with PAH.  By analyzing treatment trends and outcomes with oral agent monotherapy, patterns may be noted and opportunities for optimal therapy management identified.

METHODSData from acohort of patients (n=821) receiving bosentan, an endothelin receptor antagonist (ETRA), who were being supported by Accredo Therapeutics over a 36 month period beginning in the last quarter of 2004 and ending in the last quarter of 2007, were retrospectively analyzed.  These patients were assessed for duration on therapy, discontinuation rates, reasons for discontinuation, whether patients were escalated from oral monotherapy to inhaled or parenteral prostacyclin therapy and survival. All survival estimates were calculated using the Kaplan-Meier method.

RESULTSThe cohort of patients on monotherapy analyzed demonstrated a 67% survival at 36 months.  Of the 191 patients who expired, only 20 (10%) were escalated to additional therapies beyond bosentan monotherapy meaning 171 (90%) expired on oral monotherapy.  Duration on service was 138% longer (16 months vs. 22 months) for those who received additional therapy vs. those who did not. 

COMMENTS AND CONCLUSIONS: Escalation of therapy, including dual or multimodal medication regimens, in patients initiated on monotherapy with an ETRA is considered standard of care by national expert treatment guidelines.  The fact that 90% (171/191) of PAH patients who expired did so without changes or additions to their therapy is concerning.   Several hypotheses can be formulated based upon this observation and warrant further investigation.  These include: a) a percentage of these patients being followed on bosentan monotherapy had limited clinical documentation to support the addition or inclusion of prostacyclin therapy(s);  b) The prescribing physicians for this subset of patients were inadequately informed on treatment options and expert opinion consensus recommendations for ongoing assessment and intensification of therapy, and/or; c) Despite expert opinion recommendation for escalation to prostacyclin therapy, patient or physician refusal occurred due to perceptions regarding the complexity or invasive nature of current prostanoid delivery.  Based on these findings, Accredo Therapeutics is assessing the development of innovative programs to further clinical management and therapy optimization of patients with pulmonary arterial hypertension.