Addition of Inhaled Treprostinil to Oral Therapy for Pulmonary Arterial Hypertension

McLaughlin, Vallerie V.; Benza, Raymond L.; Rubin, Lewis J.; Channick, Richard N.; Voswinckel, Robert; Tapson, Victor F.; Robbins, Ivan M.; Olschewski, Horst; Rubenfire, Melvyn; Seeger, Werner

doi:10.1016/j.jacc.2010.01.027

Cited by 501 publications

(425 citation statements)

References 28 publications

Supporting

Mentioning

401

Contrasting

Unclassified

Order By: Relevance

“…Nevertheless, the similarity of the findings for MID in 6MWD between Gilbert's and Mathai's work raises doubts about the clinical relevance of the change in 6MWD reported in several recently completed combination therapy trials (Fig. 3), [43][44][45][46][47][48] including those with statistically significant values for their respective primary or secondary end points.…”

Section: Candidate Surrogate Markers In Pahmentioning

confidence: 84%

“…66 However, the definition of clinical worsening has varied between trials, which makes comparison of data difficult (Table 1). 24,26,28,[40][41][42][43][44]47,55 It is also not useful to use the "need for transplant" as opposed to undergoing transplant due to the variability in listing a patient. It is also most useful for the events to be as objective as possible and adjudicated blindly and independently by committee.…”

Section: Shifting the Focus From The 6mwdmentioning

confidence: 99%

See 1 more Smart Citation

From Short‐Term Benefits to Long‐Term Outcomes: The Evolution of Clinical Trials in Pulmonary Arterial Hypertension

Chakinala

Barst

2013

Pulm. circ.

View full text Add to dashboard Cite

Over the past 2 decades, major advances in our understanding of pulmonary arterial hypertension (PAH) have led to the development of new targeted therapeutics and management strategies that have provided benefits to patients with this devastating disease. Despite such improvements, no therapies are curative, and PAH remains a progressive disease associated with high morbidity and suboptimal survival in many patients. Clinical research in PAH is currently at a crossroads. To move forward, not only are new therapies needed, but novel approaches to clinical trial design are also required. Trials should be designed to assess the longer-term benefits of investigational therapies in what has become a chronic disease. Moreover, there is a need to consider moving away from short-term trials that use markers such as the 6-minute walk distance as a measure of exercise capacity as primary end points to longer-term, event-driven trials with composite end points made up of clinically relevant measures that better reflect the ultimate goals of reducing morbidity and mortality. A shift in trial design may also be useful in overcoming some of the muted results from recent pivotal phase III studies of combination therapy by allowing the potential of these regimens to be more comprehensively assessed.

show abstract

Section: Candidate Surrogate Markers In Pahmentioning

confidence: 84%

Section: Shifting the Focus From The 6mwdmentioning

confidence: 99%

From Short‐Term Benefits to Long‐Term Outcomes: The Evolution of Clinical Trials in Pulmonary Arterial Hypertension

Chakinala

Barst

2013

Pulm. circ.

View full text Add to dashboard Cite

show abstract

“…Recently, add-on therapy with inhaled treprostinil in adult PAH patients already using either bosentan or sildenafil was shown to be effective [41], but no paediatric data are available. Finally, treprostinil for oral administration, an attractive option for children, is under development.…”

Section: Treprostinilmentioning

confidence: 99%

“…epoprostenol background therapy [72], sildenafil added to i.v. epoprostenol [73] and inhaled treprostinil added to either bosentan [41,74] or sildenafil [41] have been studied, with the results indicating additional beneficial effects for the combination.…”

Section: Combination Therapymentioning

confidence: 99%

Treatment options for paediatric pulmonary arterial hypertension

Berger¹,

Bonnet²

2010

Eur Respir Rev

View full text Add to dashboard Cite

Pulmonary arterial hypertension (PAH) is a serious, progressive condition, which can present idiopathically or secondary to conditions such as systemic sclerosis or congenital heart disease. The condition exists in both adult and paediatric forms, which possess several similar characteristics. Adult and paediatric PAH can, however, be distinguished based on underlying pathology and the presence of age-specific conditions, some of which are related to poor lung development in children. Improved knowledge of vascular biology has led to the development of several PAH-specific therapies, which have demonstrated clinical benefits in adults, including improved exercise capacity and prolonged survival. Treatment data in paediatric PAH are scarce. Although limited, the existing data indicate that current treatments for paediatric PAH are well tolerated and effective, at least in the short-and medium-term. Nevertheless, the current guidelines for clinicians, which recommend use of the adult treatment algorithm in paediatric patients, appear justified when judged according to the available evidence. However, further randomised, controlled trials are necessary to increase the evidence base for treatment of paediatric PAH, especially in relation to age-specific conditions. At present, early initiation of treatment and combination pharmacological therapy may offer the most promising courses of action to improve outcomes in paediatric PAH.

show abstract

“…dosing, we would lean towards inhaled treprostinil. We are familiar with the iloprost--treprostinil switch study by Bourge et al, demonstrating improved biomarkers and patient satisfaction on inhaled treprostinil, and believe that, while highly provocative and interesting, we cannot draw meaningful conclusions regarding comparative drug efficacy in this open label, nonrandomized study [9].…”

mentioning

confidence: 91%

Inhaled prostanoid therapy for pulmonary arterial hypertension – a promise unfulfilled?

Ramani

2012

Expert Opinion on Pharmacotherapy

View full text Add to dashboard Cite

Addition of Inhaled Treprostinil to Oral Therapy for Pulmonary Arterial Hypertension

Cited by 501 publications

References 28 publications

From Short‐Term Benefits to Long‐Term Outcomes: The Evolution of Clinical Trials in Pulmonary Arterial Hypertension

From Short‐Term Benefits to Long‐Term Outcomes: The Evolution of Clinical Trials in Pulmonary Arterial Hypertension

Treatment options for paediatric pulmonary arterial hypertension

Inhaled prostanoid therapy for pulmonary arterial hypertension – a promise unfulfilled?

Contact Info

Product

Resources

About