What are therapeutic alternatives to inhaled epoprostenol for ARDS/hypoxia and PAH during shortage?

Comment by InpharmD Researcher

Inhaled epoprostenol has been compared to inhaled iloprost, trepostinil, and commonly, inhaled nitric oxide. For pulmonary arterial hypertension, overall data suggests comparable efficacy and safety between the agents. Similarly, for ARDS, efficacy and safety was also comparable between epoprostenol and inhaled nitric oxide. Notably, selection of agents may be greatly influenced by availability and cost.

Background

A 2015 review discussed the role of inhaled prostacyclin as a treatment for ARDS. Overall, there appears to be a lack of robust evidence on selective pulmonary vasodilators (SPVs), a class that includes inhaled prostacyclins, and their use in ARDS management. A referenced 1996 prospective crossover trial (N=8) compared iEPO and iNO in mechanically ventilated ARDS patients with pulmonary hypertension (PAP ≥30 mm Hg), using escalating doses of each agent. Both reduced PAP dose-dependently; iEPO lowered mean PAP from 35.1 to 29.6 mm Hg and PVR without systemic effects, while improving PaO₂/FiO₂ at 10 and 25 ng/kg/min. iNO produced more consistent oxygenation gains (PaO₂ from 116 ± 47 to 167 ± 86 mm Hg at 8 ppm) and reduced shunt, though it had limited PVR impact. Individual responses varied, with iEPO benefiting some iNO non-responders. Another referenced 1996 randomized crossover study (N=16) in severe ARDS showed both agents improved PaO₂/FiO₂ (by 29 mm Hg with iNO, 21 mm Hg with iEPO; p <0.01) and reduced shunt (~33% to ~26%; p <0.05). PAP decreased more with iEPO (–3.1 mm Hg vs. –1.8 mm Hg; p <0.05), without affecting systemic hemodynamics. No rebound hypoxemia was reported. [1], [2], [3]

Regarding safety, iEPO in general has not been associated with cytotoxic effects compared to iNO. However, iEPO may worsen ventilation-perfusion (V/Q) mismatch, cause hypotension, inhibit platelet aggregation, and induce tachycardia. These effects may be minimized or avoided due to iEPO’s pulmonary selectivity and mode of administration. Hypotension, for instance, is more common in patients receiving intravenous (IV) epoprostenol. Overall, while iEPO may improve certain parameters in ARDS patients, their benefits have not been consistently substantiated. [1]

A 2022 multicenter, retrospective observational analysis evaluated comparative effectiveness between iNO and iEPO in patients with acute respiratory failure (ARF) requiring invasive mechanical ventilation (IMV). Utilizing the Premier Healthcare Database from 2016 to 2020, the investigators identified 11,200 adult patients with ARF or ARDS who were treated with either iEPO or iNO after the initiation of IMV. To emulate a cluster-randomized trial and reduce confounding by indication, the primary analysis was restricted to a subset of 3,478 patients admitted to hospitals that exclusively used one of the two therapies, 1,812 patients at iEPO-only hospitals and 1,666 at iNO-only hospitals. The primary endpoint was successful extubation within 28 days, defined rigorously based on cessation of IMV billing codes prior to death or discharge. Results demonstrated no statistically significant difference in the likelihood of successful extubation between patients receiving iNO versus iEpo (adjusted subdistribution hazard ratio [SHR] for iNO, 0.97; 95% confidence interval [CI], 0.80–1.18). Similarly, adjusted analyses revealed no differences in rates of death or hospice discharge (aOR, 0.99; 95% CI, 0.77–1.29), renal replacement therapy (RRT) initiation (adjusted SHR, 0.69; 95% CI, 0.44–1.08), or median PaO2:FIO2 ratio improvement one day post-initiation (adjusted median difference for iNO vs iEpo, –6 mm Hg; 95% CI, –21 to 9 mm Hg). Inhaled vasodilator-specific costs were significantly higher for iNO (adjusted median cost difference, $3,255; 95% CI, $1,568–$4,942), but this disparity did not translate into a statistically significant difference in total hospitalization costs. Among hospitals in the cohort, 34.3% exclusively used iNO, 38.9% exclusively used iEPO, and only 26.7% employed both agents, with 94.7% of the variation in initial vasodilator use explained by hospital site alone. These findings collectively underscore that in real-world practice, clinical outcomes associated with iNO and iEPO appear comparable when accounting for institutional-level treatment allocation. [4]

A 2020 systematic review and meta-analysis compared the efficacy of inhaled nitric oxide (NO) with aerosolized prostacyclin and its analogues in managing pulmonary hypertension (PH) post-cardiac or pulmonary surgery. This meta-analysis included data from seven randomized controlled trials and prospective studies conducted before December 2019, encompassing a total of 195 patients. The studies primarily assessed changes in mean pulmonary arterial pressure (MPAP) and pulmonary vascular resistance (PVR). Patients were divided into two groups based on the intervention received—either inhaled NO or inhaled prostacyclin (or its analogues). The analysis aimed to determine if these treatments offered comparable outcomes for patients experiencing PH in the perioperative or postoperative setting. The results from these studies demonstrated no significant difference in the improvement of MPAP or PVR between the groups treated with inhaled NO and those treated with inhaled prostacyclin or its analogues. Specifically, the pooled difference in mean change for MPAP was 0.10 with a 95% confidence interval ranging from -3.98 to 3.78, while the pooled standardized difference in mean change for PVR was 0.27 with a 95% confidence interval of -0.60 to 0.05. Additionally, secondary outcomes such as heart rate and cardiac output also showed no statistically significant differences between the two treatment modalities. These findings suggest that inhaled prostacyclin and its analogues can be considered viable alternatives to inhaled NO for managing PH following surgical interventions. [5]

A 2020 single-center, retrospective cohort analysis conducted at a 755-bed academic medical center examined the comparative effectiveness and cost implications of fixed-dose iEPO versus iNO for adult patients with moderate-to-severe ARDS. The investigation reviewed data from 239 mechanically ventilated patients between January 2014 and October 2018 who received either iNO or a fixed-dose regimen of iEPO. Patients receiving ECMO or treated for pulmonary embolism (PE) were excluded to isolate a homogeneous ARDS population. The fixed-dose iEPO protocol utilized a continuous nebulization technique with a vibrating mesh system, consistently delivering epoprostenol without adjusting for patient weight. The primary endpoint was the change in PaO2:FIO2 at 4 hours post-initiation. The results showed the mean increase in PaO2:FIO2 at 4 hours was statistically comparable between iEPO (31.4 ± 54.6 mm Hg) and iNO (32.4 ± 42.7 mm Hg; p = 0.88), with responder rates, defined as PaO2:FIO2 improvement of ≥10%, also similar (64.7% vs. 66.0%, p = 0.84). Improvements in oxygenation were sustained up to 72 hours with both agents. Notable findings included significantly lower mean FIO2 and tidal volumes within the first 24 hours in the iEPO group. Additionally, iEPO was associated with more mechanical ventilation-free days (p = 0.003), although hospital and ICU lengths of stay and mortality rates were similar between groups. Total drug acquisition costs were markedly lower with iEPO, translating to an estimated annual cost-savings exceeding USD 1 million compared to iNO. This investigation demonstrated that a fixed-dose iEPO strategy offers comparable clinical efficacy to iNO while significantly reducing financial burden in critically ill ARDS patients. [6]

References: [1] Searcy RJ, Morales JR, Ferreira JA, Johnson DW. The role of inhaled prostacyclin in treating acute respiratory distress syndrome. Ther Adv Respir Dis. 2015;9(6):302-312. doi:10.1177/1753465815599345
[2] Zwissler B, Kemming G, Habler O, et al. Inhaled prostacyclin (PGI2) versus inhaled nitric oxide in adult respiratory distress syndrome. Am J Respir Crit Care Med. 1996;154(6 Pt 1):1671-1677. doi:10.1164/ajrccm.154.6.8970353
[3] Walmrath D, Schneider T, Schermuly R, Olschewski H, Grimminger F, Seeger W. Direct comparison of inhaled nitric oxide and aerosolized prostacyclin in acute respiratory distress syndrome. Am J Respir Crit Care Med. 1996;153(3):991-996. doi:10.1164/ajrccm.153.3.8630585
[4] Bosch NA, Law AC, Vail EA, et al. Inhaled Nitric Oxide vs Epoprostenol During Acute Respiratory Failure: An Observational Target Trial Emulation. Chest. 2022;162(6):1287-1296. doi:10.1016/j.chest.2022.08.001
[5] Chen SH, Chen LK, Teng TH, Chou WH. Comparison of inhaled nitric oxide with aerosolized prostacyclin or analogues for the postoperative management of pulmonary hypertension: a systematic review and meta-analysis. Ann Med. 2020;52(3-4):120-130. doi:10.1080/07853890.2020.1746826
[6] Buckley MS, Agarwal SK, Garcia-Orr R, Saggar R, MacLaren R. Comparison of Fixed-Dose Inhaled Epoprostenol and Inhaled Nitric Oxide for Acute Respiratory Distress Syndrome in Critically Ill Adults. J Intensive Care Med. 2021;36(4):466-476. doi:10.1177/0885066620906800
Literature Review

A search of the published medical literature revealed 7 studies investigating the researchable question:

What are therapeutic alternatives to inhaled epoprostenol for ARDS/hypoxia and PAH during shortage?

Level of evidence

B - One high-quality study or multiple studies with limitations  Read more→



Please see Tables 1-7 for your response.


Inhaled Iloprost Versus Epoprostenol in Heart Transplant Recipients
Design

Retrospective cohort study

N= 79

Objective To evaluate the clinical practice of using inhaled iloprost compared to continuous inhaled epoprostenol following orthotopic heart transplantation, focusing on the duration of mechanical ventilation and ICU stay
Study Groups

Iloprost (n= 40)

Epoprostenol (n= 39)

Inclusion Criteria Consecutive patients undergoing orthotopic heart transplantation between July 1, 2003, and August 8, 2008, who received either inhaled epoprostenol or iloprost
Exclusion Criteria Patients who received both epoprostenol and iloprost
Methods

Retrospective chart review of 80 consecutive heart transplant patients. Epoprostenol was used from July 1, 2003, to March 13, 2006, and iloprost from March 14, 2006, to August 8, 2008.

Inhaled epoprostenol was administerd at a  starting dose of 10,000 ng/mL for patients weighing 80 kg
and 20,000 ng/mL for patients weighing 80 kg, delivered via syringe pump using a MiniHEART nebulizer at a constant flow of 8 mL/h, equivalent to 80,000 ng/h for subjects weighing 80 kg.

The recommended starting dose for inhaled iloprost was 10 g inhaled over 15 min and re-dosed every 90 –120 min with hold parameters if mean pulmonary arterial pressure was below target value ( 30 mm Hg). Iloprost was delivered as aerosolized administration via a special membrane-based nebulizer. 

Duration July 1, 2003, to August 8, 2008
Outcome Measures

Primary: Duration of mechanical ventilation

Secondary: Hemodynamic values, length of ICU and hospital stay

Baseline Characteristics   Iloprost (n= 40) Epoprostenol (n= 39)
Age, years 44.8 ± 15.2 51.8 ± 12.9
Male 80% 100%
Body mass index, kg/m2 26.4 ± 6.0 28.6 ± 5.7
Cardiac index, preoperative 2.4 ± 0.6 3.2 ± 0.9
Results   Iloprost (n= 40) Epoprostenol (n= 39) p-value
Duration of mechanical ventilation (log scale), days 0.36 ± 0.20 1.00 ± 0.22 0.033
Mean arterial pressure, postoperative, mm Hg 75.0 ± 5.2 69.6 ± 3.7 0.007
Adverse Events No significant differences in safety endpoints or length of hospital stay between groups
Study Author Conclusions Use of inhaled iloprost was associated with shorter duration of mechanical ventilation compared to inhaled epoprostenol, without safety concerns. Iloprost may offer clinically meaningful advantages over continuous epoprostenol.
Critique The study's retrospective design limits the ability to control for all variables, and the lack of randomization may introduce bias. However, the use of concurrent controls helps mitigate temporal trends. The study provides valuable insights into the potential benefits of iloprost in reducing mechanical ventilation duration.
References:
[1] [1] Enomoto TM, Treggiari MM, Yanez ND, Merkel MJ. Inhaled Iloprost Versus Epoprostenol in Heart Transplant Recipients. Respir Care. 2019;64(7):743-751. doi:10.4187/respcare.06426

Inhaled Treprostinil in Pulmonary Hypertension Due to Interstitial Lung Disease
Design

Multicenter, randomized, double-blind, placebo-controlled trial

N= 326

Objective To evaluate the safety and efficacy of inhaled treprostinil in patients with pulmonary hypertension due to interstitial lung disease
Study Groups

Inhaled treprostinil (n= 163)

Placebo (n= 163)

Inclusion Criteria Patients 18 years or older with interstitial lung disease diagnosed by CT, confirmed group 3 pulmonary hypertension by right heart catheterization, pulmonary vascular resistance >3 Wood units, pulmonary capillary wedge pressure ≤15 mm Hg, mean pulmonary arterial pressure ≥25 mm Hg, baseline forced vital capacity <70% for connective tissue disease, able to walk at least 100 m during a 6-minute walk test, stable dose of lung disease treatment for at least 30 days
Exclusion Criteria Receiving approved therapy for pulmonary arterial hypertension within 60 days before randomization
Methods Patients were randomly assigned to receive inhaled treprostinil or placebo using an ultrasonic, pulsed-delivery nebulizer. Treprostinil was administered at 6 μg per breath, up to 12 breaths four times daily. 
Duration 16 weeks
Outcome Measures

Primary: Change in peak 6-minute walk distance from baseline to week 16

Secondary: Change in NT-proBNP level, time to clinical worsening, change in 6-minute walk distance at peak plasma treprostinil level at week 12, change in 6-minute walk distance at trough treprostinil level at week 15

Baseline Characteristics   Inhaled Treprostinil (N=163) Placebo (N=163) All Patients (N=326)
Female sex 85 (52.1%) 68 (41.7%) 153 (46.9%)
Mean age at randomization (range) - yr 65.6 (26–90) 67.4 (36–85) 66.5 (26–90)
Idiopathic interstitial pneumonia 65 (39.9%) 81 (49.7%) 146 (44.8%)
Use of supplemental oxygen 119 (73.0%) 114 (69.9%) 233 (71.5%)
Results   Inhaled Treprostinil (N=163) Placebo (N=163) Treatment Effect (95% CI) P Value
Change in peak 6-minute walk distance from baseline to wk 16 - m 21.08±5.12 −10.04±5.12 31.12±7.25 (16.85 to 45.39) <0.001
Change in plasma concentration of NT-proBNP from baseline to wk 16 - pg/ml −396.35±1904.90 1453.95±7296.20 0.58 (0.47 to 0.72) <0.001
Occurrence of clinical worsening - no. (%) 37 (22.7) 54 (33.1) 0.61 (0.4 to 0.92) 0.04
Adverse Events The most frequently reported adverse events were cough, headache, dyspnea, dizziness, nausea, fatigue, and diarrhea. Serious adverse events occurred in 23.3% of the patients who received inhaled treprostinil and in 25.8% of those who received placebo
Study Author Conclusions Inhaled treprostinil improved exercise capacity in patients with pulmonary hypertension due to interstitial lung disease, as shown by the 6-minute walk test, and was associated with a lower risk of clinical worsening compared to placebo.
Critique The study demonstrated significant improvements in exercise capacity and reduced clinical worsening with inhaled treprostinil. However, the trial's short duration and the lack of independent adjudication for clinical worsening events are limitations. The treatment effect size on the 6-minute walk distance was within the range of the minimum clinically important difference, which may limit the perceived clinical significance.
References:
[1] [1] Waxman A, Restrepo-Jaramillo R, Thenappan T, et al. Inhaled Treprostinil in Pulmonary Hypertension Due to Interstitial Lung Disease. N Engl J Med. 2021;384(4):325-334. doi:10.1056/NEJMoa2008470

 

A Comparison of Inhaled Nitric Oxide Versus Inhaled Epoprostenol for Acute Pulmonary Hypertension Following Cardiac Surgery
Design Single-center, retrospective, observational, cohort study N= 98
Objective To compare the relative efficacy, safety, and cost of iNO versus iEPO in patients with acute PHT following cardiac surgery
Study Groups

iNO (n= 49)

iEPO (n= 49)

Inclusion Criteria Adult patients ≥18 years old with a pulmonary artery catheter, who received iEPO in the cardiothoracic ICU between December 2012 and December 2013, compared to a historical cohort who received iNO between July 2009 and March 2012
Exclusion Criteria Patients who received both iNO and iEPO during the same encounter, had an intra-aortic balloon pump or ventricular assist device, did not have a pulmonary artery catheter, or were missing pertinent study data
Methods Patients were identified from dispensing records. iNO was limited to an initial dose of 40 ppm. iEPO was dosed at a concentration of 20,000 ng/mL, administered at 12 mL/h in the ICU and 8 mL/h in the operating room. Weaning of iNO or iEPO was at the discretion of the attending physician. Data were collected from electronic medical records.
Duration December 2012 to December 2013 for iEPO group; July 2009 to March 2012 for iNO group
Outcome Measures

Primary: Reduction of mPAP to < 30 mm Hg within 6 hours of ICU arrival

Secondary: ICU and hospital length of stay, duration of mechanical ventilation, bleeding complications, hypotension, in-hospital mortality, cost

Baseline Characteristics   iNO (n = 49) iEPO (n = 49) P Value
Age (years), mean ± SD 63 ± 16 62 ± 16 0.56
Male, n (%) 27 (55) 28 (57) 1.00
Weight (kg), mean ± SD 83.5 ± 22.8 83.8 ± 20.5 0.94
Cardiopulmonary bypass time (minutes), mean ± SD 202 ± 85.5 205 ± 88.8 0.98
Type of surgery - CABG 5 (10) 13 (27) 0.07
Type of surgery - Isolated valve 28 (57) 31 (63) 0.68
Type of surgery - CABG/valve 7 (14) 1 (2) 0.06
Type of surgery - Heart transplant 5 (10) 3 (6) 0.71
Vasopressors, n (%) 42 (86) 40 (82) 0.78
Inotropes, n (%) 47 (96) 41 (84) 0.09
Milrinone, n (%) 38 (77) 34 (69) 0.49
Results   iNO (n = 49) iEPO (n = 49) P Value
Median duration of treatment, hours [IQR] 22 [15.4-69] 18.5 [11.5-44] 0.20
Patients w/ normalization of mPAP within 6 hours, n (%) 33 (67) 35 (71) 0.83
Median ICU LOS, days [IQR] 6 [4], [5], [6], [7], [8], [9] 6 [3.8-12.3] 0.92
Median hospital LOS, days [IQR] 13.8 [8.9-20.2] 15.4 [9.1-23.8] 0.53
Median duration of mechanical ventilation, days [IQR] 2 [1], [2], [3], [4] 3 [1-7.8] 0.23
In-hospital mortality, n (%) 5 (10) 3 (6) 0.71

Adverse events

Bleeding

Hypotension

 

6 (12)

4 (8)

 

6 (12)

5 (10)

 

1.00

1.00

Adverse Events No difference in the incidence of adverse events between groups (iNO, 20% vs iEPO, 22%; P = 1.00). Bleeding (12% in both groups) and hypotension (iNO 8% vs iEPO 10%).
Study Author Conclusions The relative efficacy of iEPO appeared to be similar to that of iNO in reducing mPAP following cardiac surgery, with significant cost savings associated with the use of iEPO.
Critique The study is limited by its retrospective design and single-center setting, which may affect generalizability. The transition to a new electronic medical record system during the study period could have led to variations in documentation. Additionally, variability in weaning practices among providers and the lack of a formal protocol for tapering treatment may have influenced results. A larger patient cohort and randomized controlled trials are needed to establish iEPO as the drug of choice for acute PHT following cardiac surgery.
References:
[1] [1] McGinn K, Reichert M. A Comparison of Inhaled Nitric Oxide Versus Inhaled Epoprostenol for Acute Pulmonary Hypertension Following Cardiac Surgery. Ann Pharmacother. 2016;50(1):22-26. doi:10.1177/1060028015608865

Inhaled epoprostenol vs inhaled nitric oxide for refractory hypoxemia in critically ill patients
Design

Retrospective, single-center analysis

N= 105

Objective To compare efficacy, safety, and cost outcomes in patients who have received either inhaled epoprostenol (iEPO) or inhaled nitric oxide (iNO) for hypoxic respiratory failure
Study Groups

iEPO (n= 52)

iNO (n= 53)

Inclusion Criteria Admitted to an ICU at Brigham and Women's Hospital between January 1, 2009, and October 31, 2010; 18 years or older; received either iNO or iEPO for improvement in oxygenation
Exclusion Criteria Received greater than 2 hours of concomitant iNO and iEPO therapy
Methods Patients were started on pulmonary vasodilator therapy after failing maximal conventional therapy. iNO dose range: 1 to 80 ppm, starting at 20 ppm. iEPO dose range: 0.01 to 0.05 μg/kg per minute, starting at 0.05 μg/kg per minute. Duration of therapy based on clinical response.
Duration January 2009 to October 2010
Outcome Measures

Primary: Change in PaO2/FIO2 ratio after 1 hour of therapy

Secondary: ICU length of stay, hospital length of stay, duration of study therapy, duration of mechanical ventilation, incidence of adverse events, cost

Baseline Characteristics   iNO (n = 53) iEPO (n = 52)
Age, y (mean ± SD) 51.8 ± 17.9 56.4 ± 15.3
Sex, male, n (%) 22 (41.5) 21 (40.4)
Weight, kg (mean ± SD) 84.2 ± 28.7 102.9 ± 47.3
White 44 (83.0) 47 (90.3)
APACHE II, median (IQR) 18 (15.5-21) 18 (15-22)
Results   iNO (n = 53) iEPO (n = 52) p-value
Change in PaO2/FIO2 ratio after 1 hour 20.58 ± 91.54 33.04 ± 36.19 0.36
Duration of mechanical ventilation, d 7 (3-17) 9.5 (6-19) 0.07
ICU length of stay, d 15 (6-31) 15.5 (8-23) 0.67
Hospital length of stay, d 33 (12-53) 20.5 (14-33) 0.26
Cost of therapy per patient, USD $3930 ± $4210 $838 ± $997 <0.0001
Adverse Events No adverse events were attributed to either therapy.
Study Author Conclusions We found no difference in efficacy and safety outcomes when comparing iNO and iEPO in hypoxic, critically ill patients. Inhaled epoprostenol is associated with less drug expenditure than iNO.
Critique The study provides valuable insights into the cost-effectiveness of iEPO compared to iNO, with no significant differences in efficacy and safety. However, the retrospective design and single-center setting may limit the generalizability of the findings. Additionally, the study did not control for all potential confounding factors, such as concomitant therapies and nonpharmacologic strategies, which could have influenced the results.
References:
[1] [1] Torbic H, Szumita PM, Anger KE, Nuccio P, LaGambina S, Weinhouse G. Inhaled epoprostenol vs inhaled nitric oxide for refractory hypoxemia in critically ill patients. J Crit Care. 2013;28(5):844-848. doi:10.1016/j.jcrc.2013.03.006

A prospective, randomized, crossover pilot study of inhaled nitric oxide versus inhaled prostacyclin in heart transplant and lung transplant recipients
Design

Prospective, randomized, crossover pilot study

N= 25

Objective To compare nitric oxide and prostacyclin in the treatment of pulmonary hypertension, refractory hypoxemia, and right ventricular dysfunction in thoracic transplant recipients
Study Groups

Nitric oxide (n= 14)

Prostacyclin (n= 11)

Inclusion Criteria All adult patients undergoing heart and lung transplantation at the University of California Los Angeles
Exclusion Criteria Patients who failed to complete the study protocol due to early clinical improvement or hemodynamic instability
Methods

Heart and lung transplant recipients were randomized to nitric oxide (20 ppm) or prostacyclin (20,000 ng/mL) as initial treatment, followed by a crossover to the other agent after 6 hours.

Hemodynamic and oxygenation parameters were recorded before and after initiation of therapy, at crossover, and after crossover agent administration.

Duration 6-hour crossover trial
Outcome Measures

Primary: Mean pulmonary artery pressure

Secondary: Central venous pressure, cardiac index, mixed venous oxygen saturation, mean systemic arterial pressure, oxygenation index

Baseline Characteristics   All patients (n= 25)
Age, years 59 ± 2
Gender - Male 20
Procedure - Single-lung transplant 17
Procedure - Double-lung transplant 2
Procedure - Orthotopic heart transplant 6
Results   Nitric oxide (n= 14) Prostacyclin (n= 11)
PA systolic (mm Hg) 43 ± 2 51 ± 2
PA diastolic (mm Hg) 24 ± 1 27 ± 3
PA mean (mm Hg) 32 ± 1 37 ± 3
CVP (mm Hg) 14 ± 1 14 ± 1
Cardiac index (L/min/m2) 2.5 ± 0.2 2.6 ± 0.2
SvO2 (%) 69 ± 3 71 ± 2
Adverse Events There were no complications associated with nitric oxide or prostacyclin
Study Author Conclusions In heart transplant and lung transplant recipients, nitric oxide and prostacyclin similarly reduce pulmonary artery pressures and central venous pressure, and improve cardiac index and mixed venous oxygen saturation. Inhaled prostacyclin may offer an alternative to nitric oxide in the treatment of pulmonary hypertension in thoracic transplantation.
Critique The study provides valuable insights into the use of inhaled prostacyclin as an alternative to nitric oxide, highlighting its similar efficacy and potential advantages in terms of cost and ease of delivery. However, the small sample size and the specific patient population limit the generalizability of the findings. Additionally, the study's crossover design may not fully account for long-term effects or differences in outcomes beyond the 6-hour trial period.
References:
[1] [1] Khan TA, Schnickel G, Ross D, et al. A prospective, randomized, crossover pilot study of inhaled nitric oxide versus inhaled prostacyclin in heart transplant and lung transplant recipients. J Thorac Cardiovasc Surg. 2009;138(6):1417-1424. doi:10.1016/j.jtcvs.2009.04.063

Addition of Inhaled Treprostinil to Oral Therapy for Pulmonary Arterial Hypertension: A Randomized Controlled Clinical Trial
Design

Randomized, placebo-controlled, double-blind, multicenter study

N= 235

Objective To assess the efficacy and safety of inhaled treprostinil in pulmonary arterial hypertension (PAH) patients receiving therapy with either bosentan or sildenafil
Study Groups

Inhaled treprostinil (n= 115)

Placebo (n= 120)

Inclusion Criteria Patients aged 18-75 years with idiopathic or familial PAH or PAH associated with collagen vascular disease, HIV infection, or anorexigen use; NYHA functional class III or IV; baseline 6MWD between 200 and 450 m; receiving bosentan 125 mg daily or any prescribed dose of sildenafil for at least 3 months
Exclusion Criteria Pregnant or nursing; diagnosed with acute or chronic illness other than those associated with PAH; received investigational medications, prostanoids, or phosphodiesterase inhibitors other than sildenafil within 30 days; changed or discontinued any PAH medication within 3 months
Methods Patients were randomized to receive either inhaled treprostinil sodium or placebo 4 times daily in combination with bosentan or sildenafil. Therapy initiated at 3 breaths (18 µg)/inhalation, increased to a maximum of 9 breaths (54 µg) as tolerated. 
Duration 12 weeks
Outcome Measures

Primary: Peak 6MWD at 12 weeks 

Baseline Characteristics   Inhaled TRE (n= 115) Placebo (n= 120)
Age, yrs 55 (20–75) 52 (18–75)
Male/female 22/93 22/98

PAH etiology

IPAH or familial

CVD

 

64 (56)

40 (35)

 

67 (56)

37 (31)

Background PAH therapy

Bosentan

Sildenafil

 

77 (67)

38 (33)

 

88 (73)

32 (27)

Time on background therapy, weeks Bosentan

Sildenafil

 

99 ± 79

65 ± 60

 

90 ± 75

77 ± 69

Baseline NYHA III/IV 112/3 118/2
Baseline 6MWD, m 346 ± 63 351 ± 69
Results   Inhaled TRE (n= 115) Placebo (n= 120) p-value
Peak 6MWD change from baseline at week 12, m 21.6 (IQR: -8.0 to 54.0) 3.0 (IQR: -26.0 to 31.5) 0.0004
Adverse Events Common adverse events: Cough (54% vs. 29%), headache (41% vs. 23%), nausea (19% vs. 11%), dizziness (17% vs. 15%), flushing (15% vs. <1%)
Study Author Conclusions Inhaled treprostinil improves exercise capacity and quality of life in PAH patients who remain symptomatic on bosentan or sildenafil, and is safe and well-tolerated.
Critique The study is well-designed with a robust sample size and clear endpoints, demonstrating significant improvements in exercise capacity and quality of life. However, the short duration of 12 weeks limits the understanding of long-term effects. The exclusion of certain PAH subtypes and patients with less severe disease may limit generalizability. Potential unblinding due to side effects is a concern. Further studies are needed to explore the combination with other therapies and longer-term outcomes.
References:
[1] [1] McLaughlin VV, Benza RL, Rubin LJ, et al. Addition of inhaled treprostinil to oral therapy for pulmonary arterial hypertension: a randomized controlled clinical trial. J Am Coll Cardiol. 2010;55(18):1915-1922. doi:10.1016/j.jacc.2010.01.027

BREEZE: Open‐label clinical study to evaluate the safety and tolerability of treprostinil inhalation powder as Tyvaso DPI™ in patients with pulmonary arterial hypertension
Design

Open-label, single-sequence, multicenter study

N= 51

Objective To evaluate the safety and tolerability of treprostinil inhalation powder (TreT) in patients currently treated with treprostinil inhalation solution
Study Groups Treprostinil inhalation powder (n= 51)
Inclusion Criteria Adults (≥18 years) diagnosed with PAH, on a stable regimen of treprostinil inhalation solution for ≥3 months, FEV1 ≥60%, FEV1/FVC ratio ≥60%
Exclusion Criteria Pregnant or lactating, taking other prostacyclin analogs or agonists, history of uncontrolled sleep apnea, parenchymal lung disease, or significant left-sided heart disease
Methods Patients transitioned from treprostinil inhalation solution to TreT at a corresponding dose. Evaluations included 6MWD, PQ-ITD, PAH-SYMPACT questionnaire, and systemic exposure and pharmacokinetics for up to 5 hours. Adverse events were monitored
Duration 3-week treatment phase with an optional extension phase
Outcome Measures

Primary: Safety and tolerability of TreT

Secondary: Systemic exposure and pharmacokinetics, 6MWD, device satisfaction and preference, PAH-SYMPACT questionnaire

Baseline Characteristics   Treprostinil inhaled powder (treatment phase)
Age, years - Mean (SD) 55.9 (13.4)
Sex - Female 43 (84.3%)
Race - White 40 (78.4%)
Race - Black or African American 9 (17.6%)
Baseline BMI, kg/m2 - Mean (SD) 29.87 (6.74)
Time since PAH diagnosis, years - Mean (SD) 8.69 (6.51)
Current PAH diagnosis - Idiopathic/familial 29 (56.9%)
WHO functional class at screening - II 31 (60.8%)
Background PAH medications - Any background PAH medication 50 (98.0%)
Results Visit week 6MWD, mean (SD), m Change from baseline, mean (SD) p-Value
Baseline 418.9 (109.4) - -
3 438.9 (110.5) 11.5 (32.9) 0.0217
11 416.1 (125.2) 7.9 (45.5) 0.3354
19 439.1 (112.3) 7.8 (43.0) 0.3036
27 446.5 (122.3) 13.1 (54.7) 0.1702
35 462.4 (122.6) 17.3 (40.3) 0.0518
43 476.7 (101.9) 26.4 (53.7) 0.0522
51 467.6 (120.5) 30.1 (60.2) 0.0563
Adverse Events Common Adverse Events: Cough (35%), headache (16%), dyspnea (8%) No study drug-related serious adverse events
Study Author Conclusions Transition from treprostinil inhalation solution to TreT is safe, well-tolerated, and accompanied by statistically significant improvements in key clinical assessments and patient-reported outcomes with comparable systemic exposure between the two formulations
Critique The study demonstrated significant improvements in patient satisfaction and clinical outcomes with TreT. However, the open-label design and lack of a control group may introduce bias. The short follow-up period and small sample size limit the ability to generalize findings. Further studies with longer follow-up and larger sample sizes are needed to confirm these results
References:
[1] [1] Spikes LA, Bajwa AA, Burger CD, et al. BREEZE: Open-label clinical study to evaluate the safety and tolerability of treprostinil inhalation powder as Tyvaso DPI in patients with pulmonary arterial hypertension. Pulm Circ. 2022;12(2):e12063. Published 2022 Apr 7. doi:10.1002/pul2.12063