Inpatient initiation and titration of riociguat resulted in rapid clinical improvement and reduced oxygen requirement within 72 hours in a patient with acute decompensation of inoperable chronic thromboembolic pulmonary hypertension refractory to optimal diuresis and anticoagulation.
Key Findings
Results
Riociguat initiated as inpatient rescue therapy produced rapid clinical improvement within 72 hours in a patient with acutely decompensated inoperable CTEPH.
The patient had remained refractory to optimal diuresis and anticoagulation for 14 days prior to riociguat initiation
High-flow oxygen requirements had not decreased after 14 days of conventional therapy
Clinical improvement and reduced oxygen requirement were observed within 72 hours of riociguat initiation
Riociguat was initiated and titrated in the inpatient setting
Background
The patient presented with acute decompensation of CTEPH manifesting as worsening dyspnoea, haemoptysis, and bilateral lower limb oedema with clinical signs of right heart failure.
The patient was a man in his 70s, a retired coal miner with prior asbestos exposure and no smoking history
Initial presentation included syncope and progressive exertional dyspnoea
Initial CTPA revealed bilateral lobar pulmonary emboli and TTE showed evidence of pulmonary hypertension
Re-presentation to the emergency department included worsening dyspnoea, haemoptysis, and bilateral lower limb oedema
Repeat TTE and CTPA confirmed persistent findings of chronic embolic disease
Results
A ventilation/perfusion scan demonstrated extensive mismatched perfusion defects consistent with CTEPH, and subsequent right heart catheterisation confirmed inoperable CTEPH.
V/Q scan showed extensive mismatched perfusion defects consistent with CTEPH
Right heart catheterisation was performed after the acute admission and confirmed inoperable CTEPH
The patient was referred to the Irish National Pulmonary Hypertension Unit for further evaluation
The multidisciplinary team decided to continue riociguat as long-term medical therapy due to inoperability based on insufficient surgically accessible thromboembolic burden and coexisting left ventricular dysfunction.
The patient was reviewed at the National Pulmonary Hypertension Clinic following the acute admission
Two factors precluding surgical intervention were identified: insufficient surgically accessible thromboembolic burden and coexisting left ventricular dysfunction
Riociguat, initially used as inpatient rescue therapy, was continued as the long-term treatment strategy
The patient had been established on long-term oxygen therapy (LTOT) prior to the acute decompensation
What This Means
This case report describes a man in his 70s, a retired coal miner with asbestos exposure, who developed a serious lung condition called chronic thromboembolic pulmonary hypertension (CTEPH), where old blood clots block blood vessels in the lungs and raise blood pressure in the pulmonary circulation. He became severely ill in the hospital with worsening breathlessness, coughing up blood, and leg swelling caused by right heart failure. Despite two weeks of standard treatments including diuretics (water tablets) and blood thinners, his condition did not improve and he continued to need high levels of supplemental oxygen.
The medical team then started him on a medication called riociguat, which works by relaxing and widening the blood vessels in the lungs. Remarkably, within just 72 hours of starting this drug in the hospital, his condition improved significantly and his need for oxygen decreased. Further testing confirmed that his CTEPH was not suitable for surgery — the clots were not in locations accessible to surgeons, and he also had a weakened heart on the left side — so riociguat was continued as his ongoing long-term treatment.
This research suggests that riociguat may be a viable rescue therapy for patients hospitalized with severe, acutely worsening CTEPH who do not respond to standard treatments. The case highlights a potential role for starting and adjusting riociguat dosing during a hospital stay in this difficult-to-treat population, though findings from a single case report cannot be generalized without further research.