Background/Purpose:

Trials of therapy in  pulmonary  hypertension(PH)  have generally excluded patients with significant interstitial lung disease, but many patients with systemic sclerosis(SSc)  and  PH  have some interstitial lung disease.   There are concerns that PH drugs may cause ventilation-perfusion mismatch and worsen PH, and it remains unclear if PH drugs improve outcomes for these patients.  PHAROS , Pulmonary Hypertension Assessment and Recognition of Outcomes in Scleroderma, is a prospective observational  multi-center cohort study of  SSc-PH.   Our objective is to describe the real-world experience of SSc-PH patients with restrictive lung disease treated with a single PH medication.   

Methods:

We included  patients with PH defined as a mean pulmonary artery pressure (mPAP) ≥25mmHg  on catheterization who had a FVC <70% predicted at the time of the diagnosis.  Patients were grouped into 3 groups, based on the treatment during the first year following the PH diagnosis:  No drug treatment, treatment with  an endothelin receptor blocker antagonist (ERA), or   andphosphodiesterase inhibitor (PDE).    For this initial look at the data, patients treated with combination therapy and/or  prostacyclins (PCA)  were excluded.  The primary outcome examined was one-year survival, with secondary outcomes of hospitalization, change in functional class, 6 minute walk  and patient questionnaires at one year.  

Results:

Of the 266 patients with PH in PHAROS,  98 had a FVC <70% and were included . There were 26 receiving no therapy (No Drug), 19 receiving ERA and 33 treated with PDE.  We excluded 18 treated with combination and 2 treated with PCAs alone.  There were no significant differences in the demographics or the cardiopulmonary parameters between the 3 groups.  These patients had striking restrictive disease with a mean FVC between 50 and 55% predicted.  Although over survival was not statistically different between the 3 groups,  patients in the PDE group had fewer deaths and a better 1 and 2 year survival than the No Drug group.  (p<0.05).    There were more patients in the PDE group who had an improvement compared to the ND group and neither treatment group had an increase in hospitalizations after starting the drugs (Table 1).  

Conclusion:

SSc patients with PH and restrictive lung disease are being treated with PH drugs.  Our observations suggest that  some patients may respond favorably, without evidence of worsening PH.   We strongly encourage further study of PH drugs in SSc-ILD patients in a controlled setting.  

 

	No Drug  n=26	ERA  n=19	PDE  n=33
Age – yr, mean	54.5	57.2	56.8
Disease Duration yr, mean	10.1	8.3	9.2
% Female sex	70	79	62
% White race	76	63	64
% Diffuse  cutaneous	42	63	42
FVC% predicted	52.2	49.9	55.5
DLCO % predicted	33.6	25.9	42.0
RHC m PAP	31.85	33.3	36
PVR  (Woods)	293	343	419
Response in 1st year	23%	37%	43%
Hospitalizations	2	1	2
Deaths in 1st year	7	3	1
Survival, 1, 2 year	69%,  61%	82%,  64%	96%,  76%

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ACR Meeting Abstracts - https://acrabstracts.org/abstract/treatment-of-pulmonary-hypertension-in-scleroderma-patients-with-restricitive-lung-disease-observations-from-the-pulmonary-hypertension-assessment-and-recognition-of-outcomes-in-scleroderma-coho/