Combined therapy with Revatio (sildenafil) and Opsumit (macitentan) was effective in a patient diagnosed with pulmonary hypertension (PH) associated with a rare blood disorder called polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin changes (POEMS) syndrome, a case report shows.
The report, “Effectiveness of pulmonary vasodilators on pulmonary hypertension associated with POEMS syndrome,” was published in the journal Respirology Case Reports.
POEMS syndrome is a blood disorder that affects different parts of the body, including nerves, organs, glands, and skin. One of the telltale signs of the disease is an excessively high concentration of vascular endothelial growth factor (VEGF) in the blood.
While many patients (33-48%) with POEMS syndrome end up developing PH, the mechanisms underlying the association between the disorders is still poorly understood.
A team of researchers from Chiba University in Japan described the clinical case of a 51-year-old woman who was diagnosed with PH associated with POEMS syndrome and was successfully treated with a combination of pulmonary vasodilators (agents that induce blood vessels’ widening and relaxation).
“Although several studies have reported that PH associated with POEMS syndrome is reversible after successful treatment with steroids, thalidomide [marketed as Thalomid in the U.S.], or autologous stem cell transplantation, the reports of PH in POEMS syndrome treated with pulmonary vasodilators are limited,” researchers said.
During the woman’s first hospital visit, she was diagnosed with POEMS syndrome after being examined and showing hallmark symptoms of the disorder, including drop foot (inability to lift the foot off the ground), a sensation of “pins and needles” in the skin, peripheral nerve damage (neuropathy), enlarged spleen, kidney disease, and low platelet count.
One year later, she returned to the hospital because her peripheral neuropathy had worsened. Clinical examination also revealed that her blood VEGF levels were extremely high and she had developed high systolic pulmonary artery pressure (sPAP) linked to PH. Consequently, she started treatment with dexamethasone and thalidomide to manage her symptoms.
Subsequent examinations revealed that her levels of VEGF had decreased; however, sPAP remained high. For this reason, clinicians decided to perform an auto-peripheral blood stem cell transplantation, which consists of the infusion of a patient’s own blood-forming stem cells. This strategy improved both her VEGF and sPAP levels.
Four years later, she began to complain of shortness of breath during physical activities, and her VEGF and sPAP levels increased again.
Chest X-ray and pulmonary function tests revealed that she had prominent enlargement of the pulmonary arteries with mild obstructive ventilatory impairment and reduced gas exchange capacity. Upon right heart catheterization, the clinical team concluded that her PH had worsened.
She then started treatment with Revatio and Opsumit, which significantly reduced both VEGF and sPAP levels, and markedly improved her PH status and renal function.
“We report the effectiveness of the vasodilators, sildenafil and macitentan, in a patient with PH associated with POEMS,” researchers stated. Supported by these findings, the team says pulmonary vasodilators should be considered for the treatment of these patients “when PH remains even after adequate treatment of POEMS syndrome.”