Scleroderma

Description

Alternative Names

Treatment for Lung Complications

Cyclophosphamide. Cyclophosphamide (Cytoxan), an immunosuppressive agent, may be effective for preventing lung deterioration and is the important agent for treating pulmonary fibrosis. Cyclophosphamide, in both pulse (intravenous) and oral forms, has blocked some of the destructive actions of scleroderma in the lung. Intravenous cyclophosphamide can be life saving for patients with pneumonia from interstitial lung disease. Side effects include hair loss, infection, and bleeding into the urinary tract. To date, no other immunosuppressive agents have proven have any significant benefits.

Use of this agent may improve survival in patients who show early signs of lung deterioration, notably inflammation in the small lung airways (called alveolitis). The drug is not warranted for patents with existing stable pulmonary fibrosis and no signs of inflammation. In one study, patients with early signs of lung inflammation were given a course of intravenous pulses of the corticosteroid methylprednisolone (MP) and cyclophosphamide. Nearly all patients experienced improvement or stabilization during the first year, although the disease had progressed in two thirds of them by the end of two years.

Pulmonary Hypertension

No single regimen has proven to be effective for pulmonary hypertension in enough patients to warrant widespread use, although some are showing promise. Preliminary treatment guidelines published in 2004 suggest that oral anticoagulation is helpful in most patients. Diuretic treatment and supplemental oxygen are recommended in patients with fluid retention and low blood oxygen, respectively.

Prostaglandins (Prostacyclins). Prostaglandins (also called prostacyclins), which open blood vessels, are now the primary agents for treating pulmonary hypertension.

• Epoprostenol, which is administered intravenously, has improved exercise capacity and symptoms in both the short and long term in a number of patients. In some, survival is increased significantly. It should be noted that not all patients respond to this agent. The implanted catheter needed to deliver the agent can also cause serious complications. And the drug is not stable and may lose effectiveness.
• Iloprost is available in intravenous and inhaled form. Studies now suggest that the inhaled form improves exercise capacity and survival in some patients with pulmonary hypertension. In addition, infusions of iloprost remain effective over long periods (up to three years) of use.
• Treprostinil (Remodulin) is similar to epoprostenol but is more stable. It can also be administered using a portable pump that delivers the drug under the skin. This is less expensive, cumbersome, and invasive than the delivery methods for epoprostenol.

Potent vasodilators can have serious side effects, including worsening of pulmonary hypertension or a dangerous drop in blood pressure. Nevertheless, evidence is now strongly supporting the benefits of prostacylins for increasing exercise capacity and possibly improving some lung and heart functions in patients with pulmonary hypertension.

Endothelin Receptor Antagonists. Bosentan (Tracleer) is the first oral agent approved for pulmonary hypertension. This agent controls endothelin, a powerful molecule that causes blood vessels to narrow. Studies have reported improved exercise capacity in patients with pulmonary hypertension who took bosentan. The only side effect reported to date is headache, but long term studies are needed to determine safety and effectiveness.

Sildenafil (Viagra). Sildenafil (Viagra), the well-known agent used to treat impotence in men, is also a potent vasodilator, with specific actions in the lungs. Studies in 2002 have reported improvement in lung gas exchange when patients with pulmonary hypertension took this agent. One of the studies reported significant benefits when patients also took sildenafil along with inhaled iloprost.

Anti-Clotting Agents. Warfarin, an anti-clotting drug, has been useful for some patients.

Lung Transplantation. Lung transplantation may offer hope for people with pulmonary hypertension that do not respond to conservative measures. It should be noted that not all centers offer lung transplants to Medicaid patients. For example, there are no lung transplant services available to these patients in New York City.