Researchers at the Mayo Clinic, Rochester, MN, published a clinical case study in which a novel therapeutic approach showed promising results in treating a fairly uncommon lung disease, known as Pulmonary Langerhans cell histiocytosis (PLCH). The case-study entitled “Dramatic and sustained responsiveness of pulmonary Langerhans cell histiocytosis-associated pulmonary hypertension to vasodilator therapy,” is published in the November 2014 online edition of Respiratory Medicine Case Reports.

 Background Terminology

• Clinical Case-study: A clinical study that focuses on the medical history and outcomes of an individual patient.
• Langerhans cells: specialized immune cells most often found in the lungs, layers of the skin, and mucosa.
• Pulmonary Hypertension (PH): a condition that cause breathlessness, loss of exercise capacity, and death due to elevated pulmonary artery pressure and subsequent heart failure. It is a rare condition affecting 1 to 2 individuals for every 1 million in the US and Europe.

About PLCH

PLCH is a rare disorder that is caused by an abnormal accumulation of excess Langerhans cells in the lung tissue.  It has been attributed to partaking in the high risk behavior of smoking and it has a high mortality rate among diagnosed patients. The majority of patients are Caucasian and most often are diagnosed in their 30s or 40s; females usually present with diagnosable symptoms later in life.

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Symptoms of the disorder include PH, shortness of breath (dyspnea), nonproductive cough, fatigue, fever, weight loss, and chest pain. Around 10% of patients may experience a sudden and spontaneous deflated lung (pneumothorax).

Current treatment options are limited and they include:

• Recommend smoking cessation
• Corticosteroids and cytotoxic drugs
• If smoking has ceased a possible lung transplantation

This Case Study

In this study, the researchers tried a new approach to treating PLCH PH in a A 46-year-old Caucasian female who had been diagnosed with PLCH in 2003 and was confirmed to have PLCH PH after a lung biopsy in 2012.

After the patient failed to respond to the usual treatment options her physicians used an innovative approach and initiated a regimen of vasodilator drugs used predominantly in the treatment of advanced PH:

• bosentan 125 mg twice per day
• sildenafil 20 mg three times daily

The results were surprising. The patient showed dramatic improvement and the severity of her symptoms significantly decreased.  She was later switched to a daily dose of tadalafil 120 mg. After 10 years of taking the medication the patient showed sustained improvement despite maintaining her smoking status.

The results of this case study are important for they provide evidence that PLCH PH may be responsive to advanced PH therapies. This could possibly lead to lowered morbidity and mortality rates in patients as well as adding to the list of existing therapies that are currently available to these patients.