Gene/Protein Disease Symptom Drug Enzyme Compound
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Query: UMLS:C0019621 (Langerhans cell histiocytosis)
 3,250 document(s) hit in 31,850,051 MEDLINE articles (0.00 seconds)

Erdheim-Chester disease (ECD) is a rare non-Langerhans cell histiocytosis with a characteristic long bone lesion. Although about half of the patients have extraosseous organs, pulmonary involvement is very rare. We report a 55-year-old man with ECD who complained of severe dyspnea despite home oxygen therapy with noninvasive positive pressure ventilation. Continuous PGI2 administration was not very effective, but administration of cyclophosphamide and prednisolone induced rapid improvement of respiratory failure and the effect for six months on arterial blood gas analysis and stability of the disease state persisted.
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PMID:[A case of Erdheim-Chester disease effectively treated by cyclophosphamide and prednisolone]. 1731 26

Despite the introduction of new drugs that have changed the course of pulmonary arterial hypertension (PAH), some patients are still refractory to treatment and deteriorate rapidly. Long-acting phosphodiesterase-5 inhibitors are a new class of drugs that are effective in PAH. This prospective study assessed the potential of combination therapy with prostacyclin and tadalafil for treatment of severe PAH. We report four cases of severe PAH that deteriorated despite prostacyclin therapy. Two patients had Eisenmenger syndrome, one had pulmonary hypertension associated with scleroderma and one had histiocytosis X. All were treated with tadalafil, 10-20 mg once daily, in addition to prostacyclin. After 3 months of treatment, all patients improved clinically, with an increase in mean 6MWD from 214 to 272 m. In three patients, the New York Heart Association functional class decreased from IV to III. Echocardiograms showed no significant changes in pulmonary arterial pressure. Although this study was limited by the small sample size, it suggests that tadalafil in combination with prostacyclin is an effective treatment for severe PAH. Tadalafil may be beneficial for the treatment of patients with advanced disease.
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PMID:Combination therapy with prostacyclin and tadalafil for severe pulmonary arterial hypertension: a pilot study. 1881 91

Pulmonary hypertension is a pathophysiologic condition characterized by the increase of mean pulmonary arterial pressure > or =25 mmHg. A concomitant increase of pulmonary wedge pressure >15 mmHg may be present (post-capillary pulmonary hypertension) or not (precapillary pulmonary hypertension). The increase of pulmonary arterial pressure and of pulmonary vascular resistance and consequent elevation of the right ventricular afterload lead to right ventricular failure after variable periods of time. Pulmonary hypertension is present in multiple clinical conditions which have been classified in five groups. Pulmonary arterial hypertension (group 1) includes the familial and the idiopathic form and the forms associated with anorexigen drug use, connective tissue diseases, congenital heart diseases, HIV infection and portal hypertension. Group 2 includes all left heart diseases characterized by the increase of left atrial pressure and pulmonary wedge pressure (post-capillary pulmonary hypertension). Group 3 includes parenchymal lung diseases (chronic obstructive lung disease, lung fibrosis, ecc). Chronic thromboembolic pulmonary hypertension (group 4) is characterized by the obstruction of elastic pulmonary arteries at different levels by organized thromboembolism. Group 5 includes heterogeneous conditions such as sarcoidosis and histiocytosis X. These clinical groups are characterized by different pathobiologic and pathophysiologic mechanisms and therapeutic strategies. The exact pathobiologic mechanisms leading to pulmonary arterial hypertension (group 1) are unknown. Genetic factors (inheritable forms), predisposing factors (female gender) and exogenous factors (drugs, antibodies, viruses, congenital heart disease, etc). Endothelial dysfunction of lung microcirculation is invariably present and is characterized by the reduction of vasodilator and antiproliferative substances (prostacyclin, nitric oxide) and by the increase of vasoconstrictor and mitogenic factors (endothelin, thromboxane A2). Current approved therapies are targeted to the correction of this imbalance, which leads to the progressive increase of pulmonary vascular resistance. Different therapeutic strategies that are effective in diverse groups require an appropriate diagnostic algorithm in order to identify the precise group and specific conditions within the group. Evaluation of vasoreactivity and assessment of the severity of functional and hemodynamic changes are also required in pulmonary arterial hypertension for an appropriate therapeutic decision-making and estimate of results.
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PMID:[Pulmonary arterial hypertension. Part I: pathobiologic, pathophysiologic, clinical and diagnostic aspects]. 1953 40