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Low-Dose Pioglitazone does not Increase ROS Production in Chronic Granulomatous Disease Patients with Severe
Xiaoying Hui1, Danru Liu1, Wenjie Wang1
1Department of Allergy and Clinical Immunology, Children's Hospital of Fudan University, 399 Wanyuan Road, Shanghai, 201102, China.
Purpose:
We sought to further investigate the efficacy and safety of pioglitazone for chronic granulomatous disease (CGD) patients with severe infection.
Methods:
CGD patients with severe infection were enrolled and treated with pioglitazone for 90 days. The degree of improvement in infection and the changes of dihydrorhodamine-123 (DHR) were used to evaluate the efficacy of pioglitazone. The adverse reaction of pioglitazone was also investigated.
Results:
We planned to enroll 30 patients at first in the study. However, the study was terminated due to negative results from all 3 enrolled patients. The 3 patients were diagnosed with CGD by clinical characteristics, DHR analysis, and genetics analysis. Mutations were CYBB (c.177C>A; p.C59X) in P1, CYBB (c.1498G>T; p.D500Y) in P2, and NCF2 (c.137T>G; p.M46R) in P3, respectively. The age of onset of the 3 patients was within 2 years after birth. The most common sites of infection were lung, lymph node, skin, and soft tissue, which were experienced in all 3 patients. The age of administration with pioglitazone was 5.2 years, 16 years and 11.1 years, respectively. The 3 patients experienced no improvement in severity of infection and stimulation index of the DHR did not also improve after receiving pioglitazone 10, 45 and 90 days, respectively. No drug-related adverse reaction was found during the period of pioglitazone.
Conclusions:
Low dose of pioglitazone did not improve the severity of infection and production of ROS in CGD patients with severe infection.
Insights
Pioglitazone did not improve severe infections or reactive oxygen species production in patients with chronic granulomatous disease (CGD). This study found no efficacy or adverse effects of pioglitazone in treating CGD patients.
Area of Science:
- Immunology
- Pharmacology
Background:
- Chronic granulomatous disease (CGD) is a primary immunodeficiency characterized by impaired phagocyte function.
- Severe infections are a major cause of morbidity and mortality in CGD patients.
Purpose of the Study:
- To investigate the efficacy and safety of pioglitazone in treating severe infections in CGD patients.
- To assess the impact of pioglitazone on reactive oxygen species (ROS) production in CGD.
Main Methods:
- A clinical trial involving CGD patients with severe infections treated with pioglitazone for 90 days.
- Efficacy was evaluated by infection severity and dihydrorhodamine-123 (DHR) assay.
- Safety was monitored through adverse event reporting.
Main Results:
- The study was terminated early due to negative results in all 3 enrolled patients.
- No improvement in infection severity or DHR stimulation index was observed after pioglitazone treatment.
- No drug-related adverse reactions were reported.
Conclusions:
- Low-dose pioglitazone demonstrated no efficacy in improving infection severity or ROS production in CGD patients.
- Pioglitazone is not an effective treatment for severe infections in this CGD cohort.
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