Treatment with alpha-1 proteinase inhibitor, modified process (Alpha-1 MP) appears to be safe and well tolerated in the long term in patients with alpha-1 antitrypsin deficiency (AATD), according to an article recently published in Respiratory Investigation.

“This study demonstrated that long-term weekly infusions of Alpha-1 MP were generally safe and well-tolerated in Japanese patients with AATD, for up to four years intervention,” the authors wrote.

This prospective research included 4 adult patients previously diagnosed with AATD, 3 of whom were male. All participants received weekly intravenous infusions of 60 mg/kg of Alpha-1 MP for 213 weeks. The mean number of received dosages was 210.8, achieving a minimum concentration of 55.73 mg/dL.

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Two of the patients experienced 3 treatment-emergent adverse events (TEAEs) that were potentially related to Alpha-1 MP, including hot flush, abdominal discomfort, and arthralgia. All were mild and resolved completely, as reported by Seyama et al.

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Altogether, 54 TEAEs occurred among the patients. Of these, 96.3% were mild, while 2 patients experienced 5 severe TEAEs in total.

The severe reactions were mostly related to the lungs (pulmonary hypertension, pneumonia, and pneumothorax). The study also reported cellulitis and a large intestine polyp. None of the severe TEAEs could be associated with Alpha-1 MP treatment.

The most common TEAEs that patients reported were upper respiratory tract inflammation, back pain, abdominal discomfort, arthralgia, hepatic dysfunction, nasopharyngitis, pneumonia, and arthropod sting.

Forced expiratory volume in 1 second (FEV1) and forced vital capacity (FVC) remained mostly stable throughout the study in all patients. “In addition, FEV1 decline was assessed throughout the study period as FEV1% predicted, which accounts for age-related FEV1 deterioration,” the authors explained.

Likewise, there were no relevant changes in vital signs or laboratory parameters. The study concluded without any chronic obstructive pulmonary disease exacerbations or deaths reported.

Reference

Seyama K, Suzuki M, Tasaka S, et al. Long-term safety of Prolastin®-C, an alpha1-proteinase inhibitor, in Japanese patients with alpha1-antitrypsin deficiency. Respir Investig. Published online August 12, 2022. doi:10.1016/j.resinv.2022.07.001

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