Treatment with Hizentra in patients with primary and secondary ă immunodeficiencies: a real-life, non-interventional trial

Background: Although Hizentra is indicated for immunoglobulin ă replacement therapy in patients with primary and secondary ă immunodeficiencies, phase III trials have focused on patients with ă primary immunodeficiencies. In this 9-month, real-life, prospective, ă non-interventional, longitudinal, multicenter study of patients with ă primary and secondary immunodeficiencies in France, treatment modalities ă (primary endpoint), efficacy, safety, tolerability, quality of life, and ă treatment satisfaction were evaluated using descriptive statistics. ă Results: Starting in January 2012, 117 patients were enrolled (99 ă adults, 18 children). Secondary immunodeficiencies were present in 48.7 ă % of patients. At follow-up, injections were administered every 7 days ă in 92.2 % of patients. Nine patients (7.8 %) were taking Hizentra ă every 10-14 days. The median dose of Hizentra administered was 0.1 ă g/kg/injection. Fifty-six patients were administered doses 0.2 ă g/kg/injection. Mean trough IgG titers were 9.0 +/- 3.3 g/L (median 8.3 ă g/L). The mean yearly rate of infection was 1.2 +/- 1.9. Mean scores on ă the Short Form-36 physical and mental component summaries were 46.3 +/- ă 10.0 and 46.6 +/- 9.3, respectively. Scores on the Treatment ă Satisfaction Questionnaire for Medication ranged from 69.9 +/- 19.9 to ă 88.3 +/- 21.2 depending on the domain. Treatment with Hizentra was well ă tolerated. No single drug-related systemic reaction occurred in more ă than one patient and few local reactions were reported (n = 5). ă Conclusions: Under real-life conditions and in a cohort that included ă patients with primary and secondary immunodeficiencies, treatment with ă Hizentra was effective and well tolerated and patients were generally ă satisfied with the treatment.