No abstract

In patients with primary antibody deficiencies, subcutaneous administration of IgG (SCIG) replacement is effective, safe, well-tolerated, and can be self-administered at home. A new SCIG replacement at 20% concentration (Hizentra®) has been developed and has replaced Vivaglobin® (SCIG 16%).An observational prospective multi-centric open-label study, with retrospective comparison was conducted in 15 Italian centers, in order to investigate whether and to what extent switching to Hizentra® would affect frequency of infusions, number of infusion sites, patients’ satisfaction, and tolerability in patients previously treated with Vivaglobin® or intravenous immunoglobulins (IVIG).Any variations of dosage, frequency and duration of the infusions, and of number of infusion sites induced by Hizentra® with respect to the former treatment were recorded. Practical advantages and disadvantages of Hizentra®, with respect to the medicinal product formerly used, and the variations in patients’ therapy-related satisfaction were monitored by means of the TSQM (Treatment Satisfaction Questionnaire for Medication); number, frequency, and duration of infectious events and adverse effects were recorded.Eighty-two patients switched to Hizentra®: 19 (23.2%) from IVIG and 63 (76.8%) from Vivaglobin®. The mean interval between infusions was not affected by the shift (7.0 ± 2.0 days with previous treatment versus 7.1 ± 1.2 during Hizentra®). A decrease in the number of infusion sites with Hizentra® was recorded in 12 out of 56 patients for whom these data were available. At 6 months, 89.7% of patients were satisfied with Hizentra®; no difference in terms of effectiveness, side effects, convenience, and global satisfaction was observed. No difference in the incidence of adverse events was reported.

The administration of 20 % SCIg was beneficial and safe in maintaining a quiescent disease and in inducing a complete remission in moderately active disease in patients with DM or PM. The treatment with 20 % SCIg led to the possibility to discontinue and to reduce the use of glucocorticoids and/or the immunosuppressants. Patients reported their satisfaction in terms of contact with health professionals, quality of treatment-related information and administration convenience, with an improved quality of life.

Common variable immunodeficiency is the most common symptomatic primary immunodeficiency in adulthood. Pregnant women with common variable immunodeficiency have different needs from other patients with the same disease. Because of immature state of the fetal and neonatal immune system, transplacental transfer of immunoglobulin G (IgG) has a relevant role in protecting the infant. We here report on a high-risk pregnant woman with common variable immunodeficiency with adverse reactions to intravenous immunoglobulin that was successfully rescued with a new Ig human intravenous, 10% liquid preparation. The treatment was tailored to the health status and characteristics of the patient. The new product was safe and well tolerated. The mother did not report any infections during pregnancy and the baby had a healthy course with ‘protective’ serum IgG levels. Our case is a further demonstration that intravenous immunoglobulin tolerability in patients with immunodeficiency could be linked to a product's characteristics.

Objectives To describe disease outcome and the influence of treatment modalities in a cohort of patients with polymyositis (PM) and dermatomyositis (DM) prospectively followed by our Internal Medicine Department, between 1985 and 2013. Methods We reviewed the medical notes of 91consecutive patients with PM and DM diagnosed according to the Bohan and Peter criteria. Since the diagnosis each patient have been followed by a standardised protocol. In March-May 2013 all of patients were enrolled in this study with the objective to evaluate a) the disease outcome b) disease activity, c) damage due to disease and treatment d) and the influence of treatment in outcome in the course of the disease. Results The series comprised 91 subjects with either PM (43) or DM (48) (Table 1). Myogenic abnormalities were detected by EMG in all of the cases, whereas muscle biopsy confirmed the diagnosis in 78% of the cases. All patients were treated with glucocorticoids. 72 patients (78%) received one (n=18) or more (n=31) agents, such as immunosuppressants, IVIg (in 39 patients), SCIg (n=18). Twenty-two patients (24%) (12F =55%; mean age 66 years) died after a mean follow-up of 91±84 months. According to the age-adjusted Cox regression model, male sex and a high MITAX value at the onset significantly correlate with mortality [HR =2.4, 95% CI =1.0 to 5.6 and HR =1.5 (95% CI =1.1 to 2.1), respectively]. Kaplan-Meyer estimates higher mortality for patients treated with corticosteroids or with corticosteroids and immunosuppressants as compared to patients treated with IVIG and/or SCIg (LR test p-value =0.3). Five-, 10- and 15-year survivals were higher in patients treated with immunoglobulin as compared with those treated with glucocorticoids or glucocorticoids plus immunosuppressants (p<0.08,0.1 and 0.06,respectively). Conclusions PM and DM represent complex systemic disorders with a high rate of mortality. Patients initially treated with glucocorticoids and/or immunosuppressants had poorer outcome as compared with those treated with immunoglobulin (IV or SC). Disclosure of Interest None declared DOI 10.1136/annrheumdis-2014-eular.3245