Treatment of patients living with Primary Antibody Deficiencies (PAD) could impose demands on daily life to plan and self-care management, as a result of the need to comply with complex therapeutic schedules, while balancing family or job commitment. This can result in a ¿burden of treatment¿. Several studies indicated that PAD patients manifest poor health-related quality of life which worsen over the time, due to associated clinical conditions. PAD patients require replacement therapy for life: on this basis, the acceptability of the schedules and settings for immunoglobulins (Ig) administration is an important factor in improving quality of life. Recently, a study on 327 Italian CVID patients showed that Ig replacement therapy schedules did not impact the health-related quality of life (HRQoL) if the choice of treatment was individualized after a long educational period focused on verifying the Ig therapy schedule ongoing at enrolment time. Starting from this observation, in this proposal we aim to collect longitudinal data on HRQoL of patients affected by PAD on different schedule and route of Ig replacement therapy. We will measure the impact of the switch from intravenously immunoglobulin preparations (IVIG) and/or subcutaneous Ig (SCIG) to treatment with facilitated Ig by human recombinant hyaluronidase (fSCIG) on HRQoL in a population of PAD patients by a prospective, longitudinal, observational study. For the evaluation of HRQoL, a recently validated specific tool for Primary antibody Deficiencies ¿the CVID_QoL questionnaires¿ will be used. This instrument allowed to highlight the impact of the immunodeficiency as an overall problem on IgG replacement therapy recipients¿ well-being. Our study sought to generate information to help health care professionals to understand factors that may impact on patient¿s everyday life and contribute to maximizing patient empowerment, satisfaction with care, and burden of treatment while minimizing the impact of illness.
CVID is a primary immune deficiency disease often associated with severe and chronic infections.
Affected patients are subject to recurrent, severe, or unusual infections. The prognosis can vary from benign to very complex conditions. In some cases, patients develop autoimmune or gastrointestinal disorders and malignancies. These frequent and severe events often cause missed work and school days, or even require hospitalization. Additionally, patients can have infectious or inflammatory complications that result in permanent impairments such as chronic lung disease. For these reasons, patients with CVID report lower HRQOL compared to patients with other chronic diseases. There is substantial evidence that the standard replacement treatment with immunoglobulins prolongs survival, reduces morbidity, and exerts a positive effect on the patients' HRQoL. The current wide range of Ig presentations, including IVIG, SCIG, and fSCIG, should lend to an enhanced capacity to tailor treatment to individual patient features and preferences. This should improve outcomes in the treatment of CVID with Ig, given its life-long administration, as several factors may impact on replacement therapies (the route and setting of administration). But, independently from the IgRT schedule of administration, the psychological status of patients received Ig therapy was in general highly compromised.
In the last ten years, different studies have been performed to evaluate HRQoL in CVID. The majority of these trials were characterized by the use of generic, non-disease- specific instruments. In addition to this limitation, only observational or short-term longitudinal studies on small cohorts were performed. All the studies agreed that CVID patients have a poor quality of life, especially in the physical domain, suffer a lot, and are at risk of psychological distress. Because of the long lifetime of the disease, it is possible to speculate that quality of life in a population affected by a clinical and immunological heterogeneous disease may vary depending on age, treatment, clinical conditions, associated diseases, personal attitude, etc.
The aim of our proposal study is to evaluate HRQoL by using a specific validate tool for CVID (CVID_QoL) and to perform a longitudinal analysis differently from previous published studies.
Based on current knowledge, it is evident that studies about the HRQoL outcome in CVID should be extended from a simple assessment to multimodal and longitudinal assessments and should include the patients¿ reported outcome measures.
CVID_QoL identified 3 dimensions: emotional functioning (EF), relational functioning (RF), and gastrointestinal and skin symptoms (GSS) and had good internal consistency. The use of disease-specific tools is desirable to provide a more accurate picture of the burden of each disease and it is useful to identify QoL issues in patients with CVID that may not be addressed by generic instruments.
We suggest that improved HRQOL relies on early detection of disease and implementation of an individual treatment plan for the patient and for this reason we consider essential to evaluate differences in HRQoL related to different treatment regimens and routes of immunoglobulins administration.
HRQoL in CVID should be assessed in the frame of the wide spectrum of the severity of the disease, taking into consideration the long life course of the disease and of the treatment.
Due to the frailty of this population, the impact of illness on HRQoL is receiving increasing recognition. HRQOL measurements in CVID have an important role when analyzing not only the effect of the disease on patient well-being, but the satisfaction of patients with specific treatment regimens. Because patients with CVID require therapy indefinitely, the form of administration and setting where the product is administered are important factors that can significantly affect HRQoL.
The study sought to generate information to help health care professionals to understand factors that may impact on patient's everyday life and possibly contribute to maximizing patient empowerment and satisfaction with care, while minimizing the impact of illness.