Abstract
Primary immunodeficiency disorders (PIDs) are a group of heterogeneous rare disorders, whereby the immune system is missing or not functioning adequately. For patients requiring treatment, the most common option is immunoglobulin replacement therapy (Ig). Treatment of PIDs is simultaneously associated with both improvements in health-related quality of life (HRQoL) and increased treatment burden. This review sought to review studies investigating the burden of Ig treatment, synthesize evidence in relation to administration routes (subcutaneous or intravenous) and instruments used, as well as make recommendations for clinical and research applications in this area for patients aged 16 years and older. We searched Medline, EMBASE, and The Cochrane Library. Sifting of titles was performed by two reviewers, and the assessment of full-text articles by three. From a database which contained 3,770 unique results, 67 full texts were reviewed. Eventually, 17 studies were found to meet the inclusion criteria, and included in this review. Due to data heterogeneity, a narrative, descriptive synthesis of the evidence was undertaken. Most studies were carried out in the USA/North America, used a prospective observational design and involved patients with common variable immune deficiency. Four studies measured the burden of receiving IVIg therapy and 13 measured SCIg therapy. A wide range of measures, primarily designed to measure aspects of treatment satisfaction (e.g., life quality index or a slightly modified version) and HRQoL (e.g., The Short Form-36) had been used. Lack of a parallel control group in most studies meant that changes in outcomes could be due to factors other than changes in the treatment regimen. However, overall, PID patients appeared to report little Ig treatment burden and were satisfied with either modality. However, patient preference appeared to be the delivery of the Ig treatment in the patient's home and SCIg was preferred after switching from IVIg therapy. Individual differences appeared to affect treatment preference and therefore understanding the decision support needs of PID patients facing IG treatment choices would be valuable. Using a questionnaire specifically designed to measure the burden of Ig treatment from the patient's perspective is recommended in future research.
Highlights
Primary immunodeficiencies (PIDs) are a group of heterogeneous rare disorders whereby the immune system is missing or not working properly
The aim of this review was to synthesize the evidence in relation to the burden of Immunoglobulin replacement therapy (Ig) therapy as reported from the patient’s perspective, and to make recommendations for future clinical and research applications
Individual differences do affect treatment preference and exploring and identifying the decision support needs of Primary immunodeficiencies (PIDs) patients facing IG treatment choices would be valuable using a shared-decision making approach
Summary
Primary immunodeficiencies (PIDs) are a group of heterogeneous rare disorders whereby the immune system is missing or not working properly. Immunoglobulin replacement therapy (Ig) is the only treatment for most PID patients It involves undergoing a blood-based infusion at regular intervals to raise the antibodies needed to fight off infection. Burden of treatment (BoT) is a concept which can be defined as the consequences of receiving treatment (these may be medication, therapies, or other interventions). It describes the “work of being a patient”—everything the patient needs to do to treat and manage their illness, for example, undergoing tests and investigations, visiting doctors, adhering to treatment regimens, and making lifestyle changes (5, 6). Treatment of PIDs is simultaneously associated with both improvements in health-related quality of life (HRQoL) and increased treatment burden
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