Prognostic factors for health-related quality of life in adults and children with primary antibody deficiencies receiving SCIG home therapy
Introduction
Primary immunodeficiency disorders (PIDs) are a group of chronic diseases, with more than 120 genetically distinct subtypes identified [1]. PIDs that involve absence or low levels of antibodies (primary antibody deficiencies, PAD) result in an increased susceptibility to bacterial infections [2]. It was shown more than 50 years ago that regular replacement therapy with antibodies (immunoglobulin G, IgG) derived from human plasma decreases the risk and severity of infections [3].
IgG replacement therapy consists of either intravenous (IVIG) or subcutaneous (SCIG) IgG infusions. Both methods of administration have been shown to be safe and to effectively decrease the incidence and severity of infections in patients suffering from PAD [4], [5], [6], [7], [8], [9], [10], [11], [12], and both methods can be used as self-infusions at home [7], [8], [11], [12], [13], [14], [15], [16], [17]. Patients treated with IVIG infusions receive their therapy once every 2 to 5 weeks, while the SCIG therapy is typically given as weekly infusions [7], [8], [10], [11], [12], [18], [19]. Several previous investigations have demonstrated that following training and education, adults and children can easily and successfully self-administer SCIG therapy at home, with consistent serum IgG levels and few systemic adverse reactions [7], [8], [10], [11], [12], [17], [20], [21], [22], [23], [24], [25]. SCIG self-administered at home is highly appreciated by patients and families since it simplifies daily life [7], [17], [22], [23], [24], [25], [26], [27], [28], [29].
The introduction of SCIG self-infusions at home has improved the health-related quality of life (HRQL) and treatment satisfaction (TS) in both children and adults with PAD [17], [25], [27], [29]. Only one publication has so far shed some light on factors that are of importance for HRQL, coping, and hope in adults with PAD [30]. The identification of factors that are associated with self-reported HRQL in these patients may help to improve care by identifying vulnerable individuals and by tailoring support, treatment modalities, and training to suit patients' particular need. This study was conducted to evaluate the HRQL and TS of adults and children with PAD before and after the introduction of SCIG self-infusions at home with the main aim to identify prognostic background factors (demographic, social, medical, and patient/parent reported factors) related to HRQL.
Section snippets
Study designs
The current analyses were performed on the combined data from two prospective, multinational clinical studies evaluating the efficacy, safety, HRQL, and TS in children and adults [11], [12], [25], [27]. To summarize, in both studies, the participants or parents administered weekly subcutaneous infusions of a liquid, pasteurized, polyvalent, human 16% IgG preparation (Vivaglobin®, CSL Behring, Marburg, Germany). The patients/parents were trained on SCIG self-administration at their local
HRQL and TS in adults and children
Significant improvements in SF-36 scores from baseline to month 10 were found in four scales for adults previously on IVIG therapy at the hospital/doctor's office: “health transition” (HT, P < 0.01), role – physical” (RP, P < 0.05), “general health” (GH, P < 0.05), and “vitality” (VT, P < 0.01) (Table 2). For patients already receiving IVIG at home at study start and now only changing the mode of IgG administration route, the SF-36 scores significantly improved for the scale GH (P < 0.05) (Table 2). For
Discussion
By combining the results of two studies and thus increasing sample size, the current analyses were carried out both to investigate the effect of the introduction of SCIG self-infusions at home on PRO measures such as HRQL and TS, and to identify prognostic factors impacting HRQL changes in children and adults suffering from PAD.
Of the 125 patients, 19 did not present a final assessment because they had dropped out during the study. To exclude that the underlying process was informative we did a
Vivaglobin clinical study group
North American trial European and Brazilian trial USA Austria Arthur H. Althaus, Louisville, KY Andreas Böck, Vienna Pedro C. Avila, San Francisco, CA Brazil Melvin Berger, Cleveland, OH Beatriz Costa Carvalho, Sao Paolo Sudhir Gupta, Irvine, CA Germany Robert W. Hostoffer, South Euclid, OH Michael Borte, Leipzig Lisa J. Kobrynski, Atlanta, GA Hartmut Peter, Freiburg Sigune Schmidt, Freiburg Robyn Levy, Atlanta, GA Ilka Schulze, Berlin Laurie Myers, Durham, NC Tim Niehues, Dusseldorf Hans D. Ochs, Seattle, WA Poland
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The Experiences of Children with Primary Immunodeficiency Who Receive Immunoglobulin Subcutaneously Instead of Intravenously
2024, Journal of Pediatric Health CareImmunoglobulin administration for the treatment of CIDP: IVIG or SCIG?
2020, Journal of the Neurological SciencesCitation Excerpt :Unlike IVIG which requires administration by a health care professional, subcutaneous immunoglobulin (SCIG) can be self-administered by patients in their home [8]. The flexibility of self-administration may lessen the burden associated with infusions thereby improving a patient's health-related quality of life (HRQoL) [9–12]. In March 2018 SCIG was approved by the FDA for maintenance treatment in adults diagnosed with CIDP [13].
Subcutaneous immunoglobulins in patients with multiple myeloma and secondary hypogammaglobulinemia: a randomized trial
2018, Clinical ImmunologyCitation Excerpt :Finally, the potential efficacy of IVIg has been evaluated during the plateau-phase of MM, when the serum level of the monoclonal component is low (thus preventing the risk of hyperviscosity syndrome during the active phase of the disease): a significant reduction in both frequency and severity of infections has been demonstrated in this condition [6–9,20]. SCIg preparations have been shown to be safe, cost-effective, and able to improve HRQoL in patients with primary immunodeficiencies [10,13,21–23]. They can be self-administered at home, do not require venous access and systemic premedication, and are characterized by a gradual absorption and a low incidence of systemic adverse events [13,14].
Subcutaneous Immunoglobulin Therapy for Hypogammaglobulinemia Secondary to Malignancy or Related Drug Therapy
2017, Transfusion Medicine ReviewsCitation Excerpt :There are a number of validated instruments available to measure the impact on an individual's quality of life, such as the Short Form 36 (SF-36) (http://www.sf-36.org/tools/sf36.shtml), the European EQ-5D (http://www.euroqol.org/about-eq-5d.html), and the Child Health Questionnaire–Parental Form 50 (https://www.healthactchq.com/chq.php). Studies in PID patients have reported improved HRQoL when switching from IVIg to SCIg therapy [36-39], with most PID patients indicating a preference for SCIg therapy. Reasons provided include fewer infusion-related adverse events such as prolonged headaches [40], more independence [38], and the greater flexibility of home administration [41].
Health-Related Quality of Life in Children and Adults with Primary Immunodeficiencies: A Systematic Review and Meta-Analysis
2019, Journal of Allergy and Clinical Immunology: In PracticeCitation Excerpt :The short-form 36 was the most frequently used instrument investigating adult HRQOL and was used in 21 studies.4-6,8,21-23,25,28,29,33,34,36,38,40-44,46,48 The Child Health Questionnaire - Parent Form 50 was the most frequently used instrument (9 studies) for assessing HRQOL in children.32,36,38,40-43,49,50 Three studies used HRQOL instruments that were not validated.10,39,45
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Complete list of investigator sites is provided at the end of the article.