Quality of Life Differences for Primary Immunodeficiency Patients on Home SCIG versus IVIG

Anterasian, Christine; Duong, Richard; Gruenemeier, P.; Ernst, Carol; Kitsen, Jessica; Geng, Bob

doi:10.1007/s10875-019-00705-5

Cited by 13 publications

(11 citation statements)

References 30 publications

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“…Indeed, it has been reported that families of children who underwent HCT favored infusion at home due to reduced need for clinic visits and increased participation in their child's treatment [24]. In one study, patient satisfaction scores were higher with SCIG compared to IVIG in adults who underwent HCT [25], similar to published reports on SCIG and patient satisfaction/quality of life in PIDD children and adults [52][53][54][55]. In our cohort, there were no treatment discontinuations among SCIG, possibly related to low rate of adverse events and higher overall satisfaction with SCIG.…”

Section: Discussionsupporting

confidence: 59%

Safety and Efficacy of Hizentra ® Following Pediatric Hematopoietic Cell Transplant for Treatment of Primary Immunodeficiencies

Patel

Torgerson²,

Thakar³

et al. 2022

Preprint

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Primary Immunodeficiency disease (PIDD) comprise a group of disorders of immune function. Some of the most severe PIDD can be treated with hematopoietic cell transplant (HCT). Hizentra® is a 20% liquid IgG product approved for subcutaneous administration in adults and children greater than two years of age with PIDD-associated antibody deficiency. Limited information is available on use of Hizentra® in children following HCT for PIDD. A multicenter retrospective chart review demonstrated 37 infants and children (median age 70.1 [range 12.0 to 176.4] months) with PIDD treated by HCT who received Hizentra® infusions over a median duration of 31 (range 4-96) months post-transplant. The most common indication for HCT was IL2RG SCID (n=16). Thirty-two patients switched from IVIG to SCIG administration, due to one or more of the following reasons: patient/caregiver (n=17) or physician (n=12) preference, discontinuation of central venous catheter (n=16), desire for home infusion (n=12), improved IgG serum levels following lower levels on IVIG (n=10), and loss of venous access (n=8). Serious bacterial infections occurred at a rate of 0.041 per patient-year while on therapy. Weight percentile increased by a mean of 16% during the observation period, with females demonstrating the largest gains. Mild local reactions were observed in 24%; 76% had no local reactions. One serious adverse event (death from sepsis) was reported. Hizentra® was discontinued in 15 (41%) patients, most commonly due to recovery of B-cell function (n=11). These data demonstrate that Hizentra® is a safe and effective option in children who have received HCT for PIDD.

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Section: Discussionsupporting

confidence: 59%

Safety and Efficacy of Hizentra ® Following Pediatric Hematopoietic Cell Transplant for Treatment of Primary Immunodeficiencies

Patel

Torgerson²,

Thakar³

et al. 2022

Preprint

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“…A recent study has shown that, over an entire population of patients with PID, IVIg and SCIg use are associated with similar HRQoL. 68 Personal preferences may therefore be an important part of matching each patient to the more appropriate therapy, potentially maximizing HRQoL for each individual. Inconvenience, travel and a lack of flexibility are frequently cited as reasons for preferring home treatment to hospital IV therapy, 30,33,69 suggesting that patients' work schedules, or distance from hospital, may play a part.…”

Section: Discussionmentioning

confidence: 99%

Patient Preferences for Subcutaneous versus Intravenous Administration of Treatment for Chronic Immune System Disorders: A Systematic Review

Overton¹,

Shalet²,

Somers³

et al. 2021

PPA

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Background: For many chronic immune system disorders, the available treatments provide several options for route of administration. The objective of this systematic literature review is to inform discussions about therapy choices for individual patients by summarizing the available evidence regarding the preferences of patients with chronic immune system disorders for intravenous (IV) or subcutaneous (SC) administration. Methods: Searches of the MEDLINE, Embase and Cochrane Library databases were conducted using terms designed to capture studies reporting patient preferences between IV and SC therapy published in English. Relevant studies were limited to those in which mode of administration, including treatment frequency and setting, was the main difference between comparators. Results: In total, 49 studies were included in the review. Among 18 studies that compared IV and SC immunoglobulin therapy, 16 found patients to prefer the SC administration route. The results of the 31 studies comparing IV infusion and SC injection of non-immunoglobulin therapies were mixed, with patients favoring SC administration in 20, IV infusion in seven, and having no overall preference in four. Patient experience had a strong effect on preferences, with treatment-experienced patients preferring their current administration route in most studies. Patients preferring SC administration tended also to prefer treatment at home, mainly due to the convenience and comfort of home treatment and the avoidance of having to attend hospital. By contrast, patients preferring IV infusion tended to cite the lower treatment frequency and a dislike of self-injecting, and preferred hospital treatment, mainly due to the presence of healthcare professionals and resulting feelings of safety. Conclusion:In general patients with chronic immune system disorders tend to be more likely to choose SC administration than IV infusion, but preferences may vary according among individuals. These findings may assist discussions around appropriate treatment choices for each patient.

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“…Value-based pricing of medications is used in the UK, Australia, Canada, and Sweden before the initial launch of a drug. 67,68 By estimating the value (cost per QALY) of an intervention, prices can be set fairly and within the cost-utility threshold. This practice is reflected in the much lower IRT costs in these countries, which span from $18 000 to $19 500.…”

Section: Discussionmentioning

confidence: 99%

“…In 2018, SCIG products were 1.2 to 1.6 times higher per gram than IVIG and were recommended by manufacturers to be given at 1.3 to 1.4 times higher doses . Furthermore, efforts to improve IRT utility have primarily involved increasing dosing and/or changing to subcutaneous infusions . Hence, any utility improvements achieved through these means will be offset by increased costs associated with consuming more and/or higher-priced infusion products.…”

Section: Discussionmentioning

confidence: 99%

Cost Utility of Lifelong Immunoglobulin Replacement Therapy vs Hematopoietic Stem Cell Transplant to Treat Agammaglobulinemia

et al. 2022

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IMPORTANCELifelong immunoglobulin replacement therapy (IRT) is standard-of-care treatment for congenital agammaglobulinemia but accrues high annual costs ($30 000-$90 000 per year) and decrements to quality of life over patients' life spans. Hematopoietic stem cell transplant (HSCT) offers an alternative 1-time therapy, but has high morbidity and mortality.OBJECTIVE To evaluate the cost utility of IRT vs matched sibling donor (MSD) and matched unrelated donor (MUD) HSCT to treat patients with agammaglobulinemia in the US. DESIGN, SETTING, AND PARTICIPANTSThis economic evaluation used Markov analysis to model the base-case scenario of a patient aged 12 months with congenital agammaglobulinemia receiving lifelong IRT vs MSD or MUD HSCT. Costs, probabilities, and quality-of-life measures were derived from the literature. Microsimulations estimated premature deaths for each strategy in a virtual cohort. One-way sensitivity and probabilistic sensitivity analyses evaluated uncertainty around parameter estimates performed from a societal perspective over a 100-year time horizon. The threshold for cost-effective care was set at $100 000 per quality-adjusted life-year (QALY). This study was conducted from 2020 across a 100-year time horizon.EXPOSURES Immunoglobulin replacement therapy vs MSD or MUD HSCT for treatment of congenital agammaglobulinemia MAIN OUTCOMES AND MEASURES The primary outcomes were incremental cost-effectiveness ratio (ICER) expressed in 2020 US dollars per QALY gained and premature deaths associated with each strategy. RESULTSIn this economic evaluation of patients with congenital agammaglobulinemia, lifelong IRT cost more than HSCT

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Quality of Life Differences for Primary Immunodeficiency Patients on Home SCIG versus IVIG

Cited by 13 publications

References 30 publications

Safety and Efficacy of Hizentra ® Following Pediatric Hematopoietic Cell Transplant for Treatment of Primary Immunodeficiencies

Safety and Efficacy of Hizentra ® Following Pediatric Hematopoietic Cell Transplant for Treatment of Primary Immunodeficiencies

Patient Preferences for Subcutaneous versus Intravenous Administration of Treatment for Chronic Immune System Disorders: A Systematic Review

Cost Utility of Lifelong Immunoglobulin Replacement Therapy vs Hematopoietic Stem Cell Transplant to Treat Agammaglobulinemia

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