A clinician’s guide for administration of high-concentration and facilitated subcutaneous immunoglobulin replacement therapy in patients with primary immunodeficiency diseases

Epland, Kristin; Suez, Daniel

doi:10.1186/s13223-022-00726-7

Cited by 10 publications

(3 citation statements)

References 65 publications

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“…Furthermore, trough levels were <700 mg/dL in about 22% of patients and <500 mg/dL in about 12% of patients; about 16% of patients did not know their most recent IgG trough level. Although target IgG trough levels vary depending on the type of PID and individual patient needs, most guidelines, including Japanese guidelines, recommend trough levels >700 mg/dL ( 26 – 28 ). IgG trough levels have been shown to inversely correlate with the incidence of pneumonia, with a five-fold difference in pneumonia incidence between trough levels of 500 mg/dL compared with 1000 mg/dL; this provides strong evidence that maintaining sufficient IgG levels helps prevent serious infections ( 29 ).…”

Section: Discussionmentioning

confidence: 99%

Patient-reported outcomes in patients with primary immunodeficiency diseases in Japan: baseline results from a prospective observational study

Kanegane,

Ishimura,

Kawai

et al. 2023

Front. Immunol.

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IntroductionPrimary immunodeficiency diseases (PIDs) are rare inherited diseases resulting in impaired immunity. People with PID experience lower health-related quality of life (HR-QOL) and disease-related burdens in daily activities. This ongoing, prospective observational study aims to evaluate disease activity, treatment status, treatment-related burden, daily activities, and HR-QOL in patients with PID in Japan over a 1-year period. In this interim report (database lock: July 29, 2022), we present baseline results.MethodsParticipants were enrolled between November 2021 and May 2022; data were collected four times/year per participant until May 2023 using an online electronic patient-reported outcomes system. Patients with PID and healthy volunteers aged ≥12 years, residing in Japan, and with access to a smartphone were eligible. HR-QOL (primary endpoint) was assessed by the EuroQol-5 Dimensions-5 Levels (EQ-5D-5L) and the Medical Outcomes Study 36-Item Short Form Health Survey (SF-36). Work productivity was assessed by the Work Productivity and Activity Impairment (WPAI) Questionnaire. Other aspects of PID and burden were assessed with a new questionnaire developed in-house. The study is registered at the University hospital Medical Information Network clinical trials registry (UMIN000045622).ResultsThe full interim analysis set comprised 71 patients with PID and 47 healthy volunteers. The most common International Union of Immunological Societies PID category was primary antibody deficiency (56.3% of patients). Complications were common, especially recurrent respiratory tract infections (63.4%). Most patients with PID were treated with immunoglobulin replacement therapy (73.2%); 22.4% of these patients had serum immunoglobulin levels <700 mg/dL. Among patients who did not undergo hematopoietic cell transplantation, EQ-5D-5L (n=67) and SF-36 (n=59) Physical and Mental Component Summary scores were significantly lower than in healthy volunteers (p < 0.001). WPAI absenteeism, work productivity loss, and activity impairment scores were significantly lower in 42 working patients with PID than in 37 working healthy volunteers (p < 0.05). Other results indicated that patients with PID experience substantial burdens related to medical visits, expenses, work, and daily activities.DiscussionThis interim analysis confirms that patients with PID in Japan have lower HR-QOL and work productivity compared with healthy individuals and experience substantial limitations and burdens in their daily lives.

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Section: Discussionmentioning

confidence: 99%

Patient-reported outcomes in patients with primary immunodeficiency diseases in Japan: baseline results from a prospective observational study

Kanegane,

Ishimura,

Kawai

et al. 2023

Front. Immunol.

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“…fSCIG treatment decreases the frequency of infection and hospitalization, reduces the impact on daily activities, and improves the quality of life. It can be individualized and enables the patient to manage their treatment [14].…”

Section: Introductionmentioning

confidence: 99%

Facilitated Subcutaneous Immunoglobulin Treatment Increases the Quality of Life and Decreases the Number of Infections and Hospitalizations in Children with Primary Immunodeficiencies

Erbaş Açıcı,

Topyildiz,

Aygün

et al. 2024

Int Arch Allergy Immunol

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Introduction: Immunoglobulin replacement therapy is an effective lifelong treatment modality used in patients with primary immunodeficiency to prevent and/or reduce the incidence of serious infections. Facilitated subcutaneous immunoglobulin (fSCIG) was developed to combine the advantages of intravenous immunoglobulin (IVIG) and subcutaneous immunoglobulin (SCIG) and is the latest method of immunoglobulin G (IgG) administration. In this study, switching to fSCIG administration in primary immunodeficiency patients receiving regular IVIG or SCIG therapy was evaluated, and serum IgG trough levels, frequency of infections, frequency and duration of hospitalizations, duration of absence from school/work, and quality of life were determined. Methods: In this study, fifteen patients with primary immunodeficiency who were previously receiving IVIG or SCIG treatment, followed by fSCIG, were evaluated retrospectively. Age, diagnosis, current complications, mean IgG value, frequency of infection, frequency of hospitalization, and duration of absenteeism from school and work were recorded during and before fSCIG treatment. At the beginning of fSCIG treatment, at 6th and 12th months, “The Quality of Life Scale” was also evaluated in patients and parents. Results: The most common indications for initiation of fSCIG treatment were the difficulty of access to the hospital and the long transfusion periods. No systemic adverse reactions were reported except for redness, swelling, and mild pain on the injection site. The median IgG values for the last 1 year were 529.6 mg/dL for IVIG (n = 9), 876.2 mg/dL for SCIG (n = 6) and 856.7 mg/dL for fSCIG (n = 15, all patients) treatment. The frequency of infections and the number of hospitalizations decreased significantly in the fSCIG group compared to both previous treatment modalities. There was a significant increase in the quality of life score of the patients and their families when compared with previous treatment modalities. Conclusion: fSCIG is an effective treatment method and is well tolerated in patients with immunodeficiency. It provides stable immunoglobulin levels and excellent protection against infections and offers the patients the possibility of home-based therapy.

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“…Compared with IVIG, SCIG is associated with fewer systemic adverse reactions and can be self-administered at home instead of at a clinic or hospital by a healthcare professional [1,7,8]. However, the volume of conventional SCIG treatment that can be infused subcutaneously is limited, which results in the need for more frequent infusions with SCIG than with IVIG [1,7,8]. The use of conventional SCIG also necessitates multiple infusion sites, which may deter some patients from receiving SCIG therapy.…”

Section: Introductionmentioning

confidence: 99%

A Phase 1 Open-Label Study to Assess the Tolerability, Safety, and Immunogenicity of Hyaluronidase-Facilitated Subcutaneous Immunoglobulin 20% in Healthy Adults

Nagy,

Duff,

Bauer

et al. 2023

J Clin Immunol

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Purpose Hyaluronidase-facilitated subcutaneous immunoglobulin (fSCIG) 20% will allow reduced infusion volumes and frequency versus existing subcutaneous therapies such as fSCIG 10% and conventional subcutaneous immunoglobulin 20%, respectively. We assessed the tolerability, safety, and immunogenicity of warmed and unwarmed fSCIG 20%. Methods This phase 1, single-dose, open-label, three-arm study enrolled healthy adults aged 19–50 years (inclusive) at a single US center (NCT05059977). Post-screening, participants received a single fSCIG 20% dose comprising recombinant human hyaluronidase and varying doses of in-line warmed or unwarmed immunoglobulin G (IgG) during a 4-day treatment period in a sentinel and sequential dosing design (treatment arm 1, warmed IgG 20% 0.4 g/kg; treatment arm 2, warmed IgG 20% 1.0 g/kg; treatment arm 3, unwarmed IgG 20% 1.0 g/kg). Participants were followed for 12 (± 1) weeks post-infusion. The primary endpoint was tolerability (“tolerable” infusions were not interrupted, stopped, or reduced in rate owing to fSCIG 20%-related treatment-emergent adverse events (TEAEs)). Secondary endpoints included occurrence of TEAEs. Results Overall, 24 participants were included, 8 per treatment arm (mean age 39.0 years, 54.2% men). All participants tolerated the infusions. All TEAEs were mild (107 events, in all participants), and all participants experienced fSCIG 20%-related (105 events) and local (102 events) TEAEs. Infusion site erythema and infusion site swelling were most frequently reported. No serious TEAEs occurred, and no participants discontinued the study owing to TEAEs. Conclusion fSCIG 20% was well-tolerated with a favorable safety profile in healthy adults. Future studies will evaluate fSCIG 20% in primary immunodeficiency diseases. Trial registration number (ClinicalTrials.gov): NCT05059977 (registered 28 September 2021).

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A clinician’s guide for administration of high-concentration and facilitated subcutaneous immunoglobulin replacement therapy in patients with primary immunodeficiency diseases

Cited by 10 publications

References 65 publications

Patient-reported outcomes in patients with primary immunodeficiency diseases in Japan: baseline results from a prospective observational study

Patient-reported outcomes in patients with primary immunodeficiency diseases in Japan: baseline results from a prospective observational study

Facilitated Subcutaneous Immunoglobulin Treatment Increases the Quality of Life and Decreases the Number of Infections and Hospitalizations in Children with Primary Immunodeficiencies

A Phase 1 Open-Label Study to Assess the Tolerability, Safety, and Immunogenicity of Hyaluronidase-Facilitated Subcutaneous Immunoglobulin 20% in Healthy Adults

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