Facilitated Subcutaneous Immunoglobulin Treatment Patterns in Pediatric Patients with Primary Immunodeficiency Diseases

Abstract

Aim:

This retrospective study investigated real-world hyaluronidase-facilitated subcutaneous immunoglobulin (fSCIG) treatment patterns in pediatric patients with primary immunodeficiency diseases (PIDs) in Poland.

Methods:

Clinical and demographic information, fSCIG treatment parameters and clinical outcomes were extracted from medical records of 28 participants (aged ≤18 years) with PIDs who received fSCIG.

Results:

18 participants (64.3%) started fSCIG with a ramp-up (median duration: 35.5 days). 27 patients (96.4%) were administered fSCIG every 4 weeks and one patient every 3 weeks. 25 patients (89.3%) used one infusion site. No serious bacterial infections occurred.

Conclusion:

Data support the feasibility of administering fSCIG to children and adolescents with PIDs every 3–4 weeks using a single infusion site and indicate flexibility in modifying fSCIG infusion parameters.

Clinical Trial Registration: NCT04636502 (ClinicalTrials.gov)

Plain language summary

Antibodies, also known as immunoglobulins, are proteins that are made by the immune system to help fight infections. In primary immunodeficiency diseases (PIDs), part of the immune system may be missing or not working properly. This study looked at the use of an antibody treatment called hyaluronidase-facilitated subcutaneous immunoglobulin (or fSCIG) in Polish children aged 18 years or younger with PIDs. Information on patients, their disease, how fSCIG was being used and how patients responded to treatment was taken from medical records. Out of 28 patients, 18/28 (64.3%) had their fSCIG dose slowly increased, which took an average of 35.5 days. Overall, 27/28 patients were treated with fSCIG every 4 weeks (96.4%), and 25/28 patients used one place to inject fSCIG (89.3%). No serious infections caused by bacteria happened during the study. The study results suggest that children with PIDs could be treated every 3 to 4 weeks with fSCIG, and that flexibility in how fSCIG is injected may offer options suited to individual patients.

Tweetable abstract

Real-world Polish data on hyaluronidase-facilitated subcutaneous immunoglobulin use in pediatrics with primary immunodeficiency diseases support the feasibility of infusion every 3–4 weeks using a single site, and flexibility in modifying infusion parameters. Read more below.

Keywords::

• facilitated subcutaneous immunoglobulin
• hyaluronidase
• inborn errors of immunity
• pediatric
• primary immunodeficiency disease
• real-world

Supplementary data

To view the supplementary data that accompany this paper please visit the journal website at: www.tandfonline.com/doi/suppl/10.2217/imt-2023-0305

Author contributions

M Mach-Tomalska, S Drygała and E Heropolitańska-Pliszka conceived the study. M Kamieniak, S Drygała and E Heropolitańska-Pliszka contributed to the design of the study. All authors participated in data collection. S Drygała, M Kamieniak and J Kasprzak analyzed the data. S Drygała coordinated the project and funding for the project. All authors contributed to the manuscript and approved its final version.

Acknowledgments

The authors would like to express their deep gratitude and thank the patients and their parents for being involved in the data collection for the IG TATRY study. The authors are grateful to the following persons for their contribution to data collection, analysis, assistance with statistical analysis and critical review of the article: E Klemba, M Płaszczyca and M Kowalski.

Financial disclosure

This study is a part of the IG TATRY study funded by Takeda Pharma Sp. z o.o. M Mach-Tomalska received lecture fees and/or travel grants from CSL Behring, Octapharma and Takeda. AP Pukas-Bochenek received lecture fees and/or travel grants from Takeda. M Malanowska received lecture fees and/or travel grants from CSL Behring, Octapharma and Takeda. M Pac received lecture fees and/or travel grants from Biogen, CSL Behring, Grifols, Octapharma, Pfizer and Takeda. S Drygała was an employee of Takeda Pharma Sp. z o.o.at the time of the study and is currently employed by AstraZeneca. M Kamieniak is an employee and shareholder of Takeda Development Center Americas, Inc. J Kasprzak is a permanent employee of Takeda Pharma Sp. z o.o. E Heropolitańska-Pliszka received lecture fees and/or travel grants from CSL Behring, Kedrion, Octapharma and Takeda. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Competing interests disclosure

The authors have no competing interests or relevant affiliations with any organization or entity with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Writing disclosure

Medical writing support and editorial assistance was provided by M Kołtowska-Häggström, A Linkiewicz-Zegan and W Kurowska of Proper Medical Writing Sp. z o.o. and was funded by Takeda Pharma Sp. z o.o.

Ethical conduct of research

All patients or/and patients’ caregivers provided written informed consent, and the study was approved by the Ethics Committee at the Children’s Memorial Health Institute (approval number 45/KBE/2020).

Data sharing statement

The data sets generated and/or analyzed during the current study are available from the corresponding author on reasonable request, to researchers who provide a methodologically sound proposal. The data will be provided after their de-identification, in compliance with applicable privacy laws, data protection and requirements for consent and anonymization.