Plain language summary: How safe and effective is long-term treatment with intravenous plasminogen concentrate for...

What is this study about? Type 1 plasminogen deficiency (PLGD-1 for short) is a long-term, ultra-rare condition where the body does not make enough of a protein called plasminogen. This can cause thick layers made of a fibrous, insoluble protein called fibrin (known as lesions) to develop in different parts of the body. Intravenous plasminogen concentrate (IV PLG concentrate for short, also known as RYPLAZIM®) is the first medicine approved to treat this condition. It works by replacing plasminogen in the body. In this study, adults and children with PLGD-1 received long-term treatment with IV PLG concentrate. They had already received IV PLG concentrate before, as part of the clinical trial that led to the approval of the treatment, or through the expanded access programs. The researchers wanted to know how safe and effective long-term treatment with IV PLG concentrate was. They also wanted to explore whether giving IV PLG concentrate more or less often would affect how well the treatment worked. What were the results? Study participants had no new lesions associated with PLGD-1 or return of previous lesions when they received the treatment as prescribed. Some participants had previous lesions come back when they received the treatment less often than prescribed. These lesions rapidly improved or went away again when they started more frequent treatment, as originally prescribed. Most of the adverse events that participants experienced were classified as mild, and none were related to the study treatment. What do the results mean? Overall, long-term treatment with IV PLG concentrate is effective for people living with PLGD-1 and is well tolerated. Treatment with IV PLG concentrate can be personalized to meet people's individual needs. What is the purpose of this plain language summary? The purpose of this plain language summary is to help you understand the findings from recent research. Where can I find the original article on which this summary is based? You can read the original article titled 'Safety and efficacy of long-term treatment of type 1 plasminogen deficient patients with intravenous plasminogen replacement therapy' published in the journal Haemophilia for free at: https://doi.org/10.1111/hae.70019 Who sponsored the study and this summary? The study was sponsored by Prometic Biotherapeutics Inc., currently Kedrion Biopharma Inc. This summary was sponsored by Kedrion Biopharma Inc. Who is this summary for? This summary may be of interest to people living with PLGD-1, their families, and caregivers. Patient advocates, patient organizations, healthcare professionals, and funders of healthcare services may also find this summary useful.