- Results from the largest European and first multicenter, retrospective, observational chart review study investigating the real-world use of ECP in heart transplant patients reinforce its use as a treatment for heart transplant rejection and prevention of rejection1 -
DUBLIN, June 13, 2023 /PRNewswire/ -- Mallinckrodt plc (NYSE American: MNK), a global specialty pharmaceutical company, today announced the publication of findings from a retrospective, observational, single arm, European chart review study assessing the real-world use of extracorporeal photopheresis (ECP) and its impact on clinical outcomes in the modern era of heart transplantation.1 An online version of the data manuscript – the largest-known study of ECP in heart transplant patients – is currently published on the Journal of Heart and Lung Transplantation website in advance of print publication in the second half of 2023.
Interim results of this study were presented in a late-breaking session at the 20th Congress of the European Society for Organ Transplantation (ESOT) in 2021 in Milan, Italy.2
The study, titled "European Multicenter Study on The Real-World Use and Clinical Impact of Extracorporeal Photopheresis After Heart Transplantation," examined data from the medical charts of 105 patients who received ECP following heart transplantation at seven medical centers in Austria, Germany, France, Hungary, and Italy between 2015 – 2021. At time of data extraction, 58 patients (55.2%) had completed their ECP treatment and 47 patients' (44.8%) ECP treatment was ongoing.1
"These findings from the largest European and first multicenter study investigating the real-world use of ECP in heart transplant patients support ECP as a treatment for various types of graft rejection and in prevention of graft rejection with varied treatment schedules,1" said Markus Barten, M.D., Surgical Director of Heart Failure Clinic, University Heart and Vascular Center Hamburg. "This data not only builds upon the growing body of real-world evidence supporting the use of ECP in heart transplant patients, but also reflects the importance of supporting clinicians with treatment modalities for transplant rejection and stabilization.1"
About the Study1
Mean age of patients at start of ECP was 47.7 (SD 14.4) years (min. 16 years to max. 74 years), and most patients (70.5%) were male. They were followed for a mean time of 25.1 (SD 16.8) months from ECP treatment initiation to last visit at the transplant center (follow-up time for outcome overall survival). Mean time from ECP treatment initiation to last visit right censored at 2 years after the end of ECP treatment was 22.5 (SD 13.7) months (follow-up time for outcomes graft function, response, and complications).
Cardiomyopathy was the primary reason for heart transplantation (n=81 patients; 77.1%), followed by coronary heart disease (n=11 patients; 10.5%), heart valve disease (n=5 patients; 4.8%), and myocarditis (n=5 patients; 4.8%). The main reason to start ECP treatment was acute cellular rejection (ACR; n=37 patients; 35.2%), followed by prevention of rejection (n=34 patients; 32.4%), mixed rejection (n=19 patients; 18.1%), and antibody-mediated rejection (AMR; n=15 patients; 14.3%).
The prevention of rejection subgroup included patients who started ECP treatment without biopsy-proven rejection and with standard or reduced immunosuppressive therapy.
The effectiveness of ECP in comparison with other treatment options was not assessed due to the descriptive, single-arm design of this study. Study limitations include that data generation for this observational study was not standardized. Patient examination schedules varied and not all data were available at all centers. No source data verification was performed and therefore, transmission errors cannot be excluded. Not all demonstrated benefits may be solely attributable to ECP treatment, as transplanted patients may have received multiple therapies at time of ECP treatment. In patients with AMR or mixed rejection, ECP is commonly used in combination with other treatments.
This study was funded by Mallinckrodt.
IMPORTANT SAFETY INFORMATION FOR THE THERAKOS™ PHOTOPHERESIS PROCEDURE
The THERAKOS™ CELLEX™ Photopheresis System is indicated for the administration of photopheresis. Please refer to the appropriate product labelling for a complete list of warnings and precautions.
THERAKOS™ Photopheresis is contraindicated in:
Warnings and Precautions
THERAKOS™ Photopheresis treatments should always be performed in locations where standard medical emergency equipment is available. Volume replacement fluids and/or volume expanders should be readily available throughout the procedure. Safety in children has not been established.
Please refer to the THERAKOS™ CELLEX™ Photopheresis System Operator Manual for a complete list of warnings and precautions.
IMPORTANT SAFETY INFORMATION FOR METHOXSALEN USED IN CONJUNCTION WITH THERAKOS™ PHOTOPHERESIS
Methoxsalen is contraindicated in:
Warnings and Precautions
Refer to the package insert for methoxsalen sterile solution (20 micrograms / mL) or the oral 8-methoxpsoralen dosage formulation for a list of all warnings and precautions.
Please refer to the THERAKOS™ CELLEX™ Photopheresis System Operator Manual for a complete list of warnings and precautions and adverse events.
About Extracorporeal Photopheresis (ECP)
ECP, a blood based immunomodulatory therapy developed more than 30 years ago, is recommended by the International Society for Heart and Lung Transplantation (ISHLT)3 and other clinical societies4,5,6 as an adjunctive therapy for the prevention and treatment of ACR after heart transplantation. Additionally, ECP may be considered to treat AMR with or without donor specific antibodies.7,8 In countries where it is approved, ECP is used to treat a range of immune-mediated diseases, including skin manifestations of cutaneous T-cell lymphoma (CTCL), graft-versus-host disease (GvHD), solid organ transplant rejection and other autoimmune diseases. During ECP treatment, a small amount of white blood cells is collected and treated with a drug that is activated by ultraviolet light.
Mallinckrodt is a global business consisting of multiple wholly owned subsidiaries that develop, manufacture, market and distribute specialty pharmaceutical products and therapies. The company's Specialty Brands reportable segment's areas of focus include autoimmune and rare diseases in specialty areas like neurology, rheumatology, hepatology, nephrology, pulmonology, ophthalmology, and oncology; immunotherapy and neonatal respiratory critical care therapies; analgesics; cultured skin substitutes and gastrointestinal products. Its Specialty Generics reportable segment includes specialty generic drugs and active pharmaceutical ingredients. To learn more about Mallinckrodt, visit www.mallinckrodt.com.
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CAUTIONARY STATEMENTS RELATED TO FORWARD-LOOKING STATEMENTS
This release contains forward-looking statements, including with regard to ECP and its potential impact on patients. The statements are based on assumptions about many important factors, including the following, which could cause actual results to differ materially from those in the forward-looking statements: satisfaction of regulatory and other requirements; actions of regulatory bodies and other governmental authorities; changes in laws and regulations; issues with product quality, manufacturing or supply, or patient safety issues; and other risks identified and described in more detail in the "Risk Factors" section of Mallinckrodt's most recent Annual Report on Form 10-K and other filings with the SEC, all of which are available on its website. The forward-looking statements made herein speak only as of the date hereof and Mallinckrodt does not assume any obligation to update or revise any forward-looking statement, whether as a result of new information, future events and developments or otherwise, except as required by law.
Senior Vice President, Green Room Communications
Daniel J. Speciale
Global Corporate Controller & Chief Investor Relations Officer
Vice President, Investor Relations
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©2023 Mallinckrodt. EU-2300168 06/23
1 Barten, MJ et al. European multi-center study on the real-world use and clinical impact of extracorporeal photopheresis after heart transplantation. J Heart Lung Transplant. 2023. https://doi.org/10.1016/j.healun.2023.03.005.
2 Barten, MJ. et al. Real World Use and Clinical Impact of Extracorporeal Photopheresis in Heart Transplant Patients – Results From a European Multi-Centre Study. Abstract presented at: European Society for Organ Transplantation (ESOT) Congress 2021. August/September 2021.
3 Costanzo MR, et al. The International Society of Heart and Lung Transplantation Guidelines for the care of heart transplant recipients. J Heart Lung Trans. 2010:29(8);914–956.
4 Alfred et al. The role of extracorporeal photopheresis in the management of cutaneous T-cell lymphoma, graft-versus-host disease and organ transplant rejection: a consensus statement update from the UK Photopheresis Society. Br J Haematol. 2017;177(2):287-310.
5 Padmanabhan et al. Guidelines on the Use of Therapeutic Apheresis in Clinical Practice - Evidence-Based Approach from the Writing Committee of the American Society for Apheresis: The Eighth Special Issue. J Clin Apher. 2019;34:171–354.
6 Knobler et al. European dermatology forum - updated guidelines on the use of extracorporeal photopheresis 2020 - part 2. Eur Acad Dermatol Venereol. 2021;35(1):27-49.
7 Colvin et al. Antibody-mediated rejection in cardiac transplantation: emerging knowledge in diagnosis and management: a scientific statement from the American Heart Association. Circulation. 2015;131(18):1608-1639.
8 Barten et al. Transplant Rev (Orlando). The clinical impact of donor-specific antibodies in heart transplantation. 2018;32(4):207-217.
SOURCE Mallinckrodt plc