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Subcutaneous cladribine to treat multiple sclerosis: experience in 208 patients

OBJECTIVE: To report on safety and effectiveness of subcutaneous cladribine (Litak(®)) in multiple sclerosis (MS) patients. METHODS: Litak(®) was offered to MS-patients irrespective of disease course. Litak(®) 10 mg was administered for 3–4 days during week 1. Based on lymphocyte count at week 4, pa...

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Autores principales: Allen-Philbey, Kimberley, De Trane, Stefania, Mao, Zhifeng, Álvarez-González, Cesar, Mathews, Joela, MacDougall, Amy, Stennett, Andrea, Zhou, Xia, Yildiz, Ozlem, Adams, Ashok, Bianchi, Lucia, Blain, Camilla, Chapman, Christine, Chung, Karen, Constantinescu, Cris S, Dalton, Catherine, Farrell, Rachel A, Fisniku, Leonora, Ford, Helen, Gran, Bruno, Hobart, Jeremy, Khaleeli, Zhaleh, Mattoscio, Miriam, Pavitt, Sue, Pearson, Owen, Peruzzotti-Jametti, Luca, Scalfari, Antonio, Sharrack, Basil, Silber, Eli, Tallantyre, Emma C, Webb, Stewart, Turner, Benjamin P, Marta, Monica, Gnanapavan, Sharmilee, Juliusson, Gunnar, Giovannoni, Gavin, Baker, David, Schmierer, Klaus
Formato: Online Artículo Texto
Lenguaje:English
Publicado: SAGE Publications 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8842147/
https://www.ncbi.nlm.nih.gov/pubmed/35173808
http://dx.doi.org/10.1177/17562864211057661
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author Allen-Philbey, Kimberley
De Trane, Stefania
Mao, Zhifeng
Álvarez-González, Cesar
Mathews, Joela
MacDougall, Amy
Stennett, Andrea
Zhou, Xia
Yildiz, Ozlem
Adams, Ashok
Bianchi, Lucia
Blain, Camilla
Chapman, Christine
Chung, Karen
Constantinescu, Cris S
Dalton, Catherine
Farrell, Rachel A
Fisniku, Leonora
Ford, Helen
Gran, Bruno
Hobart, Jeremy
Khaleeli, Zhaleh
Mattoscio, Miriam
Pavitt, Sue
Pearson, Owen
Peruzzotti-Jametti, Luca
Scalfari, Antonio
Sharrack, Basil
Silber, Eli
Tallantyre, Emma C
Webb, Stewart
Turner, Benjamin P
Marta, Monica
Gnanapavan, Sharmilee
Juliusson, Gunnar
Giovannoni, Gavin
Baker, David
Schmierer, Klaus
author_facet Allen-Philbey, Kimberley
De Trane, Stefania
Mao, Zhifeng
Álvarez-González, Cesar
Mathews, Joela
MacDougall, Amy
Stennett, Andrea
Zhou, Xia
Yildiz, Ozlem
Adams, Ashok
Bianchi, Lucia
Blain, Camilla
Chapman, Christine
Chung, Karen
Constantinescu, Cris S
Dalton, Catherine
Farrell, Rachel A
Fisniku, Leonora
Ford, Helen
Gran, Bruno
Hobart, Jeremy
Khaleeli, Zhaleh
Mattoscio, Miriam
Pavitt, Sue
Pearson, Owen
Peruzzotti-Jametti, Luca
Scalfari, Antonio
Sharrack, Basil
Silber, Eli
Tallantyre, Emma C
Webb, Stewart
Turner, Benjamin P
Marta, Monica
Gnanapavan, Sharmilee
Juliusson, Gunnar
Giovannoni, Gavin
Baker, David
Schmierer, Klaus
author_sort Allen-Philbey, Kimberley
collection PubMed
description OBJECTIVE: To report on safety and effectiveness of subcutaneous cladribine (Litak(®)) in multiple sclerosis (MS) patients. METHODS: Litak(®) was offered to MS-patients irrespective of disease course. Litak(®) 10 mg was administered for 3–4 days during week 1. Based on lymphocyte count at week 4, patients received another 0–3 doses at week 5. A second course was administered 11 months later. Follow-up included adverse events, relapses, expanded disability status scale (EDSS), 9-hole-peg and Timed-25-foot-walking tests, no-evidence-of-disease-activity (NEDA), no-evidence-of-progression-or-active-disease (NEPAD), MRI, cerebrospinal fluid (CSF) neurofilament light chain (NfL), and lymphocyte counts. RESULTS: In all, 208 patients received at least one course of treatment. Age at baseline was 44 (17–72) years and EDSS 0–8.5. Cladribine was generally well tolerated. One myocardial infarction, one breast cancer, and three severe skin reactions occurred without long-term sequelae. Two patients died (one pneumonia, one encephalitis). Lymphopenia grade 3 occurred in 5% and grade 4 in 0.5%. In 94 out of 116 pwMS with baseline and follow-up (BaFU) data after two treatment courses, EDSS remained stable or improved. At 18 months, 64% of patients with relapsing MS and BaFU data (n = 39) had NEDA. At 19 months, 62% of patients with progressive MS and BaFU data (n = 13) had NEPAD. Of n = 13 patients whose CSF-NfL at baseline was elevated, 77% were normalised within 12 months. CONCLUSIONS: Litak(®) was well tolerated. Effectiveness in relapsing MS appeared similar to cladribine tablets and was encouraging in progressive MS. Our data suggest cladribine may be safe and effective in MS-patients irrespective of their disease stage.
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spelling pubmed-88421472022-02-15 Subcutaneous cladribine to treat multiple sclerosis: experience in 208 patients Allen-Philbey, Kimberley De Trane, Stefania Mao, Zhifeng Álvarez-González, Cesar Mathews, Joela MacDougall, Amy Stennett, Andrea Zhou, Xia Yildiz, Ozlem Adams, Ashok Bianchi, Lucia Blain, Camilla Chapman, Christine Chung, Karen Constantinescu, Cris S Dalton, Catherine Farrell, Rachel A Fisniku, Leonora Ford, Helen Gran, Bruno Hobart, Jeremy Khaleeli, Zhaleh Mattoscio, Miriam Pavitt, Sue Pearson, Owen Peruzzotti-Jametti, Luca Scalfari, Antonio Sharrack, Basil Silber, Eli Tallantyre, Emma C Webb, Stewart Turner, Benjamin P Marta, Monica Gnanapavan, Sharmilee Juliusson, Gunnar Giovannoni, Gavin Baker, David Schmierer, Klaus Ther Adv Neurol Disord Original Research OBJECTIVE: To report on safety and effectiveness of subcutaneous cladribine (Litak(®)) in multiple sclerosis (MS) patients. METHODS: Litak(®) was offered to MS-patients irrespective of disease course. Litak(®) 10 mg was administered for 3–4 days during week 1. Based on lymphocyte count at week 4, patients received another 0–3 doses at week 5. A second course was administered 11 months later. Follow-up included adverse events, relapses, expanded disability status scale (EDSS), 9-hole-peg and Timed-25-foot-walking tests, no-evidence-of-disease-activity (NEDA), no-evidence-of-progression-or-active-disease (NEPAD), MRI, cerebrospinal fluid (CSF) neurofilament light chain (NfL), and lymphocyte counts. RESULTS: In all, 208 patients received at least one course of treatment. Age at baseline was 44 (17–72) years and EDSS 0–8.5. Cladribine was generally well tolerated. One myocardial infarction, one breast cancer, and three severe skin reactions occurred without long-term sequelae. Two patients died (one pneumonia, one encephalitis). Lymphopenia grade 3 occurred in 5% and grade 4 in 0.5%. In 94 out of 116 pwMS with baseline and follow-up (BaFU) data after two treatment courses, EDSS remained stable or improved. At 18 months, 64% of patients with relapsing MS and BaFU data (n = 39) had NEDA. At 19 months, 62% of patients with progressive MS and BaFU data (n = 13) had NEPAD. Of n = 13 patients whose CSF-NfL at baseline was elevated, 77% were normalised within 12 months. CONCLUSIONS: Litak(®) was well tolerated. Effectiveness in relapsing MS appeared similar to cladribine tablets and was encouraging in progressive MS. Our data suggest cladribine may be safe and effective in MS-patients irrespective of their disease stage. SAGE Publications 2021-11-25 /pmc/articles/PMC8842147/ /pubmed/35173808 http://dx.doi.org/10.1177/17562864211057661 Text en © The Author(s), 2021 https://creativecommons.org/licenses/by-nc/4.0/This article is distributed under the terms of the Creative Commons Attribution-NonCommercial 4.0 License (https://creativecommons.org/licenses/by-nc/4.0/) which permits non-commercial use, reproduction and distribution of the work without further permission provided the original work is attributed as specified on the SAGE and Open Access pages (https://us.sagepub.com/en-us/nam/open-access-at-sage).
spellingShingle Original Research
Allen-Philbey, Kimberley
De Trane, Stefania
Mao, Zhifeng
Álvarez-González, Cesar
Mathews, Joela
MacDougall, Amy
Stennett, Andrea
Zhou, Xia
Yildiz, Ozlem
Adams, Ashok
Bianchi, Lucia
Blain, Camilla
Chapman, Christine
Chung, Karen
Constantinescu, Cris S
Dalton, Catherine
Farrell, Rachel A
Fisniku, Leonora
Ford, Helen
Gran, Bruno
Hobart, Jeremy
Khaleeli, Zhaleh
Mattoscio, Miriam
Pavitt, Sue
Pearson, Owen
Peruzzotti-Jametti, Luca
Scalfari, Antonio
Sharrack, Basil
Silber, Eli
Tallantyre, Emma C
Webb, Stewart
Turner, Benjamin P
Marta, Monica
Gnanapavan, Sharmilee
Juliusson, Gunnar
Giovannoni, Gavin
Baker, David
Schmierer, Klaus
Subcutaneous cladribine to treat multiple sclerosis: experience in 208 patients
title Subcutaneous cladribine to treat multiple sclerosis: experience in 208 patients
title_full Subcutaneous cladribine to treat multiple sclerosis: experience in 208 patients
title_fullStr Subcutaneous cladribine to treat multiple sclerosis: experience in 208 patients
title_full_unstemmed Subcutaneous cladribine to treat multiple sclerosis: experience in 208 patients
title_short Subcutaneous cladribine to treat multiple sclerosis: experience in 208 patients
title_sort subcutaneous cladribine to treat multiple sclerosis: experience in 208 patients
topic Original Research
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8842147/
https://www.ncbi.nlm.nih.gov/pubmed/35173808
http://dx.doi.org/10.1177/17562864211057661
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