Thomas Meyer1,2, André Maier1, Zeljko Uzelac3, Tim Hagenacker4, René Günther5,6, Olivia Schreiber-Katz7, Markus Weiler8, Robert Steinbach9, Ute Weyen10, Jan Christoph Koch11, Dagmar Kettemann1, Jenny Norden1, Johannes Dorst3, Claudia Wurster3, Albert C Ludolph3,12, Benjamin Stolte4, Maren Freigang5, Alma Osmanovic7, Susanne Petri7, Julian Grosskreutz9, Annekathrin Rödiger9, Ramona Griep2, Marcel Gaudlitz2, Bertram Walter1, Christoph Münch1,2, Susanne Spittel1,2. 1. Department of Neurology, Center for ALS, SMA and other Motor Neuron Disorders, Charité-Universitätsmedizin Berlin, Berlin, Germany. 2. Ambulanzpartner Soziotechnologie APST GmbH, Berlin, Germany. 3. Department of Neurology, University of Ulm, Ulm, Germany. 4. Department of Neurology, Universitätsklinikum Essen, Essen, Germany. 5. Department of Neurology, Technische Universität Dresden, Universitätsklinikum Carl Gustav Carus, Dresden, Germany. 6. German Center for Neurodegenerative Diseases (DZNE), Research Site Dresden, Dresden, Germany. 7. Department of Neurology, Hannover Medical School, Hannover, Germany. 8. Department of Neurology, Heidelberg University Hospital, Heidelberg, Germany. 9. Hans Berger Department of Neurology, Jena University Hospital, Jena, Germany. 10. Department of Neurology, Center for ALS and other Motor Neuron Disorders, Berufsgenossenschaftliches Universitätsklinikum Bergmannsheil, Bochum, Germany. 11. Department of Neurology, Universitätsmedizin Göttingen, Göttingen, Germany. 12. German Center for Neurodegenerative Diseases (DZNE), Research Site Ulm, Ulm, Germany.
Abstract
BACKGROUND AND PURPOSE: This was an investigation of treatment expectations and of the perception of therapy in adult patients with 5q-associated spinal muscular atrophy (5q-SMA) receiving nusinersen. METHODS: A prospective, non-interventional observational study of nusinersen treatment in adult 5q-SMA patients was conducted at nine SMA centers in Germany. The functional status, treatment expectations and perceived outcomes were assessed using the Amyotrophic Lateral Sclerosis Functional Rating Scale-extended (ALS-FRS-ex), the Measure Yourself Medical Outcome Profile (MYMOP2), the Treatment Satisfaction Questionnaire for Medication (TSQM-9) and the Net Promoter Score (NPS). RESULTS: In all, 151 patients were included with a median age of 36 years (15-69 years). SMA type 3 (n = 90, 59.6%) prevailed, followed by type 2 (33.8%) and type 1 (6.6%). In SMA types 1-3, median ALS-FRS-ex scores were 25, 33 and 46 (of 60 scale points), respectively. MYMOP2 identified distinct treatment expectations: head verticalization (n = 13), bulbar function (n = 16), arm function (n = 65), respiration (n = 15), trunk function (n = 34), leg function (n = 76) and generalized symptoms (n = 77). Median symptom severity decreased during nusinersen treatment (median observational period 6.1 months, 0.5-16 months) from 3.7 to 3.3 MYMOP2 score points (p < 0.001). The convenience of drug administration was critical (49.7 of 100 TSQM-9 points, SD 22); however, the overall treatment satisfaction was high (74.3, SD 18) and the recommendation rating very positive (NPS +66). CONCLUSIONS: Nusinersen was administered across a broad range of ages, disease durations and motor function deficits. Treatment expectations were highly differentiated and related to SMA type and functional status. Patient-reported outcomes demonstrated a positive perception of nusinersen therapy in adult patients with 5q-SMA.
BACKGROUND AND PURPOSE: This was an investigation of treatment expectations and of the perception of therapy in adult patients with 5q-associated spinal muscular atrophy (5q-SMA) receiving nusinersen. METHODS: A prospective, non-interventional observational study of nusinersen treatment in adult 5q-SMA patients was conducted at nine SMA centers in Germany. The functional status, treatment expectations and perceived outcomes were assessed using the Amyotrophic Lateral Sclerosis Functional Rating Scale-extended (ALS-FRS-ex), the Measure Yourself Medical Outcome Profile (MYMOP2), the Treatment Satisfaction Questionnaire for Medication (TSQM-9) and the Net Promoter Score (NPS). RESULTS: In all, 151 patients were included with a median age of 36 years (15-69 years). SMA type 3 (n = 90, 59.6%) prevailed, followed by type 2 (33.8%) and type 1 (6.6%). In SMA types 1-3, median ALS-FRS-ex scores were 25, 33 and 46 (of 60 scale points), respectively. MYMOP2 identified distinct treatment expectations: head verticalization (n = 13), bulbar function (n = 16), arm function (n = 65), respiration (n = 15), trunk function (n = 34), leg function (n = 76) and generalized symptoms (n = 77). Median symptom severity decreased during nusinersen treatment (median observational period 6.1 months, 0.5-16 months) from 3.7 to 3.3 MYMOP2 score points (p < 0.001). The convenience of drug administration was critical (49.7 of 100 TSQM-9 points, SD 22); however, the overall treatment satisfaction was high (74.3, SD 18) and the recommendation rating very positive (NPS +66). CONCLUSIONS: Nusinersen was administered across a broad range of ages, disease durations and motor function deficits. Treatment expectations were highly differentiated and related to SMA type and functional status. Patient-reported outcomes demonstrated a positive perception of nusinersen therapy in adult patients with 5q-SMA.
Authors: Thomas Meyer; Susanne Spittel; André Maier; Marcel Gaudlitz; Torsten Grehl; Ute Weyen; Robert Steinbach; Julian Grosskreutz; Annekathrin Rödiger; Jan Christoph Koch; Teresa Lengenfeld; Patrick Weydt; René Günther; Joachim Wolf; Petra Baum; Moritz Metelmann; Johannes Dorst; Albert C Ludolph; Dagmar Kettemann; Jenny Norden; Ruhan Yasemin Koc; Bertram Walter; Barbara Hildebrandt; Christoph Münch Journal: Sci Rep Date: 2022-06-10 Impact factor: 4.996
Authors: Andreas Thimm; Svenja Brakemeier; Kathrin Kizina; Juan Munoz Rosales; Benjamin Stolte; Andreas Totzeck; Cornelius Deuschl; Christoph Kleinschnitz; Tim Hagenacker Journal: Front Neurol Date: 2022-01-24 Impact factor: 4.003