TY - JOUR
T1 - Efficacy and safety of abobotulinumtoxinA for upper limb spasticity in children with cerebral palsy
T2 - a randomized repeat-treatment study
AU - the Dysport in PUL study group
AU - Delgado, Mauricio R.
AU - Tilton, Ann
AU - Carranza-Del Río, Jorge
AU - Dursun, Nigar
AU - Bonikowski, Marcin
AU - Aydin, Resa
AU - Maciag-Tymecka, Iwona
AU - Oleszek, Joyce
AU - Dabrowski, Edward
AU - Grandoulier, Anne Sophie
AU - Picaut, Philippe
AU - Renders, Anne
AU - Kraus, Josef
AU - Minks, Eduard
AU - Givon, Uri
AU - Sadaka, Yair
AU - Weigl, Daniel
AU - Fattal-Valevski, Aviva
AU - Ben-Pazi, Hilla
AU - Gonzalez, Jose Alberto Moreno
AU - Perez Flores, Elsa Maria Ivon
AU - Jozwiak, Marek
AU - Garreta Figuera, Roser
AU - Alonso Curco, Xenia
AU - Melendez Plumed, Mar
AU - Peker, Ozlen
AU - Phillips, John P.
AU - Revivo, Gadi
AU - Evans, Sarah H.
AU - Wright, Edward A.
AU - Wilson, Jenny Lupovici
AU - Kim, Heakyung
AU - Aylward, Shawn
AU - Gormley, Mark E.
N1 - Funding Information:
The members of the Dysport in PUL study group are as follows: Anne Renders, Josef Kraus, Eduard Minks, Uri Givon, Yair Sadaka, Daniel Weigl, Aviva Fattal‐Valevski, Hilla Ben‐Pazi, Jorge Carranza‐del Río, Jose Alberto Moreno Gonzalez, Elsa Maria Ivon Perez Flores, Marcin Bonikowski, Marek Jozwiak, Roser Garreta Figuera, Xenia Alonso Curco, Mar Melendez Plumed, Nigar Dursun, Resa Aydin, Ozlen Peker, Mauricio R Delgado, Joyce Oleszek, Ann Tilton, John P Phillips, Gadi Revivo, Sarah H Evans, Edward A Wright, Jenny Lupovici Wilson, Edward Dabrowski, Heakyung Kim, Shawn Aylward, Mark E Gormley. The authors thank all children and their families involved in the study, as well as the investigators and research staff in participating institutions. We also thank Anita Chadha‐Patel, PhD, of ACP Clinical Communications Ltd (Hertfordshire, UK) for providing medical writing support, which was funded by Ipsen (Paris, France) in accordance with Good Publication Practice guidelines.
Funding Information:
MRD, AT, JCR, ND, MB, RA, IM‐T, JO, and ED were investigators in Ipsen‐sponsored clinical trials, and they or their institutions received payment for participation. In addition, MRD reports personal fees from Ipsen, Allergan, and Kashiv Pharma for consultancy. AT reports research support and educational grants from Ipsen, and personal fees for consultancy from Ipsen. JCR reports personal fees for consultancy and speaking from Ipsen. ND reports research support from Ipsen, Allergan, and Merz, and personal fees for consultancy and speaking from Ipsen and Allergan. MB reports research support from Ipsen, Allergan, and Merz, and personal fees for consultancy and speaking from Ipsen and Allergan. RA and IM‐T have nothing further to report. JO reports consultancy fees for Ipsen and Allergan. ED reports personal fees from Ipsen and Allergan for speaking, Solstice Neurosciences for consultancy, and serves on a US speaker bureau. PP was employed by Ipsen at the time of study.
PY - 2020
Y1 - 2020
N2 - Aim: To assess the efficacy and safety of repeat abobotulinumtoxinA injections in reducing upper limb spasticity in children with cerebral palsy (CP). Method: This was a double-blind, repeat-cycle study (NCT02106351) in children with CP (2–17y). Children were randomized to receive 2U/kg (control), 8U/kg, or 16U/kg abobotulinumtoxinA injections into the target muscle group (wrist or elbow flexors) and additional muscles alongside occupational therapy via a home-exercise therapy program (HETP; minimum five 15min sessions/wk). Children received 8U/kg or 16U/kg plus HETP in cycles 2 to 4. Results: During cycle 1, 210 children (126 males, 84 females; mean age [SD] 9y [4y 5mo], range 2–17y; n=70/group) had at least one upper limb abobotulinumtoxinA injection and 209 complied with the HETP. At week 6 of cycle 1, children in the 8U/kg or 16U/kg groups had significantly lower Modified Ashworth scale scores versus the 2U/kg group (primary outcome: treatment differences of –0.4 [p=0.012] and –0.7 [p<0.001] respectively). All groups improved on Physician Global Assessment and children in all groups achieved their treatment goals at least as expected. Therapeutic benefits were sustained during cycles 2 to 4; muscular weakness was the only treatment-related adverse event reported in at least one child/group (4.3% and 5.7% vs 1.4% respectively). Interpretation: Treatment with 8U/kg or 16U/kg abobotulinumtoxinA significantly reduced upper limb spasticity versus the 2U/kg control dose. Therapeutic benefits of abobotulinumtoxinA plus HETP were sustained with repeat treatment cycles.
AB - Aim: To assess the efficacy and safety of repeat abobotulinumtoxinA injections in reducing upper limb spasticity in children with cerebral palsy (CP). Method: This was a double-blind, repeat-cycle study (NCT02106351) in children with CP (2–17y). Children were randomized to receive 2U/kg (control), 8U/kg, or 16U/kg abobotulinumtoxinA injections into the target muscle group (wrist or elbow flexors) and additional muscles alongside occupational therapy via a home-exercise therapy program (HETP; minimum five 15min sessions/wk). Children received 8U/kg or 16U/kg plus HETP in cycles 2 to 4. Results: During cycle 1, 210 children (126 males, 84 females; mean age [SD] 9y [4y 5mo], range 2–17y; n=70/group) had at least one upper limb abobotulinumtoxinA injection and 209 complied with the HETP. At week 6 of cycle 1, children in the 8U/kg or 16U/kg groups had significantly lower Modified Ashworth scale scores versus the 2U/kg group (primary outcome: treatment differences of –0.4 [p=0.012] and –0.7 [p<0.001] respectively). All groups improved on Physician Global Assessment and children in all groups achieved their treatment goals at least as expected. Therapeutic benefits were sustained during cycles 2 to 4; muscular weakness was the only treatment-related adverse event reported in at least one child/group (4.3% and 5.7% vs 1.4% respectively). Interpretation: Treatment with 8U/kg or 16U/kg abobotulinumtoxinA significantly reduced upper limb spasticity versus the 2U/kg control dose. Therapeutic benefits of abobotulinumtoxinA plus HETP were sustained with repeat treatment cycles.
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U2 - 10.1111/dmcn.14733
DO - 10.1111/dmcn.14733
M3 - Article
C2 - 33206382
AN - SCOPUS:85096717960
JO - Developmental Medicine and Child Neurology
JF - Developmental Medicine and Child Neurology
SN - 0012-1622
ER -