Intrathecal baclofen treatment in children with neurological disorders
Bonouvrié, L.A.
2019
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Bonouvrié, L. A. (2019). Intrathecal baclofen treatment in children with neurological disorders.
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SUMMARY
DŽǀĞŵĞŶƚĚŝƐŽƌĚĞƌƐŝŶĐŚŝůĚŚŽŽĚĂƌĞŵŽƐƚůLJĐĂƵƐĞĚďLJĚLJƐĨƵŶĐƟŽŶŽĨƚŚĞĚĞǀĞůŽƉŝŶŐ ďƌĂŝŶ ĚƵĞ ƚŽ ďƌĂŝŶ ůĞƐŝŽŶƐ Žƌ ďƌĂŝŶ ĂďŶŽƌŵĂůŝƟĞƐ͘ dŚĞ /ŶƚĞƌŶĂƟŽŶĂů ůĂƐƐŝĮĐĂƟŽŶ ŽĨ &ƵŶĐƟŽŶŝŶŐ͕ ĚŝƐĂďŝůŝƚLJ ĂŶĚ ŚĞĂůƚŚ ĨŽƌ ŚŝůĚƌĞŶ ĂŶĚ zŽƵƚŚ ;/&ͲzͿ ĐĂŶ ďĞ ƵƐĞĚ ĂƐĂĨƌĂŵĞǁŽƌŬƚŽĐĂƚĞŐŽƌŝnjĞƉĞƌƐŽŶĂůŝnjĞĚƚƌĞĂƚŵĞŶƚŐŽĂůƐ͘dŚĞ/&ͲzƐŚŽǁƐƚŚĂƚ ŝŵƉĂŝƌŵĞŶƚƐŽŶƚŚĞůĞǀĞůŽĨďŽĚLJĨƵŶĐƟŽŶƐĂŶĚƐƚƌƵĐƚƵƌĞƐ;Ğ͘Ő͘ƐƉĂƐƟĐŝƚLJŽƌĚLJƐŬŝŶĞƐŝĂͿ ĐĂŶůĞĂĚƚŽƉƌŽďůĞŵƐŝŶĂĐƟǀŝƟĞƐŽĨĚĂŝůLJůŝĨĞĂŶĚƉĂƌƟĐŝƉĂƟŽŶŝŶƐŽĐŝĞƚLJƐƵĐŚĂƐŵŽďŝůŝƚLJ͕ ƐĞůĨͲĐĂƌĞ͕ĐŽŵŵƵŶŝĐĂƟŽŶĂŶĚůĞĂƌŶŝŶŐ͘ĐƟǀŝƟĞƐĂŶĚƉĂƌƟĐŝƉĂƟŽŶĂƌĞĨƵƌƚŚĞƌŵŽƌĞ ŝŶŇƵĞŶĐĞĚďLJĞŶǀŝƌŽŶŵĞŶƚĂůĨĂĐƚŽƌƐŝŶĐůƵĚŝŶŐƚŚĞĂǀĂŝůĂďŝůŝƚLJŽĨĂƐƐŝƐƚĂŶĐĞĨŽƌƉĞƌƐŽŶĂů care and aids for mobility.The spectrum of the severity of cerebral movement disorders is broad. This thesis will ĨŽĐƵƐŽŶƐĞǀĞƌĞůLJĂīĞĐƚĞĚĐŚŝůĚƌĞŶ͗ĐŚŝůĚƌĞŶǁŝƚŚĐĞƌĞďƌĂůƉĂůƐLJ;WͿĐůĂƐƐŝĮĞĚǁŝƚŚƚŚĞ 'ƌŽƐƐDŽƚŽƌ&ƵŶĐƟŽŶŝŶŐůĂƐƐŝĮĐĂƟŽŶ^LJƐƚĞŵ;'D&^ͿŝŶůĞǀĞůƐ/sĂŶĚsĂŶĚĐŚŝůĚƌĞŶ ǁŝƚŚƉƌŽŐƌĞƐƐŝǀĞŶĞƵƌŽůŽŐŝĐĂůĚŝƐŽƌĚĞƌƐ;WEͿǁŚŽĂƌĞĞƋƵĂůůLJĂīĞĐƚĞĚ͘dŚĞƐĞĐŚŝůĚƌĞŶ ĂƌĞ ŶŽƚ ĂďůĞ ƚŽ ǁĂůŬ ƵŶĂƐƐŝƐƚĞĚ Žƌ ĚŽ ŶŽƚ ǁĂůŬ Ăƚ Ăůů͕ ĂŶĚ ŵĂŝŶůLJ ƵƐĞ Ă ;ƉŽǁĞƌĞĚͿ wheelchair for mobility.
The most common cause of cerebral movement disorders and physical disability ŝŶĐŚŝůĚŚŽŽĚŝƐW͘^ƉĂƐƟĐĂŶĚĚLJƐŬŝŶĞƟĐWĂƌĞƚŚĞƚǁŽŵŽƐƚĐŽŵŵŽŶŵŽǀĞŵĞŶƚ ĚŝƐŽƌĚĞƌƐ;ϳϮͲϵϭйĂŶĚĂƉƉƌŽdžŝŵĂƚĞůLJϭϱйŽĨWƌĞƐƉĞĐƟǀĞůLJͿ͘^ƉĂƐƟĐĂŶĚĚLJƐŬŝŶĞƟĐ ŵŽǀĞŵĞŶƚĚŝƐŽƌĚĞƌƐĐĂŶĂůƐŽďĞĐĂƵƐĞĚďLJWE͘dŚĞƌĞĂƌĞŵĂŶLJĚŝīĞƌĞŶƚĚŝĂŐŶŽƐĞƐ ƌĞůĂƚĞĚƚŽWEŝŶĐŚŝůĚŚŽŽĚ͕ĂůůŽĨƚŚĞŵƌĂƌĞ͘ tŚĞŶƐƉĂƐƟĐŝƚLJŽƌĚLJƐƚŽŶŝĂŝƐƐĞǀĞƌĞĂŶĚŝŶƚĞƌĨĞƌĞƐǁŝƚŚĐŽŵĨŽƌƚ͕ƋƵĂůŝƚLJŽĨůŝĨĞŽƌ ĂĐƟǀŝƟĞƐŽĨĚĂŝůLJůŝĨĞ͕ƚŚĞĮƌƐƚƐƚĞƉŝƐŽƌĂůƉŚĂƌŵĂĐŽůŽŐŝĐĂůƚƌĞĂƚŵĞŶƚ͘tŚĞŶƚŚŝƐŝƐ ŝŶƐƵĸĐŝĞŶƚ͕ƚŚĞŶĞdžƚƐƚĞƉƐĂƌĞĂĚǀĂŶĐĞĚƚƌĞĂƚŵĞŶƚŽƉƟŽŶƐ͘KŶĞŽĨƚŚĞƐĞŽƉƟŽŶƐŝƐ ŝŶƚƌĂƚŚĞĐĂůďĂĐůŽĨĞŶ;/dͿ͘tŝƚŚ/d͕ďĂĐůŽĨĞŶŝƐĂĚŵŝŶŝƐƚĞƌĞĚŝŶƚƌĂƚŚĞĐĂůůLJƵƐŝŶŐĂŶ ŝŵƉůĂŶƚĞĚŵŝĐƌŽͲŝŶĨƵƐŝŽŶƉƵŵƉ;DĞĚƚƌŽŶŝĐ^LJŶĐŚƌŽŵĞĚ//Ϳ͘dŚĞƉƵŵƉŝƐŝŵƉůĂŶƚĞĚ ƐƵďĐƵƚĂŶĞŽƵƐůLJ͕ŵŽƐƚůLJŝŶƚŚĞůĞŌůŽǁĞƌĂďĚŽŵĞŶ͘ĐĂƚŚĞƚĞƌĐŽŶŶĞĐƚƐƚŚĞƉƵŵƉǁŝƚŚ the intrathecal space.
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dŚĞ /ŶƚƌĂƚŚĞĐĂů ďĂĐůŽĨĞŶ ŝŶ LJƐŬŝŶĞƟĐ ĐĞƌĞďƌĂů ƉĂůƐLJ ;/z^Ϳ ƚƌŝĂů ŝƐ Ă ŵƵůƟͲĐĞŶƚƌĞ͕ randomised, double-blind, placebo-controlled trial with the aim to provide evidence ĨŽƌƚŚĞĞīĞĐƚŽĨ/dŝŶĚLJƐŬŝŶĞƟĐW͘dŚĞƐƚƵĚLJƉƌŽƚŽĐŽůŝƐƉƌĞƐĞŶƚĞĚŝŶchapter 4. WĂƟĞŶƚƐǁŝƚŚĚLJƐŬŝŶĞƟĐW͕'D&^/sĂŶĚs͕ǁŚŽĂƌĞĞůŝŐŝďůĞĨŽƌ/dƚƌĞĂƚŵĞŶƚǁĞƌĞ ŝŶĐůƵĚĞĚ͘dŚĞLJǁĞƌĞĂƐƐŝŐŶĞĚďLJďůŽĐŬĞĚƌĂŶĚŽŵŝnjĂƟŽŶ;Ϯ͗ϮͿ͕ƚŽƌĞĐĞŝǀĞĞŝƚŚĞƌƉůĂĐĞďŽ Žƌ/dĨŽƌϯŵŽŶƚŚƐǀŝĂĂŶŝŵƉůĂŶƚĞĚŵŝĐƌŽͲŝŶĨƵƐŝŽŶƉƵŵƉ͘ŌĞƌƚŚƌĞĞŵŽŶƚŚƐ͕ŽƵƚĐŽŵĞ ŵĞĂƐƵƌĞƐǁĞƌĞĂƐƐĞƐƐĞĚĂŶĚĂůůƉĂƟĞŶƚƐĐŽŶƟŶƵĞĚŽŶ/dƚŚĞƌĞĂŌĞƌ͘dŚĞƉƌŝŵĂƌLJ ŽƵƚĐŽŵĞ ŵĞĂƐƵƌĞŵĞŶƚ ǁĂƐ ĂƩĂŝŶŵĞŶƚ ŽĨ ŝŶĚŝǀŝĚƵĂů ƚƌĞĂƚŵĞŶƚ ŐŽĂůƐ ƵƐŝŶŐ 'ŽĂů ƩĂŝŶŵĞŶƚ^ĐĂůŝŶŐ;'^Ϳ͘^ĞĐŽŶĚĂƌLJŽƵƚĐŽŵĞŵĞĂƐƵƌĞƐŝŶĐůƵĚĞĚĚLJƐƚŽŶŝĂ;^ĂŶĚ LJƐŬŝŶĞƐŝĂ/ŵƉĂŝƌŵĞŶƚ^ĐĂůĞ;/^ͿͿ͕ƐƉĂƐƟĐŝƚLJ;ƐƉĂƐƟĐŝƚLJƚĞƐƚ;^WdͿĂŶĚ,ŽīŵĂŶŶƌĞŇĞdž ;ŚͲƌĞŇĞdžͿͿ͕ƌĂŶŐĞŽĨŵŽƟŽŶ;ZKDͿ͕ƉĂŝŶ;s^Ϳ͕ĐŽŵĨŽƌƚ;s^ͿĂŶĚƚŚĞĐŚĂŶŐĞŝŶƌŝƐŬ ŽĨƐůĞĞƉͲƌĞůĂƚĞĚďƌĞĂƚŚŝŶŐĚŝƐŽƌĚĞƌƐ;ƋƵĞƐƟŽŶŶĂŝƌĞͿ͘ĚǀĞƌƐĞĞǀĞŶƚƐǁĞƌĞŵŽŶŝƚŽƌĞĚ͘ WŽǁĞƌĐĂůĐƵůĂƟŽŶƐǁĞƌĞĚŽŶĞĂŶĚĂƚŽƚĂůŽĨϭϯƉĂƟĞŶƚƐƉĞƌŐƌŽƵƉǁŽƵůĚƉƌŽǀŝĚĞ enough power.
dŚĞƌĞƐƵůƚƐŽĨƚŚĞ/z^ƚƌŝĂůĂƌĞĚĞƐĐƌŝďĞĚŝŶchapter 5. To prevent the study from ďĞĐŽŵŝŶŐƵŶĚĞƌƉŽǁĞƌĞĚĚƵĞƚŽĐŽŵƉůŝĐĂƟŽŶƐ͕ϭϴƉĂƟĞŶƚƐƉĞƌŐƌŽƵƉǁĞƌĞŝŶĐůƵĚĞĚ͘ ĂƚĂĨŽƌĮŶĂů'^ĂŶĂůLJƐŝƐǁĞƌĞĂǀĂŝůĂďůĞĨŽƌϭϲƉĂƟĞŶƚƐŝŶƚŚĞƉůĂĐĞďŽŐƌŽƵƉĂŶĚ ϭϳŝŶƚŚĞ/dŐƌŽƵƉ͘dŚĞŵĞĂŶ'^dͲƐĐŽƌĞƐĂƚƚŚƌĞĞŵŽŶƚŚƐǁĞƌĞƐŝŐŶŝĮĐĂŶƚůLJŵŽƌĞ ĨĂǀŽƵƌĂďůĞĨŽƌ/d͕ĐŽŵƉĂƌĞĚƚŽƉůĂĐĞďŽ͘dŚĞ/^ĚLJƐƚŽŶŝĂƐƵďƐĐŽƌĞĂŶĚĚLJƐƚŽŶŝĂƌĞƐƚ ƐƵďƐĐŽƌĞƐŚŽǁĞĚĂƐŝŐŶŝĮĐĂŶƚĚŝīĞƌĞŶĐĞŝŶĨĂǀŽƵƌŽĨ/d͘dŚŝƐĚŝīĞƌĞŶĐĞǁĂƐĐĂƵƐĞĚďLJ an increase of dystonia in the placebo group, compared to no change in the ITB group. KŶƚŚĞŽƚŚĞƌƐĞĐŽŶĚĂƌLJŽƵƚĐŽŵĞŵĞĂƐƵƌĞƐ͕ŶŽĚŝīĞƌĞŶĐĞďĞƚǁĞĞŶƉůĂĐĞďŽĂŶĚ/dǁĂƐ ĨŽƵŶĚ͘EƵŵďĞƌĂŶĚƚLJƉĞƐŽĨ;ƐĞƌŝŽƵƐͿĂĚǀĞƌƐĞĞǀĞŶƚƐǁĞƌĞƐŝŵŝůĂƌďĞƚǁĞĞŶŐƌŽƵƉƐ͘tŝƚŚ ƚŚĞ/z^ƚƌŝĂů͕ǁĞƉƌŽǀŝĚĞŚŝŐŚůĞǀĞůŽĨĞǀŝĚĞŶĐĞĨŽƌƚŚĞĞīĞĐƚŽĨ/dŽŶƚŚĞĂƩĂŝŶŵĞŶƚ ŽĨŝŶĚŝǀŝĚƵĂůƚƌĞĂƚŵĞŶƚŐŽĂůƐ͘ƵƌƌĞŶƚĐůŝŶŝĐĂůŽďƐĞƌǀĂƟŽŶƐĐĂůĞƐƚŽƌĂƚĞĚLJƐƚŽŶŝĂĂƌĞ limited in their use due to unknown test-retest reliability. More reliable measures are ŶĞĞĚĞĚƚŽŵĞĂƐƵƌĞĐŚĂŶŐĞƐŝŶĚLJƐƚŽŶŝĂŝŶƉĂƟĞŶƚƐŽŶ/d͘
