Response to second treatment after initial failed treatment in a multicenter prospective infantile spasms cohort

the Pediatric Epilepsy Research Consortium

Research output: Contribution to journalArticle

17 Citations (Scopus)

Abstract

Objective: Infantile spasms (IS) represent a severe epileptic encephalopathy presenting in the first 2 years of life. Recommended first-line therapies (hormonal therapy or vigabatrin) often fail. We evaluated response to second treatment for IS in children in whom the initial therapy failed to produce both clinical remission and electrographic resolution of hypsarhythmia and whether time to treatment was related to outcome. Methods: The National Infantile Spasms Consortium established a multicenter, prospective database enrolling infants with new diagnosis of IS. Children were considered nonresponders to first treatment if there was no clinical remission or persistence of hypsarhythmia. Treatment was evaluated as hormonal therapy (adrenocorticotropic hormone [ACTH] or oral corticosteroids), vigabatrin, or “other.” Standard treatments (hormonal and vigabatrin) were compared to all other nonstandard treatments. We compared response rates using chi-square tests and multivariable logistic regression models. Results: One hundred eighteen infants were included from 19 centers. Overall response rate to a second treatment was 37% (n = 44). Children who received standard medications with differing mechanisms for first and second treatment had higher response rates than other sequences (27/49 [55%] vs. 17/69 [25%], p < 0.001). Children receiving first treatment within 4 weeks of IS onset had a higher response rate to second treatment than those initially treated later (36/82 [44%] vs. 8/34 [24%], p = 0.040). Significance: Greater than one third of children with IS will respond to a second medication. Choosing a standard medication (ACTH, oral corticosteroids, or vigabatrin) that has a different mechanism of action appears to be more effective. Rapid initial treatment increases the likelihood of response to the second treatment.

Original languageEnglish (US)
Pages (from-to)1834-1842
Number of pages9
JournalEpilepsia
Volume57
Issue number11
DOIs
StatePublished - Nov 1 2016

Fingerprint

Infantile Spasms
Vigabatrin
Therapeutics
Adrenocorticotropic Hormone
Adrenal Cortex Hormones
Logistic Models
Brain Diseases
Chi-Square Distribution

Keywords

  • Adrenocorticotropic hormone
  • Infantile spasms
  • Second-line treatment
  • Vigabatrin

ASJC Scopus subject areas

  • Neurology
  • Clinical Neurology

Cite this

Response to second treatment after initial failed treatment in a multicenter prospective infantile spasms cohort. / the Pediatric Epilepsy Research Consortium.

In: Epilepsia, Vol. 57, No. 11, 01.11.2016, p. 1834-1842.

Research output: Contribution to journalArticle

the Pediatric Epilepsy Research Consortium. / Response to second treatment after initial failed treatment in a multicenter prospective infantile spasms cohort. In: Epilepsia. 2016 ; Vol. 57, No. 11. pp. 1834-1842.
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abstract = "Objective: Infantile spasms (IS) represent a severe epileptic encephalopathy presenting in the first 2 years of life. Recommended first-line therapies (hormonal therapy or vigabatrin) often fail. We evaluated response to second treatment for IS in children in whom the initial therapy failed to produce both clinical remission and electrographic resolution of hypsarhythmia and whether time to treatment was related to outcome. Methods: The National Infantile Spasms Consortium established a multicenter, prospective database enrolling infants with new diagnosis of IS. Children were considered nonresponders to first treatment if there was no clinical remission or persistence of hypsarhythmia. Treatment was evaluated as hormonal therapy (adrenocorticotropic hormone [ACTH] or oral corticosteroids), vigabatrin, or “other.” Standard treatments (hormonal and vigabatrin) were compared to all other nonstandard treatments. We compared response rates using chi-square tests and multivariable logistic regression models. Results: One hundred eighteen infants were included from 19 centers. Overall response rate to a second treatment was 37{\%} (n = 44). Children who received standard medications with differing mechanisms for first and second treatment had higher response rates than other sequences (27/49 [55{\%}] vs. 17/69 [25{\%}], p < 0.001). Children receiving first treatment within 4 weeks of IS onset had a higher response rate to second treatment than those initially treated later (36/82 [44{\%}] vs. 8/34 [24{\%}], p = 0.040). Significance: Greater than one third of children with IS will respond to a second medication. Choosing a standard medication (ACTH, oral corticosteroids, or vigabatrin) that has a different mechanism of action appears to be more effective. Rapid initial treatment increases the likelihood of response to the second treatment.",
keywords = "Adrenocorticotropic hormone, Infantile spasms, Second-line treatment, Vigabatrin",
author = "{the Pediatric Epilepsy Research Consortium} and Knupp, {Kelly G.} and Erin Leister and Jason Coryell and Nickels, {Katherine C.} and Nicole Ryan and Elizabeth Juarez-Colunga and Gaillard, {William D.} and Mytinger, {John R.} and Berg, {Anne T.} and John Millichap and Nordli, {Douglas R.} and Sucheta Joshi and Shellhaas, {Ren{\'e}e A.} and Tobias Loddenkemper and Dennis Dlugos and Elaine Wirrell and Joseph Sullivan and Hartman, {Adam L.} and Kossoff, {Eric H.} and Grinspan, {Zachary M.} and Lorie Hamikawa and Amy Brooks-Kayal and Cynthia Stack and Lawrence Brown and Cynthia Keator and Mitchell, {Wendy G.} and Jansen, {Laura A.} and Shilpi Kumar and Gogi Kumar and Elizabeth Theile and Catherine Chu and Kelley, {Sarah A.} and Elissa Yozawitz and Yozawitz, {Elissa G.} and Ignacio Valencia and Wusthoff, {Courtney J.} and Novotny, {Edward J.} and Saneto, {Russell P.} and Hussain, {Shaun A.}",
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T1 - Response to second treatment after initial failed treatment in a multicenter prospective infantile spasms cohort

AU - the Pediatric Epilepsy Research Consortium

AU - Knupp, Kelly G.

AU - Leister, Erin

AU - Coryell, Jason

AU - Nickels, Katherine C.

AU - Ryan, Nicole

AU - Juarez-Colunga, Elizabeth

AU - Gaillard, William D.

AU - Mytinger, John R.

AU - Berg, Anne T.

AU - Millichap, John

AU - Nordli, Douglas R.

AU - Joshi, Sucheta

AU - Shellhaas, Renée A.

AU - Loddenkemper, Tobias

AU - Dlugos, Dennis

AU - Wirrell, Elaine

AU - Sullivan, Joseph

AU - Hartman, Adam L.

AU - Kossoff, Eric H.

AU - Grinspan, Zachary M.

AU - Hamikawa, Lorie

AU - Brooks-Kayal, Amy

AU - Stack, Cynthia

AU - Brown, Lawrence

AU - Keator, Cynthia

AU - Mitchell, Wendy G.

AU - Jansen, Laura A.

AU - Kumar, Shilpi

AU - Kumar, Gogi

AU - Theile, Elizabeth

AU - Chu, Catherine

AU - Kelley, Sarah A.

AU - Yozawitz, Elissa

AU - Yozawitz, Elissa G.

AU - Valencia, Ignacio

AU - Wusthoff, Courtney J.

AU - Novotny, Edward J.

AU - Saneto, Russell P.

AU - Hussain, Shaun A.

PY - 2016/11/1

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N2 - Objective: Infantile spasms (IS) represent a severe epileptic encephalopathy presenting in the first 2 years of life. Recommended first-line therapies (hormonal therapy or vigabatrin) often fail. We evaluated response to second treatment for IS in children in whom the initial therapy failed to produce both clinical remission and electrographic resolution of hypsarhythmia and whether time to treatment was related to outcome. Methods: The National Infantile Spasms Consortium established a multicenter, prospective database enrolling infants with new diagnosis of IS. Children were considered nonresponders to first treatment if there was no clinical remission or persistence of hypsarhythmia. Treatment was evaluated as hormonal therapy (adrenocorticotropic hormone [ACTH] or oral corticosteroids), vigabatrin, or “other.” Standard treatments (hormonal and vigabatrin) were compared to all other nonstandard treatments. We compared response rates using chi-square tests and multivariable logistic regression models. Results: One hundred eighteen infants were included from 19 centers. Overall response rate to a second treatment was 37% (n = 44). Children who received standard medications with differing mechanisms for first and second treatment had higher response rates than other sequences (27/49 [55%] vs. 17/69 [25%], p < 0.001). Children receiving first treatment within 4 weeks of IS onset had a higher response rate to second treatment than those initially treated later (36/82 [44%] vs. 8/34 [24%], p = 0.040). Significance: Greater than one third of children with IS will respond to a second medication. Choosing a standard medication (ACTH, oral corticosteroids, or vigabatrin) that has a different mechanism of action appears to be more effective. Rapid initial treatment increases the likelihood of response to the second treatment.

AB - Objective: Infantile spasms (IS) represent a severe epileptic encephalopathy presenting in the first 2 years of life. Recommended first-line therapies (hormonal therapy or vigabatrin) often fail. We evaluated response to second treatment for IS in children in whom the initial therapy failed to produce both clinical remission and electrographic resolution of hypsarhythmia and whether time to treatment was related to outcome. Methods: The National Infantile Spasms Consortium established a multicenter, prospective database enrolling infants with new diagnosis of IS. Children were considered nonresponders to first treatment if there was no clinical remission or persistence of hypsarhythmia. Treatment was evaluated as hormonal therapy (adrenocorticotropic hormone [ACTH] or oral corticosteroids), vigabatrin, or “other.” Standard treatments (hormonal and vigabatrin) were compared to all other nonstandard treatments. We compared response rates using chi-square tests and multivariable logistic regression models. Results: One hundred eighteen infants were included from 19 centers. Overall response rate to a second treatment was 37% (n = 44). Children who received standard medications with differing mechanisms for first and second treatment had higher response rates than other sequences (27/49 [55%] vs. 17/69 [25%], p < 0.001). Children receiving first treatment within 4 weeks of IS onset had a higher response rate to second treatment than those initially treated later (36/82 [44%] vs. 8/34 [24%], p = 0.040). Significance: Greater than one third of children with IS will respond to a second medication. Choosing a standard medication (ACTH, oral corticosteroids, or vigabatrin) that has a different mechanism of action appears to be more effective. Rapid initial treatment increases the likelihood of response to the second treatment.

KW - Adrenocorticotropic hormone

KW - Infantile spasms

KW - Second-line treatment

KW - Vigabatrin

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