Efficacy and Safety of Sirolimus in the Treatment of Complicated Vascular Anomalies
Identifieur interne : 001127 ( Main/Exploration ); précédent : 001126; suivant : 001128Efficacy and Safety of Sirolimus in the Treatment of Complicated Vascular Anomalies
Auteurs : Denise M. Adams [États-Unis] ; Cameron C. Trenor ; Adrienne M. Hammill [États-Unis] ; Alexander A. Vinks [États-Unis] ; Manish N. Patel [États-Unis] ; Gulraiz Chaudry ; Mary Sue Wentzel [États-Unis] ; Paula S. Mobberley-Schuman [États-Unis] ; Lisa M. Campbell [États-Unis] ; Christine Brookbank [États-Unis] ; Anita Gupta [États-Unis] ; Carol Chute [États-Unis] ; Jennifer Eile ; Jesse Mckenna ; Arnold C. Merrow [États-Unis] ; Lin Fei [États-Unis] ; Lindsey Hornung [États-Unis] ; Michael Seid [États-Unis] ; A. Roshni Dasgupta [États-Unis] ; Belinda H. Dickie [États-Unis] ; Ravindhra G. Elluru [États-Unis] ; Anne W. Lucky [États-Unis] ; Brian Weiss [États-Unis] ; Richard G. Azizkhan [États-Unis]Source :
- Pediatrics [ 0031-4005 ] ; 2016.
Abstract
Complicated vascular anomalies have limited therapeutic options and cause significant morbidity and mortality. This Phase II trial enrolled patients with complicated vascular anomalies to determine the efficacy and safety of treatment with sirolimus for 12 courses; each course was defined as 28 days.
Treatment consisted of a continuous dosing schedule of oral sirolimus starting at 0.8 mg/m2 per dose twice daily, with pharmacokinetic-guided target serum trough levels of 10 to 15 ng/mL. The primary outcomes were responsiveness to sirolimus by the end of course 6 (evaluated according to functional impairment score, quality of life, and radiologic assessment) and the incidence of toxicities and/or infection
Sixty-one patients were enrolled; 57 patients were evaluable for efficacy at the end of course 6, and 53 were evaluable at the end of course 12. No patient had a complete response at the end of course 6 or 12 as anticipated. At the end of course 6, a total of 47 patients had a partial response, 3 patients had stable disease, and 7 patients had progressive disease. Two patients were taken off of study medicine secondary to persistent adverse effects. Grade 3 and higher toxicities attributable to sirolimus included blood/bone marrow toxicity in 27% of patients, gastrointestinal toxicity in 3%, and metabolic/laboratory toxicity in 3%. No toxicity-related deaths occurred.
Sirolimus was efficacious and well tolerated in these study patients with complicated vascular anomalies. Clinical activity was reported in the majority of the disorders.
Url:
DOI: 10.1542/peds.2015-3257
PubMed: 26783326
PubMed Central: 4732362
Affiliations:
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Le document en format XML
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</author>
<author><name sortKey="Seid, Michael" sort="Seid, Michael" uniqKey="Seid M" first="Michael" last="Seid">Michael Seid</name>
<affiliation wicri:level="2"><nlm:aff id="aff1">Cincinnati Children’s Hospital Medical Center, Cincinnati, Ohio;</nlm:aff>
<country xml:lang="fr">États-Unis</country>
<placeName><region type="state">Ohio</region>
</placeName>
<wicri:cityArea>Cincinnati Children’s Hospital Medical Center, Cincinnati</wicri:cityArea>
</affiliation>
</author>
<author><name sortKey="Dasgupta, A Roshni" sort="Dasgupta, A Roshni" uniqKey="Dasgupta A" first="A. Roshni" last="Dasgupta">A. Roshni Dasgupta</name>
<affiliation wicri:level="2"><nlm:aff id="aff1">Cincinnati Children’s Hospital Medical Center, Cincinnati, Ohio;</nlm:aff>
<country xml:lang="fr">États-Unis</country>
<placeName><region type="state">Ohio</region>
</placeName>
<wicri:cityArea>Cincinnati Children’s Hospital Medical Center, Cincinnati</wicri:cityArea>
</affiliation>
<affiliation><nlm:aff>NONE</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Dickie, Belinda H" sort="Dickie, Belinda H" uniqKey="Dickie B" first="Belinda H." last="Dickie">Belinda H. Dickie</name>
<affiliation wicri:level="2"><nlm:aff id="aff1">Cincinnati Children’s Hospital Medical Center, Cincinnati, Ohio;</nlm:aff>
<country xml:lang="fr">États-Unis</country>
<placeName><region type="state">Ohio</region>
</placeName>
<wicri:cityArea>Cincinnati Children’s Hospital Medical Center, Cincinnati</wicri:cityArea>
</affiliation>
<affiliation><nlm:aff>NONE</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Elluru, Ravindhra G" sort="Elluru, Ravindhra G" uniqKey="Elluru R" first="Ravindhra G." last="Elluru">Ravindhra G. Elluru</name>
<affiliation wicri:level="2"><nlm:aff wicri:cut="; and" id="aff4">Dayton Children’s Medical Center, Dayton, Ohio</nlm:aff>
<country xml:lang="fr">États-Unis</country>
<placeName><region type="state">Ohio</region>
</placeName>
<wicri:cityArea>Dayton Children’s Medical Center, Dayton</wicri:cityArea>
</affiliation>
</author>
<author><name sortKey="Lucky, Anne W" sort="Lucky, Anne W" uniqKey="Lucky A" first="Anne W." last="Lucky">Anne W. Lucky</name>
<affiliation wicri:level="2"><nlm:aff id="aff1">Cincinnati Children’s Hospital Medical Center, Cincinnati, Ohio;</nlm:aff>
<country xml:lang="fr">États-Unis</country>
<placeName><region type="state">Ohio</region>
</placeName>
<wicri:cityArea>Cincinnati Children’s Hospital Medical Center, Cincinnati</wicri:cityArea>
</affiliation>
</author>
<author><name sortKey="Weiss, Brian" sort="Weiss, Brian" uniqKey="Weiss B" first="Brian" last="Weiss">Brian Weiss</name>
<affiliation wicri:level="2"><nlm:aff id="aff1">Cincinnati Children’s Hospital Medical Center, Cincinnati, Ohio;</nlm:aff>
<country xml:lang="fr">États-Unis</country>
<placeName><region type="state">Ohio</region>
</placeName>
<wicri:cityArea>Cincinnati Children’s Hospital Medical Center, Cincinnati</wicri:cityArea>
</affiliation>
<affiliation><nlm:aff>NONE</nlm:aff>
</affiliation>
</author>
<author><name sortKey="Azizkhan, Richard G" sort="Azizkhan, Richard G" uniqKey="Azizkhan R" first="Richard G." last="Azizkhan">Richard G. Azizkhan</name>
<affiliation wicri:level="2"><nlm:aff id="aff5">Omaha Children’s Hospital and Medical Center, Omaha, Nebraska</nlm:aff>
<country xml:lang="fr">États-Unis</country>
<placeName><region type="state">Nebraska</region>
</placeName>
<wicri:cityArea>Omaha Children’s Hospital and Medical Center, Omaha</wicri:cityArea>
</affiliation>
</author>
</analytic>
<series><title level="j">Pediatrics</title>
<idno type="ISSN">0031-4005</idno>
<idno type="eISSN">1098-4275</idno>
<imprint><date when="2016">2016</date>
</imprint>
</series>
</biblStruct>
</sourceDesc>
</fileDesc>
<profileDesc><textClass></textClass>
</profileDesc>
</teiHeader>
<front><div type="abstract" xml:lang="en"><sec id="s01"><title>BACKGROUND AND OBJECTIVES:</title>
<p>Complicated vascular anomalies have limited therapeutic options and cause significant morbidity and mortality. This Phase II trial enrolled patients with complicated vascular anomalies to determine the efficacy and safety of treatment with sirolimus for 12 courses; each course was defined as 28 days.</p>
</sec>
<sec id="s02"><title>METHODS:</title>
<p>Treatment consisted of a continuous dosing schedule of oral sirolimus starting at 0.8 mg/m<sup>2</sup>
per dose twice daily, with pharmacokinetic-guided target serum trough levels of 10 to 15 ng/mL. The primary outcomes were responsiveness to sirolimus by the end of course 6 (evaluated according to functional impairment score, quality of life, and radiologic assessment) and the incidence of toxicities and/or infection<bold>-</bold>
related deaths.</p>
</sec>
<sec id="s03"><title>RESULTS:</title>
<p>Sixty-one patients were enrolled; 57 patients were evaluable for efficacy at the end of course 6, and 53 were evaluable at the end of course 12. No patient had a complete response at the end of course 6 or 12 as anticipated. At the end of course 6, a total of 47 patients had a partial response, 3 patients had stable disease, and 7 patients had progressive disease. Two patients were taken off of study medicine secondary to persistent adverse effects. Grade 3 and higher toxicities attributable to sirolimus included blood/bone marrow toxicity in 27% of patients, gastrointestinal toxicity in 3%, and metabolic/laboratory toxicity in 3%. No toxicity-related deaths occurred.</p>
</sec>
<sec id="s04"><title>CONCLUSIONS:</title>
<p>Sirolimus was efficacious and well tolerated in these study patients with complicated vascular anomalies. Clinical activity was reported in the majority of the disorders.</p>
</sec>
</div>
</front>
</TEI>
<affiliations><list><country><li>États-Unis</li>
</country>
<region><li>Nebraska</li>
<li>Ohio</li>
</region>
</list>
<tree><noCountry><name sortKey="Chaudry, Gulraiz" sort="Chaudry, Gulraiz" uniqKey="Chaudry G" first="Gulraiz" last="Chaudry">Gulraiz Chaudry</name>
<name sortKey="Eile, Jennifer" sort="Eile, Jennifer" uniqKey="Eile J" first="Jennifer" last="Eile">Jennifer Eile</name>
<name sortKey="Mckenna, Jesse" sort="Mckenna, Jesse" uniqKey="Mckenna J" first="Jesse" last="Mckenna">Jesse Mckenna</name>
<name sortKey="Trenor, Cameron C" sort="Trenor, Cameron C" uniqKey="Trenor C" first="Cameron C." last="Trenor">Cameron C. Trenor</name>
</noCountry>
<country name="États-Unis"><region name="Ohio"><name sortKey="Adams, Denise M" sort="Adams, Denise M" uniqKey="Adams D" first="Denise M." last="Adams">Denise M. Adams</name>
</region>
<name sortKey="Azizkhan, Richard G" sort="Azizkhan, Richard G" uniqKey="Azizkhan R" first="Richard G." last="Azizkhan">Richard G. Azizkhan</name>
<name sortKey="Brookbank, Christine" sort="Brookbank, Christine" uniqKey="Brookbank C" first="Christine" last="Brookbank">Christine Brookbank</name>
<name sortKey="Campbell, Lisa M" sort="Campbell, Lisa M" uniqKey="Campbell L" first="Lisa M." last="Campbell">Lisa M. Campbell</name>
<name sortKey="Chute, Carol" sort="Chute, Carol" uniqKey="Chute C" first="Carol" last="Chute">Carol Chute</name>
<name sortKey="Dasgupta, A Roshni" sort="Dasgupta, A Roshni" uniqKey="Dasgupta A" first="A. Roshni" last="Dasgupta">A. Roshni Dasgupta</name>
<name sortKey="Dickie, Belinda H" sort="Dickie, Belinda H" uniqKey="Dickie B" first="Belinda H." last="Dickie">Belinda H. Dickie</name>
<name sortKey="Elluru, Ravindhra G" sort="Elluru, Ravindhra G" uniqKey="Elluru R" first="Ravindhra G." last="Elluru">Ravindhra G. Elluru</name>
<name sortKey="Fei, Lin" sort="Fei, Lin" uniqKey="Fei L" first="Lin" last="Fei">Lin Fei</name>
<name sortKey="Gupta, Anita" sort="Gupta, Anita" uniqKey="Gupta A" first="Anita" last="Gupta">Anita Gupta</name>
<name sortKey="Hammill, Adrienne M" sort="Hammill, Adrienne M" uniqKey="Hammill A" first="Adrienne M." last="Hammill">Adrienne M. Hammill</name>
<name sortKey="Hornung, Lindsey" sort="Hornung, Lindsey" uniqKey="Hornung L" first="Lindsey" last="Hornung">Lindsey Hornung</name>
<name sortKey="Lucky, Anne W" sort="Lucky, Anne W" uniqKey="Lucky A" first="Anne W." last="Lucky">Anne W. Lucky</name>
<name sortKey="Merrow, Arnold C" sort="Merrow, Arnold C" uniqKey="Merrow A" first="Arnold C." last="Merrow">Arnold C. Merrow</name>
<name sortKey="Mobberley Schuman, Paula S" sort="Mobberley Schuman, Paula S" uniqKey="Mobberley Schuman P" first="Paula S." last="Mobberley-Schuman">Paula S. Mobberley-Schuman</name>
<name sortKey="Patel, Manish N" sort="Patel, Manish N" uniqKey="Patel M" first="Manish N." last="Patel">Manish N. Patel</name>
<name sortKey="Seid, Michael" sort="Seid, Michael" uniqKey="Seid M" first="Michael" last="Seid">Michael Seid</name>
<name sortKey="Vinks, Alexander A" sort="Vinks, Alexander A" uniqKey="Vinks A" first="Alexander A." last="Vinks">Alexander A. Vinks</name>
<name sortKey="Weiss, Brian" sort="Weiss, Brian" uniqKey="Weiss B" first="Brian" last="Weiss">Brian Weiss</name>
<name sortKey="Wentzel, Mary Sue" sort="Wentzel, Mary Sue" uniqKey="Wentzel M" first="Mary Sue" last="Wentzel">Mary Sue Wentzel</name>
</country>
</tree>
</affiliations>
</record>
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