PittsburghV1, Main, Exploration, bibRecord, 001458

Sapropterin dihydrochloride use in pregnant women with phenylketonuria: an interim report of the PKU MOMS sub-registry.

Identifieur interne : 001458 ( Main/Exploration ); précédent : 001457; suivant : 001459

Sapropterin dihydrochloride use in pregnant women with phenylketonuria: an interim report of the PKU MOMS sub-registry.

Auteurs : Dorothy K. Grange [États-Unis] ; Richard E. Hillman [États-Unis] ; Barbara K. Burton [États-Unis] ; Shoji Yano [États-Unis] ; Jerry Vockley [États-Unis] ; Chin-To Fong [États-Unis] ; Joellen Hunt [États-Unis] ; John J. Mahoney [États-Unis] ; Jessica L. Cohen-Pfeffer [États-Unis]

Source :

Molecular genetics and metabolism [ 1096-7206 ] ; 2014.

RBID : pubmed:24667082

Descripteurs français

KwdFr :
- Accouchement prématuré (), Adulte, Avortement spontané (), Bioptérine (administration et posologie), Bioptérine (analogues et dérivés), Bioptérine (effets indésirables), Femelle, Grossesse, Humains, Issue de la grossesse, Jeune adulte, Phénylalanine (sang), Phénylcétonurie maternelle (diétothérapie), Phénylcétonurie maternelle (traitement médicamenteux).
MESH :
- administration et posologie : Bioptérine.
- analogues et dérivés : Bioptérine.
- diétothérapie : Phénylcétonurie maternelle.
- effets indésirables : Bioptérine.
- sang : Phénylalanine.
- traitement médicamenteux : Phénylcétonurie maternelle.
- Accouchement prématuré, Adulte, Avortement spontané, Femelle, Grossesse, Humains, Issue de la grossesse, Jeune adulte.

English descriptors

KwdEn :
- Abortion, Spontaneous (chemically induced), Adult, Biopterin (administration & dosage), Biopterin (adverse effects), Biopterin (analogs & derivatives), Female, Humans, Obstetric Labor, Premature (chemically induced), Phenylalanine (blood), Phenylketonuria, Maternal (diet therapy), Phenylketonuria, Maternal (drug therapy), Pregnancy, Pregnancy Outcome, Young Adult.
MESH :
- chemical , administration & dosage : Biopterin.
- chemical , adverse effects : Biopterin.
- chemical , analogs & derivatives : Biopterin.
- chemical , blood : Phenylalanine.
- chemically induced : Abortion, Spontaneous, Obstetric Labor, Premature.
- diet therapy : Phenylketonuria, Maternal.
- drug therapy : Phenylketonuria, Maternal.
- Adult, Female, Humans, Pregnancy, Pregnancy Outcome, Young Adult.

Abstract

For pregnant women with phenylketonuria (PKU), maintaining blood phenylalanine (Phe)<360μmol/L is critical due to the toxicity of elevated Phe to the fetus. Sapropterin dihydrochloride (sapropterin) lowers blood Phe in tetrahydrobiopterin (BH4) responsive patients with PKU, in conjunction with a Phe-restricted diet, but clinical evidence supporting its use during pregnancy is limited. As of June 3, 2013, the Maternal Phenylketonuria Observational Program (PKU MOMS) sub-registry contained data from 21 pregnancies - in women with PKU who were treated with sapropterin either before (N=5) or during (N=16) pregnancy. Excluding data for spontaneous abortions (N=4), the data show that the mean of median blood Phe [204.7±126.6μmol/L (n=14)] for women exposed to sapropterin during pregnancy was 23% lower, and had a 58% smaller standard deviation, compared to blood Phe [267.4±300.7μmol/L (n=3)] for women exposed to sapropterin prior to pregnancy. Women on sapropterin during pregnancy experienced fewer blood Phe values above the recommended 360μmol/L threshold. When median blood Phe concentration was <360μmol/L throughout pregnancy, 75% (12/16) of pregnancy outcomes were normal compared to 40% (2/5) when median blood Phe was >360μmol/L. Severe adverse events identified by the investigators as possibly related to sapropterin use were premature labor (N=1) and spontaneous abortion (N=1) for the women and hypophagia for the offspring [premature birth (35w4d), N=1]. One congenital malformation (cleft palate) of unknown etiology was reported as unrelated to sapropterin. Although there is limited information regarding the use of sapropterin during pregnancy, these sub-registry data show that sapropterin was generally well-tolerated and its use during pregnancy was associated with lower mean blood Phe. Because the teratogenicity of elevated maternal blood Phe is without question, sapropterin should be considered as a treatment option in pregnant women with PKU who cannot achieve recommended ranges of blood Phe with dietary therapy alone.

DOI: 10.1016/j.ymgme.2014.02.016
PubMed: 24667082

Affiliations:

Le document en format XML

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<sourceDesc><biblStruct><analytic><title xml:lang="en">Sapropterin dihydrochloride use in pregnant women with phenylketonuria: an interim report of the PKU MOMS sub-registry.</title>
<author><name sortKey="Grange, Dorothy K" sort="Grange, Dorothy K" uniqKey="Grange D" first="Dorothy K" last="Grange">Dorothy K. Grange</name>
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<settlement type="city">Saint-Louis (Missouri)</settlement>
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<orgName type="university">École de médecine (Université Washington de Saint-Louis)</orgName>
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<author><name sortKey="Hillman, Richard E" sort="Hillman, Richard E" uniqKey="Hillman R" first="Richard E" last="Hillman">Richard E. Hillman</name>
<affiliation wicri:level="2"><nlm:affiliation>University of Missouri Health Care, One Hospital Drive, University of Missouri, Columbia, MO 65212, USA.</nlm:affiliation>
<country xml:lang="fr">États-Unis</country>
<wicri:regionArea>University of Missouri Health Care, One Hospital Drive, University of Missouri, Columbia, MO 65212</wicri:regionArea>
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<author><name sortKey="Burton, Barbara K" sort="Burton, Barbara K" uniqKey="Burton B" first="Barbara K" last="Burton">Barbara K. Burton</name>
<affiliation wicri:level="2"><nlm:affiliation>Ann and Robert H. Lurie Children's Hospital of Chicago, 225 E. Chicago Ave., Box #59, Chicago, IL 60611-2605, USA.</nlm:affiliation>
<country xml:lang="fr">États-Unis</country>
<wicri:regionArea>Ann and Robert H. Lurie Children's Hospital of Chicago, 225 E. Chicago Ave., Box #59, Chicago, IL 60611-2605</wicri:regionArea>
<placeName><region type="state">Illinois</region>
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</affiliation>
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<author><name sortKey="Yano, Shoji" sort="Yano, Shoji" uniqKey="Yano S" first="Shoji" last="Yano">Shoji Yano</name>
<affiliation wicri:level="2"><nlm:affiliation>Los Angeles County & University of Southern California Medical Center, 1801 Marengo Street, Rm 1G-24, Los Angeles, CA 90033, USA.</nlm:affiliation>
<country xml:lang="fr">États-Unis</country>
<wicri:regionArea>Los Angeles County & University of Southern California Medical Center, 1801 Marengo Street, Rm 1G-24, Los Angeles, CA 90033</wicri:regionArea>
<placeName><region type="state">Californie</region>
</placeName>
</affiliation>
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<author><name sortKey="Vockley, Jerry" sort="Vockley, Jerry" uniqKey="Vockley J" first="Jerry" last="Vockley">Jerry Vockley</name>
<affiliation wicri:level="2"><nlm:affiliation>University of Pittsburgh School of Medicine, Children's Hospital of Pittsburgh, 4401 Penn Ave, Pittsburgh, PA 15224, USA; University of Pittsburgh Graduate School of Public Health, Pittsburgh, PA 15224, USA.</nlm:affiliation>
<country xml:lang="fr">États-Unis</country>
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<placeName><region type="state">Pennsylvanie</region>
</placeName>
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<author><name sortKey="Fong, Chin To" sort="Fong, Chin To" uniqKey="Fong C" first="Chin-To" last="Fong">Chin-To Fong</name>
<affiliation wicri:level="2"><nlm:affiliation>University of Rochester Medical Center, Clinic of Inherited Metabolic Disease, Box 777, Genetics 601, Elmwood Avenue, Rochester, NY 14642-8315, USA.</nlm:affiliation>
<country xml:lang="fr">États-Unis</country>
<wicri:regionArea>University of Rochester Medical Center, Clinic of Inherited Metabolic Disease, Box 777, Genetics 601, Elmwood Avenue, Rochester, NY 14642-8315</wicri:regionArea>
<placeName><region type="state">État de New York</region>
</placeName>
</affiliation>
</author>
<author><name sortKey="Hunt, Joellen" sort="Hunt, Joellen" uniqKey="Hunt J" first="Joellen" last="Hunt">Joellen Hunt</name>
<affiliation wicri:level="2"><nlm:affiliation>BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.</nlm:affiliation>
<country xml:lang="fr">États-Unis</country>
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<author><name sortKey="Mahoney, John J" sort="Mahoney, John J" uniqKey="Mahoney J" first="John J" last="Mahoney">John J. Mahoney</name>
<affiliation wicri:level="2"><nlm:affiliation>BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.</nlm:affiliation>
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<author><name sortKey="Cohen Pfeffer, Jessica L" sort="Cohen Pfeffer, Jessica L" uniqKey="Cohen Pfeffer J" first="Jessica L" last="Cohen-Pfeffer">Jessica L. Cohen-Pfeffer</name>
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<series><title level="j">Molecular genetics and metabolism</title>
<idno type="eISSN">1096-7206</idno>
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<profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>Abortion, Spontaneous (chemically induced)</term>
<term>Adult</term>
<term>Biopterin (administration & dosage)</term>
<term>Biopterin (adverse effects)</term>
<term>Biopterin (analogs & derivatives)</term>
<term>Female</term>
<term>Humans</term>
<term>Obstetric Labor, Premature (chemically induced)</term>
<term>Phenylalanine (blood)</term>
<term>Phenylketonuria, Maternal (diet therapy)</term>
<term>Phenylketonuria, Maternal (drug therapy)</term>
<term>Pregnancy</term>
<term>Pregnancy Outcome</term>
<term>Young Adult</term>
</keywords>
<keywords scheme="KwdFr" xml:lang="fr"><term>Accouchement prématuré ()</term>
<term>Adulte</term>
<term>Avortement spontané ()</term>
<term>Bioptérine (administration et posologie)</term>
<term>Bioptérine (analogues et dérivés)</term>
<term>Bioptérine (effets indésirables)</term>
<term>Femelle</term>
<term>Grossesse</term>
<term>Humains</term>
<term>Issue de la grossesse</term>
<term>Jeune adulte</term>
<term>Phénylalanine (sang)</term>
<term>Phénylcétonurie maternelle (diétothérapie)</term>
<term>Phénylcétonurie maternelle (traitement médicamenteux)</term>
</keywords>
<keywords scheme="MESH" type="chemical" qualifier="administration & dosage" xml:lang="en"><term>Biopterin</term>
</keywords>
<keywords scheme="MESH" type="chemical" qualifier="adverse effects" xml:lang="en"><term>Biopterin</term>
</keywords>
<keywords scheme="MESH" type="chemical" qualifier="analogs & derivatives" xml:lang="en"><term>Biopterin</term>
</keywords>
<keywords scheme="MESH" type="chemical" qualifier="blood" xml:lang="en"><term>Phenylalanine</term>
</keywords>
<keywords scheme="MESH" qualifier="administration et posologie" xml:lang="fr"><term>Bioptérine</term>
</keywords>
<keywords scheme="MESH" qualifier="analogues et dérivés" xml:lang="fr"><term>Bioptérine</term>
</keywords>
<keywords scheme="MESH" qualifier="chemically induced" xml:lang="en"><term>Abortion, Spontaneous</term>
<term>Obstetric Labor, Premature</term>
</keywords>
<keywords scheme="MESH" qualifier="diet therapy" xml:lang="en"><term>Phenylketonuria, Maternal</term>
</keywords>
<keywords scheme="MESH" qualifier="diétothérapie" xml:lang="fr"><term>Phénylcétonurie maternelle</term>
</keywords>
<keywords scheme="MESH" qualifier="drug therapy" xml:lang="en"><term>Phenylketonuria, Maternal</term>
</keywords>
<keywords scheme="MESH" qualifier="effets indésirables" xml:lang="fr"><term>Bioptérine</term>
</keywords>
<keywords scheme="MESH" qualifier="sang" xml:lang="fr"><term>Phénylalanine</term>
</keywords>
<keywords scheme="MESH" qualifier="traitement médicamenteux" xml:lang="fr"><term>Phénylcétonurie maternelle</term>
</keywords>
<keywords scheme="MESH" xml:lang="en"><term>Adult</term>
<term>Female</term>
<term>Humans</term>
<term>Pregnancy</term>
<term>Pregnancy Outcome</term>
<term>Young Adult</term>
</keywords>
<keywords scheme="MESH" xml:lang="fr"><term>Accouchement prématuré</term>
<term>Adulte</term>
<term>Avortement spontané</term>
<term>Femelle</term>
<term>Grossesse</term>
<term>Humains</term>
<term>Issue de la grossesse</term>
<term>Jeune adulte</term>
</keywords>
</textClass>
</profileDesc>
</teiHeader>
<front><div type="abstract" xml:lang="en">For pregnant women with phenylketonuria (PKU), maintaining blood phenylalanine (Phe)<360μmol/L is critical due to the toxicity of elevated Phe to the fetus. Sapropterin dihydrochloride (sapropterin) lowers blood Phe in tetrahydrobiopterin (BH4) responsive patients with PKU, in conjunction with a Phe-restricted diet, but clinical evidence supporting its use during pregnancy is limited. As of June 3, 2013, the Maternal Phenylketonuria Observational Program (PKU MOMS) sub-registry contained data from 21 pregnancies - in women with PKU who were treated with sapropterin either before (N=5) or during (N=16) pregnancy. Excluding data for spontaneous abortions (N=4), the data show that the mean of median blood Phe [204.7±126.6μmol/L (n=14)] for women exposed to sapropterin during pregnancy was 23% lower, and had a 58% smaller standard deviation, compared to blood Phe [267.4±300.7μmol/L (n=3)] for women exposed to sapropterin prior to pregnancy. Women on sapropterin during pregnancy experienced fewer blood Phe values above the recommended 360μmol/L threshold. When median blood Phe concentration was <360μmol/L throughout pregnancy, 75% (12/16) of pregnancy outcomes were normal compared to 40% (2/5) when median blood Phe was >360μmol/L. Severe adverse events identified by the investigators as possibly related to sapropterin use were premature labor (N=1) and spontaneous abortion (N=1) for the women and hypophagia for the offspring [premature birth (35w4d), N=1]. One congenital malformation (cleft palate) of unknown etiology was reported as unrelated to sapropterin. Although there is limited information regarding the use of sapropterin during pregnancy, these sub-registry data show that sapropterin was generally well-tolerated and its use during pregnancy was associated with lower mean blood Phe. Because the teratogenicity of elevated maternal blood Phe is without question, sapropterin should be considered as a treatment option in pregnant women with PKU who cannot achieve recommended ranges of blood Phe with dietary therapy alone.</div>
</front>
</TEI>
<affiliations><list><country><li>États-Unis</li>
</country>
<region><li>Californie</li>
<li>Illinois</li>
<li>Missouri (État)</li>
<li>Pennsylvanie</li>
<li>État de New York</li>
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<settlement><li>Saint-Louis (Missouri)</li>
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<tree><country name="États-Unis"><region name="Missouri (État)"><name sortKey="Grange, Dorothy K" sort="Grange, Dorothy K" uniqKey="Grange D" first="Dorothy K" last="Grange">Dorothy K. Grange</name>
</region>
<name sortKey="Burton, Barbara K" sort="Burton, Barbara K" uniqKey="Burton B" first="Barbara K" last="Burton">Barbara K. Burton</name>
<name sortKey="Cohen Pfeffer, Jessica L" sort="Cohen Pfeffer, Jessica L" uniqKey="Cohen Pfeffer J" first="Jessica L" last="Cohen-Pfeffer">Jessica L. Cohen-Pfeffer</name>
<name sortKey="Fong, Chin To" sort="Fong, Chin To" uniqKey="Fong C" first="Chin-To" last="Fong">Chin-To Fong</name>
<name sortKey="Hillman, Richard E" sort="Hillman, Richard E" uniqKey="Hillman R" first="Richard E" last="Hillman">Richard E. Hillman</name>
<name sortKey="Hunt, Joellen" sort="Hunt, Joellen" uniqKey="Hunt J" first="Joellen" last="Hunt">Joellen Hunt</name>
<name sortKey="Mahoney, John J" sort="Mahoney, John J" uniqKey="Mahoney J" first="John J" last="Mahoney">John J. Mahoney</name>
<name sortKey="Vockley, Jerry" sort="Vockley, Jerry" uniqKey="Vockley J" first="Jerry" last="Vockley">Jerry Vockley</name>
<name sortKey="Yano, Shoji" sort="Yano, Shoji" uniqKey="Yano S" first="Shoji" last="Yano">Shoji Yano</name>
</country>
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EXPLOR_STEP=$WICRI_ROOT/Wicri/Amérique/explor/PittsburghV1/Data/Main/Exploration

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HfdSelect -h $EXPLOR_AREA/Data/Main/Exploration/biblio.hfd -nk 001458 | SxmlIndent | more

Pour mettre un lien sur cette page dans le réseau Wicri

{{Explor lien
   |wiki=    Wicri/Amérique
   |area=    PittsburghV1
   |flux=    Main
   |étape=   Exploration
   |type=    RBID
   |clé=     pubmed:24667082
   |texte=   Sapropterin dihydrochloride use in pregnant women with phenylketonuria: an interim report of the PKU MOMS sub-registry.
}}

Pour générer des pages wiki

HfdIndexSelect -h $EXPLOR_AREA/Data/Main/Exploration/RBID.i   -Sk "pubmed:24667082" \
       | HfdSelect -Kh $EXPLOR_AREA/Data/Main/Exploration/biblio.hfd   \
       | NlmPubMed2Wicri -a PittsburghV1

This area was generated with Dilib version V0.6.38.
Data generation: Fri Jun 18 17:37:45 2021. Site generation: Fri Jun 18 18:15:47 2021

Serveur d'exploration sur Pittsburgh

Sapropterin dihydrochloride use in pregnant women with phenylketonuria: an interim report of the PKU MOMS sub-registry.

Sapropterin dihydrochloride use in pregnant women with phenylketonuria: an interim report of the PKU MOMS sub-registry.

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Pour mettre un lien sur cette page dans le réseau Wicri

Pour générer des pages wiki

	Serveur d'exploration sur Pittsburgh
	Attention, ce site est en cours de développement ! Attention, site généré par des moyens informatiques à partir de corpus bruts. Les informations ne sont donc pas validées.