Chii chinonzi Pegvaliase: Zvayakashandiswa, Dosage, Side Effects uye Zvimwe

Pegvaliase mushonga wakakosha wekutsiva enzyme wakagadzirirwa zvakananga vanhu vakuru vane phenylketonuria (PKU), chirwere chisingawanzoitiki chegenetic uko muviri usingakwanise kuputsa amino acid phenylalanine. Mushonga uyu unoshanda nekupa enzyme inoshayikwa iyo muviri wako unoda kuti ugadzirise phenylalanine zvakanaka, ichibatsira kudzikisa mazinga ane njodzi muropa rako.

Kana iwe kana mumwe munhu waunoda aine PKU, unogona kunzwa wakanyanya nekusarudza nzira dzekurapa. Pegvaliase inomiririra kubudirira kukuru kune vanhu vasina kusvika padanho re phenylalanine kuburikidza nekudya chete, ichipa tariro yekuvandudza manejimendi echirwere uye kuvandudza hupenyu.

Chii chinonzi Pegvaliase?

Pegvaliase ndiyo vhezheni yakagadzirwa nemunhu ye enzyme inonzi phenylalanine ammonia lyase iyo muviri wako usina kana uine PKU. Funga nezvazvo sekupa muviri wako chishandiso chinoshayikwa chaunoda kuti uputse phenylalanine kubva kumapuroteni aunodya.

Mushonga uyu unouya semvura yakajeka yaunojekesa pasi peganda rako, zvakafanana nemaitiro anoshandiswa nevanhu vane chirwere cheshuga insulin. Zita remhando ye pegvaliase ndiPalynziq, uye yakagadzirirwa zvakananga vanhu vakuru vane makore 18 zvichikwira vane PKU.

Chinonyanya kuita kuti pegvaliase ive yakasarudzika ndeyekuti inobva kubva kubhakitiriya uye yakagadzirirwa zvakanyatsonaka kuti ishande zvakachengeteka mumuviri wemunhu. Mushonga unopfuura nekucheneswa kwakanyanya kuti uve nechokwadi chekuti unosangana nematanho akasimba ekuchengetedza usati wasvika varwere.

Chii chinonzi Pegvaliase Chinoshandiswa Kuti?

Pegvaliase inorairwa chete kune vanhu vakuru vane phenylketonuria (PKU) avo mazinga e phenylalanine eropa anoramba akakwira zvakanyanya kunyangwe vachitevera kudya kwakanyanya-protein. PKU chirwere chegenetic uko muviri wako usingakwanise kuputsa phenylalanine, amino acid inowanikwa muzvokudya zvakawanda zvine mapuroteni.

Kana phenylalanine ikawanda muropa rako, inogona kukonzera matambudziko makuru ehutano anosanganisira kuremara kwepfungwa, kubatwa, nyaya dzekuzvibata, uye mamwe matambudziko ekuita zvepfungwa. Vanhu vane PKU vanowanzoita kuti mamiriro avo avepo kuburikidza nekudya kunodzora zvakanyanya kunodzora kupinda kweprotein, asi nzira iyi haishande kune vese.

Chiremba wako angakurudzira pegvaliase kana mazinga ako e phenylalanine achiramba achipfuura 600 micromol/L (anenge 10 mg/dL) pasinei nekuedza kwako kwese nekutarisira kudya. Chinangwa ndechekuunza mazinga aya pasi kusvika kune yakachengeteka, zvakanaka pasi pe360 micromol/L (6 mg/dL).

Pegvaliase Inoshanda Sei?

Pegvaliase inoshanda nekupa muviri wako enzyme yainoshaya kuti iparadze phenylalanine. Mune vanhu vasina PKU, enzyme inonzi phenylalanine hydroxylase inoshandura phenylalanine kuita imwe amino acid inonzi tyrosine. Paunenge uine PKU, enzyme iyi haishande zvakanaka.

Pegvaliase inotora imwe nzira nekushandisa enzyme inonzi phenylalanine ammonia lyase, iyo inoparadza phenylalanine kuburikidza neimwe nzira yemakemikari. Iyi nzira imwe inobatsira kuderedza huwandu hwe phenylalanine inotenderera muropa rako, kunyangwe iwe uchidya chikafu chine protein.

Mishonga inofungidzirwa kuti ndiyo mushonga wakasimba uye unoshanda wePKU kana kutarisira kudya chete kusina kukwana. Zvisinei, zvinowanzo tora mwedzi yakawanda kuti uone mhedzisiro yakazara, uye iwe uchada bvunzo dzeropa dzenguva dzose kuti uongorore mazinga ako e phenylalanine uye uchinje mushonga wako zvinoenderana.

Ndinofanira Kutora Sei Pegvaliase?

Pegvaliase inopiwa sejojo subcutaneous, zvinoreva kuti unoiisa mumatishu ane mafuta ari pasi peganda rako. Chikwata chako chehutano chichakudzidzisa maitiro ekuzvipa majekiseni aya zvakachengeteka uye zvakanaka kumba.

Nzvimbo dzakajairika dzekubaya jekiseni dzinosanganisira chidya chako, ruoko rwepamusoro, kana dumbu. Unofanira kuchinjisa nzvimbo dzekubaya jekiseni kudzivirira kutsamwa kweganda uye kudzivisa kubaya munzvimbo imwechete kakawanda. Chenesa nzvimbo yekubaya jekiseni nedoro pamberi pejekiseni rega rega uye shandisa tsono itsva nguva dzese.

Unogona kutora pegvaliase uine kana usina chikafu, uye hapana zvirambidzo zvakakosha zvekudya zvine chekuita nekuti unobaya mushonga rini. Zvisinei, unofanira kuramba uchitevera PKU yako yakarairwa sezvakarairwa nechikwata chako chehutano, kunyangwe uchishandisa pegvaliase.

Chengeta mabhodhoro asina kuvhurwa mufiriji pakati pe36°F kusvika 46°F (2°C kusvika 8°C). Usatonhodze mushonga kana kuzunza bhodhoro zvakasimba. Usati wabaya, rega mushonga usvike tembiricha yemumba kwemaminitsi anenge 15-30 kuti jekiseni rive nyore.

Ndingatora Pegvaliase Kwenguva Yakareba Sei?

Pegvaliase inowanzoita kurapwa kwenguva refu kwaunozoda kuramba uchitora kwenguva isingagumi kuti uchengetedze mazinga epasi e phenylalanine. Sezvo PKU iri mamiriro ejeneti e hupenyu hwese, kumisa mushonga ungakonzera kuti mazinga ako e phenylalanine akwirezve.

Chiremba wako achakutanga padosi yakaderera uye zvishoma nezvishoma achiwedzera pamusoro pemwedzi yakati wandei zvichienderana nekuti muviri wako unopindura sei uye unotsungirira sei mushonga. Iyi nzira, inonzi dose titration, inogona kutora mwedzi 6-12 kana kupfuura kuti usvike padosi yako yakakwana.

Miedzo yeropa yenguva dzose ichabatsira chikwata chako chehutano kuti chitarise mazinga ako e phenylalanine uye kuona kana kugadzirisa dosi kuchidiwa. Vamwe vanhu vangada shanduko yedosi nekufamba kwenguva zvichienderana nezvinhu zvakaita sekuchinja kwekudya, uremu, kana kuti muviri wavo unopindura sei kumushonga.

Usambomira kutora pegvaliase kamwechete usina kutanga wataura nachiremba wako. Kana iwe uchida kumisa mushonga nechikonzero chipi zvacho, chikwata chako chehutano chichakubatsira kugadzira chirongwa chekugadzirisa PKU yako kuburikidza nedzimwe nzira.

Ndezvipi Zvakaipa ZvePegvaliase?

Sezvinongoita mishonga yese, pegvaliase inogona kukonzera matambudziko, kunyangwe kwete munhu wese anoawana. Matambudziko anowanzoitika ane chekuita nekupindura kwenzvimbo yekujekisira uye kupindura kwemuviri wako kumishonga.

Kunzwisisa zvekutarisira kunogona kukubatsira kuti unzwe wakagadzirira uye uine chivimbo pakutanga kurapwa uku. Heano matambudziko aunogona kusangana nawo, akarongwa kubva kune anowanzoitika kusvika kune asingawanzoitiki:

Matambudziko anowanzoitika (anokanganisa vanhu vanopfuura 10%):

• Kupindura kwenzvimbo yekujekisira senge kutsvuka, kuzvimba, kurwadziwa, kana kupwanya
• Kurwadziwa kwemajoini kana kuoma
• Kupindura kweallergy kubva pakutsvuka kweganda kusvika kune zviratidzo zvakanyanya
• Kutemwa nemusoro
• Kuneta kana kunzwa kuneta
• Nausea kana dumbu rinogumbuka
• Kurwadziwa kwemhasuru
• Fivha kana zviratidzo zvakaita sefuruwenza

Matambudziko asingawanzoitiki asi anogona kuva akakomba:

• Kupindura kwakanyanya kweallergy (anaphylaxis) kunoda kutariswa kwekurapa nekukasira
• Kupindura kweganda kwakanyanya kusanganisira kutsvuka, hives, kana kupepuka
• Kutadza kufema kana kuchema
• Kuzvimba kwechiso, miromo, rurimi, kana huro
• Kurwadziwa kwemajoini akanyanya kana kuzvimba
• Fivha inoramba iripo kana zviratidzo zvehutachiona

Zvisingawanzoitiki asi zvakakomba matambudziko:

• Kupindura kweAutoimmune uko muviri wako unorwisa tishu ine hutano
• Kupindura kwenzvimbo yekujekisira kwakanyanya kusingaporesi zvakanaka
• Kuchinja muhuwandu hwemasero eropa
• Kuchinja kwekuita basa rechiropa

Kupindura kwenzvimbo yekujekisira kunowanzoita kuti kuve kwakapfava uye kunovandudzika mukati memazuva mashoma. Zvisinei, kana iwe ukasangana nekupindura kwakanyanya kweallergy, kutadza kufema, kana chero zviratidzo zvinokunetsa, tsvaga kutariswa kwekurapa nekukasira.

Ndiani Asingafaniri Kutora Pegvaliase?

Pegvaliase haina kukodzera munhu wese ane PKU. Chiremba wako achanyatsoongorora kana mushonga uyu wakakukodzera zvichibva pazvinhu zvakawanda zvakakosha.

Haufaniri kutora pegvaliase kana muchiziva kuti mune allergy yakakomba kumushonga uyu kana kune chero zvinhu zvawo. Izvi zvinosanganisira kuita zvakanyanya kuipa kare kune pegvaliase kana mamwe marapirwo akafanana ekutsiva enzyme.

Mamwe mamiriro ezvinhu ekurapa uye mamiriro anoda kufungwa zvakanyanya musati matanga pegvaliase:

Mamiriro anoda kuongororwa zvakanyanya:

• Asthma yakakomba kana mamwe mamiriro akakomba ekufema
• Nhoroondo yezvinhu zvakanyanya kuipa kune mishonga kana chikafu
• Zvirwere zvinoshanda zve autoimmune
• Chiropa chakakomba kana chirwere cheitsvo
• Zvirwere zvinoshanda kana immune system yakakanganiswa
• Matambudziko ekubuda ropa kana kutora mishonga inoderedza ropa

Vanhu vakakosha vanoda kungwarira zvakanyanya:

• Vakadzi vane pamuviri kana vanoyamwisa (data rekuchengeteka riripo)
• Vanhu vari kuronga kuvhiyiwa kana maitiro emazino
• Avo vari kutora mishonga inodzvinyirira immune system
• Vanhu vane nhoroondo ye allergy kumishonga

Zera zvakare chinhu nekuti pegvaliase inongobvumidzwa chete kune vanhu vakuru vane makore 18 zvichikwira. Vana nevechiri kuyaruka vane PKU vanofanirwa kuenderera mberi nekudya uye mamwe marapirwo akabvumidzwa kusvika vasvika pakukura.

Zita reBrand rePegvaliase

Zita rebrand re pegvaliase ndiPalynziq, inogadzirwa neBioMarin Pharmaceutical. Iri ndiro zita rebrand chete rinowanikwa re pegvaliase muUnited States nemamwe mazana enyika.

Palynziq inouya mumavhiri ekushandisa kamwe chete ane huwandu hwakasiyana hwemushonga. Chiremba wako achanyora huwandu chaihwo uye chiyero chakakodzera zvaunoda.

Sezvo pegvaliase iri mushonga wakakosha wemamiriro asingawanzoitika, inowanikwa chete kuburikidza nemakemisi akakosha ane ruzivo rwekugadzirisa marapirwo ekutsiva enzyme. Chikwata chako chehutano chichabatsira kuronga kuwana mushonga wako kuburikidza nemigero yakakodzera.

Zvimwe Zvingashandiswa zvePegvaliase

Nepo pegvaliase ndiyo chete mushonga wekutsiva enzyme unowanikwa we PKU, kune dzimwe nzira dzekurapa dzinogona kufungwa nachiremba wako zvichienderana nemamiriro ako chaiwo uye zvaunoda.

Imwe nzira huru yekutsiva pegvaliase inoramba iri manejimendi yechikafu yakasimba, iyo inosanganisira kutevera chikafu chine mapuroteni mashoma uye kushandisa chikafu chakakosha chemishonga chakagadzirirwa vanhu vane PKU. Iyi nzira inoshanda zvakanaka kune vanhu vazhinji asi inoda shanduko huru yehupenyu uye kunyatsoongorora.

Zvimwe zvingasarudzwa zvekutariswa zvinosanganisira:

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• Sapropterin (Kuvan) - mushonga unogona kubatsira vamwe vanhu vane mhando dzePKU kugadzirisa phenylalanine zviri nani
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• Zvimwe zvinowedzerwa zveamino acid zvakakura zvinogona kubatsira kuderedza kunyudzwa kwe phenylalanine
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• Glycomacropeptide-based chikafu chemishonga chinopa mapuroteni ane phenylalanine yakaderera
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• Kurapa kwekuongorora kuri kudzidzwa mumiedzo yekiriniki
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Chikwata chako chehutano chichashanda newe kuti chizive musanganiswa wakanakisa wekurapa zvichienderana nemazinga ako e phenylalanine, zvaunofarira zvekudya, mararamiro, uye kuti unopindura sei kune nzira dzakasiyana.

Ko Pegvaliase Zviri Nani Pane Sapropterin?

Pegvaliase uye sapropterin (Kuvan) zvinoshanda kuburikidza nemaitiro akasiyana uye zvinowanzo shandiswa kune mhando dzakasiyana dzevarwere vePKU, saka kuzvienzanisa zvakananga hakuna kunanga. Sarudzo

Pegvaliase, kune rimwe divi, inoshanda kune vanhu vazhinji vane PKU zvisinei nerudzi rwavo rwemajini, asi inoda majekiseni uye inotakura njodzi yakakura yekugumbuka kwemuviri. Inowanzo fungwa kana manejimendi ekudya uye sapropterin zvisina kusvika padanho re phenylalanine.

Vamwe vanhu vangabatsirwa nekushandisa mishonga yese pamwechete pasi pekutariswa kwechiremba. Chiremba wako achabatsira kuona nzira ipi yakakodzera mamiriro ako chaiwo zvichibva pamiedzo yemajini, mazinga e phenylalanine aripo, uye zvinangwa zvako zvekutariswa.

Mibvunzo Inowanzo Bvunzwa Nezve Pegvaliase

Pegvaliase Yakachengeteka Here Panguva Yemimba?

Kuchengeteka kwe pegvaliase panguva yekuzvitakura hakuna kunyatsoiswa pachena kuburikidza nekudzidza kukuru kwekiriniki. Zvisinei, kuchengetedza mazinga akakodzera e phenylalanine panguva yekuzvitakura kwakakosha kune hutano hwemukadzi nekukura kwemwana.

Mazinga epamusoro e phenylalanine panguva yekuzvitakura anogona kukonzera zvikanganiso zvakakomba zvekuzvarwa zvinosanganisira hurema hwehungwaru, matambudziko emoyo, uye nyaya dzekukura mumwana ari kukura. Mamiriro aya anonzi maternal PKU syndrome, uye anogona kuitika kunyangwe mwana asina PKU.

Kana iwe wakazvitakura kana kuronga kuzvitakura uchitora pegvaliase, shanda pedyo nenyanzvi yako yePKU uye chiremba wako weobstetrician. Vachabatsira kuyera njodzi nemabhenefiti ekuenderera mberi nekurapa vachipesana nekuchinja kune manejimendi ekudya akasimba panguva yekuzvitakura.

Ndoitei Kana Ndikashandisa Zvakawandisa Pegvaliase Netsaona?

Kana iwe ukabaya netsaona pegvaliase yakawanda kupfuura yakarairwa, taura nemupi wako wehutano nekukasika kana kufonera nzvimbo yako yezvinhu zvine chepfu. Usamirira kuti uone kana zviratidzo zvichikura, sezvo kutariswa kwekurapa nekukurumidza kwakakosha nechero kupfuura muyero wemishonga.

Kunyange zvazvo kutora pegvaliase yakawanda zvisingaiti kuti zvikonzere matambudziko anotyisidzira upenyu, zvinogona kuwedzera njodzi yako yemigumisiro yakadai semhinduro dzakakomba panzvimbo yekudzivirira kana mhinduro dzekurwara. Chiremba wako angada kukutarisa zvakanyanya kana kugadzirisa chirongwa chako chemishonga mune ramangwana.

Kuti udzivise kuwandisa kwemishonga netsaona, gara uchitarisa kaviri chiyero chako chakanyorwa usati wadzivirira, shandisa kuwanda kwakakodzera kwemushonga, uye chengeta log yekudzivirira kuti utarise paunenge watora mushonga wako. Kana usina chokwadi nezve chiyero chako, taura nechikwata chako chehutano usati wadzivirira.

Ndingaita Sei Kana Ndikakanganwa Chiyero chePegvaliase?

Kana ukakanganwa chiyero chepegvaliase, tora pakarepo paunorangarira, kunze kwekunge yava nguva yechiyero chako chinotevera. Muchiitiko ichocho, siya chiyero chakakanganwa uye uenderere mberi nechikwata chako chemishonga yenguva dzose.

Usambofa wakatora chiyero kaviri kuti ugadzirise chakakanganwa, sezvo izvi zvinogona kuwedzera njodzi yako yemigumisiro. Kukanganwa chiyero dzimwe nguva hazvingaiti kuti zvikonzere matambudziko nekukasira, asi edza kuchengeta chirongwa chako chenguva dzose sezvinobvira kuti uwane mhedzisiro yakanaka.

Kana iwe uchikanganwa kazhinji zviyero, funga kutumira zviyeuchidzo zvefoni, uchishandisa murongi wemishonga, kana kukumbira nhengo dzemhuri kuti dzikubatsire kukuyeuchidza. Kushandisa mishonga nguva dzose kwakakosha kuchengetedza mazinga akagadzikana e phenylalanine.

Ndingamira Rini Kutora Pegvaliase?

Haufanire kumira kutora pegvaliase usati wataurirana nechikwata chako chehutano. Sezvo PKU iri mamiriro ehupenyu hwese, kumisa pegvaliase zvichaita kuti mazinga ako e phenylalanine akwirezve, zvichikonzera matambudziko ehutano.

Panogona kuva nemamiriro ezvinhu apo chiremba wako anokurudzira kumisa pegvaliase kwenguva pfupi, sekunge panguva yekurwara kwakanyanya, pamberi pekuvhiyiwa, kana kana iwe ukagadzira migumisiro yakakomba. Muzviitiko izvi, chikwata chako chehutano chichakubatsira kubata PKU yako kuburikidza nedzimwe nzira panguva yekuzorora kwekurapa.

Kana muchifunga kumisa mushonga we pegvaliase nekuda kwemigumisiro kana zvimwe zvinokunetsa, taurai zvakananga nachiremba wenyu nezvezvamakasangana nazvo. Vanogona kugadzirisa mushonga wenyu, kupa rubatsiro rwakawedzerwa rwekubata nemigumisiro, kana kutsvaga dzimwe nzira dzekurapa.

Ndingafamba here ndichitora Pegvaliase?

Hongu, unogona kufamba uchitora pegvaliase, asi zvinoda kuronga kuti uve nemishonga yakakwana uye unogona kuchengeta zvakanaka. Nguva dzose takura mishonga yakawanda kana paine kunonoka kwemafambiro kana mamiriro asingatarisirwi.

Paunenge uchibhururuka, isa pegvaliase yako mumutoro wako wekufambisa pamwe chete netsamba yechirongwa kubva kuna chiremba wako ichitsanangura zvaunoda pamushonga uye zvekujekesa. Izvi zvinobatsira kudzivirira matambudziko nekuchengetedzwa kwendege uye zvinovimbisa kuti mushonga wako haurasike nemutoro wakatariswa.

Kufamba kwenyika dzakawanda, tsvaga mitemo yekutakura mishonga yechirongwa kunyika yaunoenda. Dzimwe nyika dzinoda zvinyorwa zvakakosha kana dzine zvirambidzo pamishonga yakati, saka taura ne embassy yavo kana consulate pamberi kuti udzivise matambudziko.