Yini i-Patisiran: Ukusetshenziswa, Umthamo, Imiphumela Eceleni nokuningi

I-Patisiran iyimithi ekhethekile eyenzelwe ukwelapha isimo esingavamile sezakhi zofuzo esibizwa ngokuthi i-hereditary transthyretin-mediated amyloidosis (hATTR). Lesi simo sithinta uhlelo lwakho lwezinzwa lapho amaprotheni anezinkinga akha emzimbeni wakho futhi alimaza izinzwa zakho.

Uma wena noma othile omaziyo etholiwe ukuthi une-hATTR, cishe uzizwa ukhungathekile ngamagama ezokwelapha nezinketho zokwelapha. Lo mhlahlandlela uzokusiza ukuthi uqonde i-patisiran ngamagama alula, ukuze uzizwe unethemba elikhulu ngezinqumo zakho zokunakekelwa kwezempilo.

Yini i-Patisiran?

I-Patisiran iyimithi kadokotela eyingxenye yeqembu lemithi ebizwa ngokuthi i-RNA interference (RNAi) therapeutics. Isebenza ngokuvimbela isibindi sakho ekwenzeni iprotheni enezinkinga ebanga i-hATTR amyloidosis.

Cabanga nge-patisiran njengomthunywa wama-molecular okhuluma namaseli athile esibindini sakho ukuthi ayeke ukukhiqiza iprotheni enezinkinga. Lokhu kusiza ekuvikeleni ukulimala kwezinzwa okuqhubekayo futhi kunganciphisa ukuqhubeka kwezimpawu zakho.

Umuthi uza njengesixazululo esiwuketshezi esinikezwa ngqo emgudwini wakho wegazi ngomjovo we-IV. Uzothola lokhu kwelashwa endaweni yokunakekelwa kwezempilo, ngokuvamile njalo emavikini amathathu.

Isetshenziselwani i-Patisiran?

I-Patisiran ivunyelwe ngokuqondile ukwelapha i-hereditary transthyretin-mediated amyloidosis (hATTR) kubantu abadala. Lesi yisimo esingavamile sezakhi zofuzo lapho umzimba wakho ukhiqiza khona amaprotheni e-transthyretin angajwayelekile aqoqana futhi alimaze izinzwa zakho.

Abantu abane-hATTR bavame ukuzwa ukulimala kwezinzwa okuqhubekayo okuthinta ikhono labo lokuhamba, ukuzwa imizwa ezandleni nasezinyaweni zabo, nokwenza imisebenzi yansuku zonke. Lesi simo singase futhi sithinte inhliziyo yakho, uhlelo lokugaya ukudla, nezinye izitho ngokuqhubeka kwesikhathi.

Udktela wakho uzonika imithi ye-patisiran kuphela uma utholwe ukuthi une-hATTR ngokuhlolwa kwezakhi zofuzo kanye nokuhlolwa okukhethekile. Ayisetshenziselwa ezinye izinhlobo ze-amyloidosis noma izimo zemizwa.

Isebenza Kanjani I-Patisiran?

I-Patisiran isebenza ngokusebenzisa inqubo yezinto eziphilayo eyinkimbinkimbi ebizwa ngokuthi i-RNA interference ukunciphisa ukukhiqizwa kwamaprotheni e-transthyretin angalungile esibindini sakho. Ithathwa njengokwelashwa okuqondiswe kakhulu futhi okuphumelelayo kwe-hATTR.

Isibindi sakho ngokuvamile senza amaprotheni e-transthyretin, kodwa kubantu abane-hATTR, lawa maprotheni awamile kahle futhi athambekele ekunamatheleni ndawonye. Lezi ziqoqwana zamaprotheni bese zihamba ngegazi lakho futhi zifakwe emizweni yakho, enhliziyweni, nakwezinye izicubu, okubangela umonakalo.

I-Patisiran iqukethe izingcezu ezincane zezinto zofuzo eziqondise ngqo imiyalelo amaseli esibindi sakho awasebenzisayo ukwenza i-transthyretin. Lapho i-patisiran ifinyelela esibindini sakho, empeleni itshela amaseli ukuthi ayeke ukulandela le miyalelo engalungile.

Lo muthi unamandla kakhulu ekunciphiseni amazinga e-transthyretin. Ucwaningo lubonisa ukuthi lunganciphisa lawa maprotheni ayingozi ngamaphesenti angaba ngu-80% noma ngaphezulu kubantu abaningi abathola ukwelashwa.

Kufanele Ngiyithathe Kanjani I-Patisiran?

I-Patisiran inikezwa njenge-intravenous (IV) infusion njalo emavikini amathathu endaweni yokunakekelwa kwezempilo. Awukwazi ukuthatha lo muthi ekhaya, njengoba kudinga ukuqapha ngokucophelela nokulungiswa okukhethekile.

Ngaphambi kwe-infusion ngayinye, uzothola imithi yangaphambi kokwelashwa ukusiza ukuvimbela ukusabela komzimba. Lokhu kuvamile ukuhlanganisa i-antihistamine, i-corticosteroid, kanye nomuthi wokudambisa ubuhlungu. Ithimba lakho lokunakekelwa kwezempilo lizokunika le mithi cishe imizuzu engama-60 ngaphambi kwe-patisiran infusion yakho.

I-patisiran infusion yangempela ithatha imizuzu engaba ngu-80 ukuqedela. Ngesikhathi esifanayo, uzoqashwa nganoma yiziphi izimpawu zokusabela kwe-infusion, okungabandakanya ukukhanya, ukuphefumula okuncane, noma ukungakhululeki esifubeni.

Akudingeki ukuthi ulandele imikhawulo ekhethekile yokudla ngaphambi kokwelashwa kwakho. Nokho, kubalulekile ukuhlala unamanzi kahle futhi uthole ukuphumula okwanele ngaphambi kokuqokwa ngakunye kokufakwa.

Kufanele Ngithathe Isikhathi Esingakanani I-Patisiran?

I-Patisiran ngokuvamile iyindlela yokwelapha yesikhathi eside ozoqhubeka nayo uma nje isiza ukulawula izimpawu zakho ze-hATTR futhi umzimba wakho uyayibekezelela kahle. Abantu abaningi badinga ukwelashwa okuqhubekayo ukuze bagcine izinzuzo.

Udokotela wakho uzoqapha inqubekela phambili yakho ngokuhlolwa kwegazi okuvamile, ukuhlolwa komsebenzi wemizwa, nokuhlolwa kwekhwalithi yempilo. Lokhu kuhlolwa kusiza ukunquma ukuthi i-patisiran isebenza ngempumelelo yini kuwe.

Abanye abantu baqala ukuqaphela ukuthuthuka ezimpawini zabo ezinyangeni ezimbalwa nje ngemva kokuqala ukwelashwa, kanti abanye bangase bathathe isikhathi eside ukubona izinzuzo. Umuthi usebenza kancane kancane, ngakho ukubekezela kubalulekile ngesikhathi sokuqala sokwelashwa.

Ithimba lakho lezempilo lizoxoxa nawe ngezinhloso zokwelashwa futhi lilungise uhlelo lwakho lokunakekela ngokusekelwe ekutheni usabela kanjani emuthini kanye nanoma yimaphi imiphumela emibi ongase uyithole.

Yiziphi Imiphumela Emibi ye-Patisiran?

Njengayo yonke imithi, i-patisiran ingabangela imiphumela emibi, nakuba kungathi akubona bonke abantu abayitholayo. Imiphumela emibi eminingi iyalawuleka, futhi ithimba lakho lezempilo lizokuqapha eduze ngesikhathi sokwelashwa.

Imiphumela emibi evame kakhulu ongase uyithole ihlanganisa ukusabela ngesikhathi noma ngemva nje kokufakwa kwakho. Lokhu kungase kuzwakale kukhathaza, kodwa ithimba lakho lezokwelapha lilungele kahle ukulisingatha uma kwenzeka.

Imiphumela emibi ejwayelekile ihlanganisa:

• Ukuvuvukala ezingalweni zakho noma emilenzeni (i-peripheral edema)
• Ukusabela okuhlobene nokufakwa njengokukhukhula, ubuhlungu emhlane, noma isicanucanu
• Ukutheleleka kwepheshana eliphezulu lokuphefumula
• Ama-spasms emisipha noma ama-cramps
• Ubuhlungu emalungeni
• Ukukhathala noma ukuzizwa ukhathele kunokujwayelekile
• Isiyezi noma ukuzwa ukukhanya ekhanda
• Ukubomvu kwesikhumba noma ukuqubuka

Lezi zimo ezivamile ezithinta umzimba ngokuvamile zithambile kuya kokulingene futhi zivame ukuthuthuka njengoba umzimba wakho ujwayela ukwelashwa. Ithimba lakho lezempilo linganikeza amasu okusiza ukuphatha lezi zimpawu.

Imiphumela emibi engathi sína kodwa engavamile ingabandakanya ukusabela okukhulu komzimba ngesikhathi sokufakwa kwemithi, ukwehla okukhulu kwengcindezi yegazi, noma ukuphuma kwegazi okungavamile. Ngenkathi lokhu kungavamile, ithimba lakho lezokwelapha liyakubheka ngaso sonke isikhathi sokwelashwa.

Ngokungavamile kakhulu, abanye abantu bangase babe namazinga e-vitamin A anciphisiwe egazini labo, okungathinta umbono ebusuku. Udokotela wakho uzoqapha amazinga akho e-vitamin A futhi angase ancome izithasiselo uma kudingeka.

Ubani Ongafanele Athole i-Patisiran?

I-Patisiran ayifanele wonke umuntu, ngisho nalabo abane-hATTR amyloidosis. Udokotela wakho uzoqapha ngokucophelela ukuthi ngabe lo muthi ulungile yini esimweni sakho esithile.

Akufanele uthole i-patisiran uma uke waba nokusabela okukhulu komzimba emuthini noma kunoma yiziphi izithako zawo esikhathini esidlule. Lokhu kufaka phakathi ukusabela okufana nobunzima bokuphefumula, ukuvuvukala okukhulu, noma ukwehla okuyingozi kwengcindezi yegazi.

Abantu abanezimo ezithile zezokwelapha bangase badinge ukuqashwa okukhethekile noma bangase bangabi ngabazongenela ukwelashwa nge-patisiran. Udokotela wakho uzodinga ukwazi ngomlando wakho wezokwelapha ophelele ngaphambi kokuqala ukwelashwa.

Izimo ezidinga ukucatshangelwa ngokucophelela zihlanganisa:

• Izinkinga zenhliziyo ezinzima noma ukuhlaselwa yinhliziyo kwakamuva
• Izifo ezisebenzayo noma amasosha omzimba ancishisiwe
• Isifo sesibindi ngaphandle kwalokho okubangelwa yi-hATTR
• Ukuphuma kwegazi noma ukusetshenziswa kwamanje kwama-blood thinners
• Ukukhulelwa noma izinhlelo zokukhulelwa
• Ukuncelisa

Uma ukhulelwe noma uhlela ukukhulelwa, xoxa ngalokhu nodokotela wakho ngokushesha. Imiphumela ye-patisiran ekukhulelweni ayaziwa ngokugcwele, ngakho-ke ukucatshangelwa ngokucophelela kuyadingeka.

Amagama Omkhiqizo we-Patisiran

I-Patisiran itholakala ngaphansi kwegama lomkhiqizo elithi Onpattro. Leli yigama lomkhiqizo elivunyelwe kuphela lolu hlobo lomuthi emazweni amaningi, okuhlanganisa i-United States ne-Europe.

Uma uthola ukwelashwa kwakho, ibhodlela lomuthi lizobhalwa ngokuthi Onpattro, futhi leli yigama ozolibona emarekhodini akho ezokwelapha kanye nemibhalo yomshuwalense. Okwamanje awekho amagama ajwayelekile e-patisiran atholakalayo.

Uma uhamba noma uthola ukunakekelwa ezikhungweni ezihlukene, hlala ukhuluma ngamagama omuthi ajwayelekile (patisiran) negama lomkhiqizo (Onpattro) ukuze kuqinisekiswe ukuxhumana okucacile nabanakekeli bakho bezempilo.

Ezinye Izindlela Zokwelapha I-Patisiran

Ngenkathi i-patisiran isebenza kahle kakhulu ku-hATTR amyloidosis, zikhona ezinye izindlela zokwelapha ezitholakalayo. Udokotela wakho angase acabange ezinye izindlela ngokusekelwe ezimpawini zakho ezithile, ukuqhubeka kwesifo, kanye nezimo zomuntu ngamunye.

I-Inotersen ngomunye umuthi osuselwe ku-RNA onikezwa njengesijovo esingaphansi kwesikhumba masonto onke. Isebenza ngendlela efana ne-patisiran kodwa inezinye izinto ezingezinhle futhi ineshejuli yokunikezwa ehlukile.

I-Tafamidis ngumuthi omlomo osiza ukuzinzisa amaprotheni e-transthyretin, ukuwavimbela ukuthi angazigobi ngendlela engafanele. Ilungele ikakhulukazi abantu abanezinkinga zenhliziyo ezivela ku-hATTR.

Ezinye izindlela zokwelapha ezisekelayo zingabandakanya:

• Imithi yokwelapha ubuhlungu bemizwa nezimpawu
• Ukwelashwa ngokomzimba nokwelashwa komsebenzi
• Ukusekelwa kokudla okunomsoco kanye nezithasiselo
• Imithi yenhliziyo uma kukhona ukuhileleka kwenhliziyo
• Ukufakelwa isibindi ezimweni ezinzima kakhulu

Ithimba lakho lezempilo lizosebenza nawe ukunquma indlela engcono kakhulu yokwelapha ngokusekelwe ohlotsheni lwakho oluthile lwe-hATTR, ubunzima bezimpawu, nesimo sempilo yakho esiphelele.

Ingabe i-Patisiran Ingcono Kune-Inotersen?

Kokubili i-patisiran ne-inotersen ziyizindlela zokwelapha eziphumelelayo ze-hATTR amyloidosis, kodwa zinezinto ezihlukene eziyinzuzo kanye nezinto okufanele zicatshangelwe. Ukukhetha okuthi "kungcono" kuncike ezimweni zakho kanye nezintandokazi zakho.

I-Patisiran inikezwa njalo ngemva kwamaviki amathathu ngokufakwa kwe-IV endaweni yokunakekelwa kwezempilo. Lokhu kusho izinsuku zokwelashwa ezimbalwa ngonyaka, kodwa uzodinga ukuvakashela umtholampilo ngomthamo ngamunye. Umuthi ubonise imiphumela emihle ezivivinyweni zomtholampilo ngokunciphisa okukhulu ekuqhubekeleni phambili kwesifo.

I-Inotersen inikezwa njengesijovo samaviki onke ngaphansi kwesikhumba ongakwazi ukuzifundela ukuzinika ekhaya. Lokhu kunikeza lula nokuguquguquka okwengeziwe, kodwa kudinga ukulinganiswa okuvame kakhulu kanye nokuqapha njalo kwegazi ngenxa yemiphumela engaba khona kuma-platelets kanye nomsebenzi wezinso.

Ngokuqondene nokusebenza kahle, yomibili imithi inciphisa kakhulu ukukhiqizwa kwamaprotheni e-transthyretin ayingozi. Izifundo zikhomba ukuthi zombili zisebenza kahle kakhulu, yize izifundo zokuqhathanisa ngokuqondile zilinganiselwe.

Udokotela wakho uzokusiza ukuthi uhlole izinto ezifana nokunethezeka kwakho ngezindlela ezihlukene zokuphatha, izidingo zokuhamba, isimiso somsebenzi, kanye nezinto eziyingozi zemiphumela emibi ethile lapho ukhetha phakathi kwalezi zinketho.

Imibuzo Evame Ukubuzwa Mayelana ne-Patisiran

Ingabe i-Patisiran iphephile kubantu abanezinkinga zenhliziyo?

I-Patisiran ingasetshenziswa kubantu abahileleke enhliziyweni evela ku-hATTR amyloidosis, kodwa idinga ukuqapha ngokucophelela. Abantu abaningi abane-hATTR bathuthukisa izinkinga zenhliziyo njengengxenye yesimo sabo, ngakho-ke lokhu kuyinkathazo ejwayelekile.

Udokotela wakho wenhliziyo kanye nochwepheshe be-hATTR bazosebenzisana ukuqapha ukusebenza kwenhliziyo yakho ngaso sonke isikhathi sokwelashwa. Bazosebenzisa izivivinyo ezinjenge-echocardiograms kanye nezimpawu zegazi ukulandelela ukuthi inhliziyo yakho isabela kanjani ekwelashweni.

Abanye abantu empeleni babona ngcono ekusebenzeni kwenhliziyo yabo ngemuva kokuqala i-patisiran, njengoba ukunciphisa amazinga amaprotheni ayingozi kunganciphisa ukulimala kwenhliziyo okuqhubekayo. Kodwa-ke, ukulimala kwenhliziyo okukhona kungenzeka kungabi ngaphendukayo.

Kufanele Ngenzeni Uma Ngamukela I-Patisiran Eningi Ngengozi?

Ukweqisa kwe-patisiran akunakwenzeka ngoba umuthi ulungiswa futhi unikezwa ochwepheshe bezempilo abaqeqeshiwe endaweni elawulwayo. Umthamo ubhalwa ngokucophelela ngokuya ngesisindo somzimba wakho futhi unikezwa kancane kancane cishe imizuzu engama-80.

Uma ukhathazekile ngomthamo wakho noma uba nezimpawu ezingavamile ngemuva kokufakwa, xhumana nethimba lakho lezempilo ngokushesha. Bangahlola isimo sakho futhi banikeze ukuqapha okufanele noma ukwelashwa uma kudingeka.

Isikhungo sezempilo lapho uthola khona ukwelashwa sizoba nezimiso zokubhekana nanoma yimaphi amaphutha emithi noma ukusabela okungase kwenzeke ngesikhathi sokufakwa kwakho.

Kufanele Ngenzeni Uma Ngedlula Umthamo We-Patisiran?

Uma udlula ukufakwa kwakho kwe-patisiran okuhleliwe, xhumana nethimba lakho lezempilo ngokushesha ngangokunokwenzeka ukuze uhlele kabusha. Kubalulekile ukugcina izikhawu zokwelashwa ezihambisanayo uma kungenzeka ukuze ugcine amazinga akho amaprotheni e-transthyretin ecindezelwe.

Udokotela wakho angase alungise uhlelo lwakho lokwelashwa kancane ukuze kulungiswe umthamo odliwe, kodwa zama ukubuyela emkhondweni ngezikhawu zakho ezivamile zamasonto amathathu. Ungakhathazeki uma udinga ukudlula umthamo ngezikhathi ezithile ngenxa yokugula noma ezinye izimo.

Uma uvame ukudlula imithamo ngenxa yezingxabano zokuhlela noma ezinye izinkinga, xoxa ngalokhu nethimba lakho lezempilo. Bangase bakwazi ukukusiza ukuthi uthole izixazululo noma ucabangele ezinye izindlela zokwelapha.

Ngingayeka Nini Ukuthatha I-Patisiran?

Isinqumo sokuyeka i-patisiran kufanele senziwe ngaso sonke isikhathi ngokubonisana nodokotela wakho onguchwepheshe we-hATTR amyloidosis. Abantu abaningi badinga ukuqhubeka nokwelashwa isikhathi eside ukuze bagcine izinzuzo futhi bavimbele ukuqhubeka kwesifo.

Udkt wakho uzohlola njalo ukuthi usabela kanjani emuthini ngokusebenzisa izivivinyo zomsebenzi wemizwa, ukuhlolwa kwekhwalithi yempilo, kanye nokubheka amazinga akho amaprotheni e-transthyretin. Lokhu kuhlola kusiza ukunquma ukuthi ukuqhubeka nokwelashwa kuyazuzisa yini.

Ezinye izizathu ezingase zikwenze uyeke ukuthatha i-patisiran zihlanganisa imiphumela emibi engathuthuki ngokuphathwa, ukungabi bikho kwenzuzo ngemva kwesikhathi esanele sokuzama, noma uma isimo sakho sempilo yonke sishintsha kakhulu.

Uma uyeka ukuthatha i-patisiran, amazinga akho amaprotheni e-transthyretin cishe azobuyela emazingeni angaphambilini phakathi nezinyanga ezimbalwa, futhi izimpawu zakho ze-hATTR zingase ziqale ukuqhubeka futhi.

Ngingahamba Ngesikhathi Ngithatha I-Patisiran?

Yebo, ungahamba ngenkathi uthola ukwelashwa nge-patisiran, kodwa kudinga ukuhlela okuthile. Njengoba udinga ukufakwa njalo ngemva kwamasonto amathathu, kuzodingeka ukuthi uhlele uhambo lwakho ngokuya ngokuqokwa kwakho kokwelashwa.

Uma uhlela uhambo olude, xoxa ngalokhu nethimba lakho lezempilo kusenesikhathi. Bangase bakwazi ukukusiza ukuthi uhlele ukwelashwa kwesikhashana endaweni eseduze nendawo oya kuyo, nakuba lokhu kungaba nzima ngenxa yohlobo olukhethekile lomuthi.

Ohambweni olufushane, ungase ukwazi ukulungisa uhlelo lwakho lokwelashwa kancane ukuze luvumelane nezinhlelo zakho zokuhamba. Hlala uphethe imibhalo mayelana nesimo sakho nokwelashwa uma uhamba, ikakhulukazi uma uwela imingcele yamazwe ngamazwe.

Khumbula ukuthi i-patisiran idinga ukuqandiswa nokusingathwa okukhethekile, ngakho awukwazi ukuyithutha ngokwakho. Zonke izinhlelo kumele zenziwe ngezikhungo zezempilo eziqinisekisiwe.