Singakhuluma ngomuthi we-Amvuttra wesifo senhliziyo obizwa ngokuthi i-ATTR-CM? (ATTR-CM kanye ne-Amvuttra)

Kungenzeka ukuthi awukaze uzwe ngezifo ezenzeka lapho uhlobo oluthile lweprotheyini emzimbeni wethu luqoqana ngendlela engafanele futhi lufakwa ezithweni ezibalulekile njengenhliziyo nemizwa. Lezi zivame ukuba yizifo ezingavamile. Kwezokwelapha, lolu hlobo lokufakwa kwamaprotheni lubizwa ngokuthi i-amyloidosis . Namuhla, sizokhuluma ngesifo esingavamile esibizwa ngokuthi i-ATTR-CM, esithinta ngqo inhliziyo, okungukuthi, lapho la maprotheni efakwa khona emisipheni yenhliziyo, kanye nomuthi wesimanje obizwa ngokuthi i-Amvuttra osetshenziswa kuwo.

Iyini ngempela i-ATTR-CM?

Kalula nje, i-ATTR-CM yisimo lapho, ngenxa yesici sofuzo noma okwenzeka ngokwemvelo ngokuguga, iphrotheni i-TTR, ekhiqizwa emzimbeni wethu, ishintsha isimo futhi ihlangane ndawonye bese izibeka ezindongeni zemisipha yenhliziyo. Igama eliphelele lalokhu yi-Transthyretin Amyloid Cardiomyopathy.

Cabanga ngakho, izindonga zenhliziyo yethu kudingeka ziguquguquke ukuze zikwazi ukupompa kahle. Kodwa uma la maprotheni eqoqana, izindonga zenhliziyo ziyaqina futhi ziqine . Lokhu sikubiza ngokuthi i-cardiomyopathy. Lokhu kwenza inhliziyo ingakwazi ukupompa kahle igazi. Ngokuhamba kwesikhathi, lokhu kungaqhubekela phambili ekuhlulekeni kwenhliziyo.

Abanye abantu abane-ATTR-CM ababi nazo izimpawu, kodwa abanye baba nezimpawu ezifana nezesifo senhliziyo esivamile.

• Ukuphefumula okuncane (i-dyspnea)
• Ukuvuvukala, ikakhulukazi emilenzeni nasemaqakaleni
• Ukudideka noma ukuphelelwa yithemba
• Ukwanda kwesilinganiso senhliziyo noma ukushaya kwenhliziyo okungajwayelekile
• Ubuhlungu besifuba

Lokhu kungakwenza uzizwe ukhathele ngokushesha lapho wenza izinto ezifana nokukhwela izitebhisi, ukwenza imisebenzi yasekhaya, noma ukuhamba ibanga elifushane.

Ngaphezu kwalokho, kungase kube nezinye izimpawu ezingahlobene nenhliziyo. Isibonelo, ubuthakathaka, ukungazweli noma ubuhlungu ezingalweni nasemilenzeni, ukujuluka ngokweqile, isiyezi noma ukuquleka, kanye nezinkinga zokugaya ukudla.

Umuthi we-Amvuttra usebenza kanjani?

Ukuze siqonde lokhu, sidinga ukwazi okuncane ngeprotheni ebizwa ngokuthi i-TTR. I-TTR (i-Transthyretin) yiprotheni ekhiqizwa kakhulu esibindini sethu. Umsebenzi wayo oyinhloko ukuthutha ama-hormone e-thyroid kanye ne-vitamin A emzimbeni wonke.

Kubantu abane-ATTR-CM, isici sofuzo sibangela ukuthi iphrotheni ye-TTR ikhiqizwe ngesimo esingalungile. Lesi simo esingalungile sephrotheni siyaqoqana futhi sihlale enhliziyweni.

Umuthi i-Amvuttra usebenza njengesithulisi sezakhi zofuzo. Okusho ukuthi, lo muthi uya ngqo esibindini futhi uvimbele leyo phrotheni ye-TTR enephutha.Kunciphisa umsebenzi wejini edalwayo. Kufana nokunciphisa ivolumu yomsakazo.

I-Amvuttra iwumjovo ongaphansi kwesikhumba. Lo mjovo uvame ukunikezwa uchwepheshe wezempilo njalo ezinyangeni ezintathu .

Impumelelo yalo muthi yahlolwa kanjani?

Njenganoma yimuphi umuthi omusha, ukusebenza kahle kwe- AMVUTTRA kuqinisekiswe ngocwaningo olukhulu. Kulolu cwaningo, iziguli ezingu-655 ze-ATTR-CM zanikezwa umuthi wokugomela i-Amvuttra kanti elinye iqembu lanikezwa umuthi wokugomela i-placebo ongenawo umuthi njalo ezinyangeni ezingu-12 iminyaka emithathu.

Abacwaningi babheke izici eziningana ukuze bahlole ukuthi lo muthi uyasebenza yini.

• Ukuhlolwa kokuhamba ngezinyawo kwemizuzu eyisithupha: Lokhu kulinganisa ukuthi ungahamba ibanga elingakanani ngesivinini esijwayelekile ngemizuzu eyi-6. Leli banga liyancipha njengoba isimo senhliziyo yakho siba sibi kakhulu.
• Ukuhlukaniswa kwe-New York Heart Association (NYHA): Lokhu kulinganisa ubukhulu bezimpawu zokwehluleka kwenhliziyo. Kunezigaba ezine (I, II, III, IV). Uma inani lesigaba liphezulu, kulapho isifo siba sibi kakhulu.

• Imibuzo Yekhwalithi Yokuphila (KCCQ-OS): Lokhu kulinganisa umthelela wesifo kwikhwalithi yokuphila kwesiguli.

Ucwaningo luthole ini?

Ekupheleni kwesikhathi sokwelashwa seminyaka emithathu, abacwaningi bahlaziya imiphumela.

Okutholakele okuyinhloko kwaba ukuthi iziguli ezathola i-Amvuttra zazinengozi ephansi ngo-28% yokufa noma yokulaliswa esibhedlela ngokuzumayo noma yokuvakashela igumbi lezimo eziphuthumayo (i-ETU) ngenxa yokwehluleka kwenhliziyo , uma kuqhathaniswa nalabo abathola i-placebo (umuthi wokugomela ongelona iqiniso).

Enye into ebalulekile ukuthi ekupheleni kwezinyanga ezingu-42, ingozi yokufa nganoma yisiphi isizathu yehliswe ngo-35% eqenjini elathola i-Amvuttra.

Ngaphezu kwalokho, iziguli ezathola i-Amvuttra zazinokwehla okulawulwayo kwamandla okuhamba kanye nekhwalithi yokuphila.

Kodwa khumbula, le miphumela yocwaningo ingase ingafani kuwo wonke umuntu. Wena nodokotela wakho kuzodingeka ninqume ndawonye ukuthi i-Amvuttra ikufanele yini nokuthi izinzuzo zayo zidlula yini imiphumela emibi.

Ake sifunde ngemiphumela kanye nemiphumela emibi.

Kuthatha isikhathi esingakanani ukubona imiphumela?

I-Amvuttra umuthi osebenza ngokuhamba kwesikhathi. Ngakho-ke, imiphumela ayibonakali ngokushesha. Kungathatha izinyanga eziningana noma ngaphezulu ukubona ukuthuthuka kwezimpawu.

Ungayiphatha kanjani imiphumela emibi?

Omunye wemiphumela emibi eyinhloko ye-Amvuttra ukwehla kwamazinga e-vitamin A egazini. Lokhu kungenxa yokuthi iphrotheni ye-TTR yiyo ethwala i-vitamin A emzimbeni wonke. Uma umuthi unciphisa ukukhiqizwa kwe-TTR, ukuthuthwa kwe-vitamin A nakho kuyancipha.

• Udokotela wakho uzohlala e-oda ukuhlolwa kwegazi ukuze ahlole amazinga akho e-vitamin A. Kubaluleke kakhulu ukwenza lokhu kuhlolwa ngesikhathi.
• Faka ukudla okucebile ngevithamini A (isib. izaqathi, ubhatata, isipinashi, amaqanda, ubisi, upelepele obomvu ) ekudleni kwakho.
• Uma ubona noma yiziphi izinguquko embonweni wakho (ikakhulukazi ukwehla kombono wasebusuku), yazisa udokotela wakho ngokushesha.
• Kuye ngezinga lakho levithamini A, udokotela wakho angase ancome ukuthi uthathe isithasiselo sevithamini A.

Lena akuyona yodwa imiphumela emibi ye-Amvuttra. Uma unezinye izimpawu ezikukhathazayo, qiniseka ukuthi ukhuluma nodokotela wakho .