He aha ʻo Patisiran: Nā Hoʻohana, Nā Ana, Nā ʻAoʻao ʻAoʻao a me nā mea hou aku

He lāʻau lapaʻau kūikawā ʻo Patisiran i hoʻolālā ʻia e mālama i kahi maʻi genetic laha ʻole i kapa ʻia ʻo hereditary transthyretin-mediated amyloidosis (hATTR). Hoʻopilikia kēia maʻi i kāu ʻōnaehana nerve ke kūkulu ʻia nā protein hewa i loko o kou kino a hōʻino i kou mau aʻalolo.

Inā ua ʻike ʻia ʻoe a i ʻole kekahi āu e ʻike nei me hATTR, e manaʻo ana paha ʻoe i ka nui o nā huaʻōlelo lapaʻau a me nā koho lapaʻau. E kōkua kēia alakaʻi iā ʻoe e hoʻomaopopo iā patisiran ma nā huaʻōlelo maʻalahi, i hiki iā ʻoe ke manaʻoʻiʻo e pili ana i kāu mau hoʻoholo mālama ola.

He aha ʻo Patisiran?

He lāʻau lapaʻau ʻo Patisiran i loaʻa i ka papa o nā lāʻau i kapa ʻia ʻo RNA interference (RNAi) therapeutics. Hana ia ma ka hoʻōki ʻana i kou ate mai ka hana ʻana i ka protein hewa e hana ai i ka hATTR amyloidosis.

E noʻonoʻo iā patisiran ma ke ʻano he ʻelele molecular e haʻi ana i nā cell kikoʻī i kou ate e hoʻōki i ka hana ʻana i ka protein pilikia. Kōkua kēia i ka pale ʻana i ka pōʻino nerve hou a hiki ke hoʻolohi i ka holomua o kāu mau hōʻailona.

Hele mai ka lāʻau lapaʻau ma ke ʻano he wai wai i hāʻawi pololei ʻia i loko o kou kahe koko ma o ka IV infusion. E loaʻa iā ʻoe kēia lapaʻau ma kahi keʻena mālama ola, maʻamau i kēlā me kēia ʻekolu pule.

No ke aha i hoʻohana ʻia ai ʻo Patisiran?

Ua ʻae ʻia ʻo Patisiran e mālama i ka hereditary transthyretin-mediated amyloidosis (hATTR) i nā pākeke. He maʻi genetic laha ʻole kēia kahi e hana ai kou kino i nā protein transthyretin abnormal e hui pū a hōʻino i kou mau aʻalolo.

ʻO ka poʻe me hATTR e ʻike pinepine i ka pōʻino nerve holomua e pili ana i ko lākou hiki ke hele, manaʻo i nā manaʻo i ko lākou mau lima a me nā wāwae, a hana i nā hana i kēlā me kēia lā. Hiki nō hoʻi i ke kūlana ke hoʻopilikia i kou puʻuwai, ʻōnaehana digestive, a me nā ʻōkana ʻē aʻe i ka manawa.

E kuhikuhi wale nō kāu kauka i ka patisiran inā ua ʻike ʻia ʻoe me ka hATTR ma o ka hoʻāʻo genetic a me nā hoʻokolokolo kūikawā ʻē aʻe. ʻAʻole hoʻohana ʻia ia no nā ʻano amyloidosis ʻē aʻe a i ʻole nā maʻi nerve.

Pehea e Hana ai ʻo Patisiran?

Hana ʻo Patisiran ma ka hoʻohana ʻana i kahi hana olaola paʻakikī i kapa ʻia ʻo RNA interference e hōʻemi i ka hana ʻana o nā protein transthyretin hewa i loko o kou ate. Manaʻo ʻia he lapaʻau i manaʻo nui ʻia a maikaʻi no ka hATTR.

Hana maʻamau kou ate i nā protein transthyretin, akā i ka poʻe me ka hATTR, ua hewa kēia mau protein a pili pū kekahi. A laila hele kēia mau puʻupuʻu protein ma kou kahe koko a waiho i loko o kou mau aʻalolo, ka puʻuwai, a me nā ʻiʻo ʻē aʻe, e hōʻeha ai.

Aia i loko o Patisiran nā ʻāpana liʻiliʻi o nā mea genetic e kuhikuhi pono i nā ʻōlelo a kou mau keena ate e hoʻohana ai e hana i ka transthyretin. Ke hiki ka patisiran i kou ate, haʻi maoli ia i nā keena e hooki i ka hahai ʻana i kēia mau ʻōlelo hewa.

He mana nui kēia lāʻau lapaʻau i ka hōʻemi ʻana i nā pae transthyretin. Hōʻike nā haʻawina hiki iā ia ke hoʻemi i kēia mau protein ʻino ma kahi o 80% a ʻoi aku paha i ka hapa nui o ka poʻe e loaʻa ana i ka lapaʻau.

Pehea wau e lawe ai i ka Patisiran?

Hāʻawi ʻia ʻo Patisiran ma ke ʻano he intravenous (IV) infusion i kēlā me kēia ʻekolu pule ma kahi keʻena mālama ola. ʻAʻole hiki iā ʻoe ke lawe i kēia lāʻau lapaʻau ma ka home, no ka mea, pono ia i ka nānā pono ʻana a me ka hoʻomākaukau kūikawā.

Ma mua o kēlā me kēia infusion, e loaʻa iā ʻoe nā lāʻau lapaʻau e kōkua i ka pale ʻana i nā hopena maʻi. ʻO kēia mau mea e pili ana i kahi antihistamine, kahi corticosteroid, a me kahi mea hoʻomaha ʻeha. E hāʻawi kāu hui mālama ola iā ʻoe i kēia mau lāʻau lapaʻau ma kahi o 60 mau minuke ma mua o kāu patisiran infusion.

Lawe ka patisiran infusion maoli ma kahi o 80 mau minuke e hoʻopau. I kēia manawa, e nānā ʻia ʻoe no nā hōʻailona o nā hopena infusion, hiki ke komo i ka ʻulaʻula, ka pōkole o ka hanu, a i ʻole ka ʻoluʻolu o ka umauma.

ʻAʻole pono ʻoe e hahai i nā palena meaʻai kūikawā ma mua o kāu lapaʻau. Akā naʻe, he mea nui e noho maikaʻi i ka wai a e hoʻomaha pono ma mua o kēlā me kēia manawa o ka hoʻokomo ʻana.

Pehea ka lōʻihi e lawe ai au i ka Patisiran?

He lapaʻau lōʻihi ka Patisiran, e hoʻomau ʻoe no ka lōʻihi e kōkua ana i ka mālama ʻana i kāu mau hōʻailona hATTR a ke ʻae maikaʻi nei kou kino. Pono ka hapa nui o nā kānaka i ka lapaʻau mau e mālama i nā pōmaikaʻi.

E nānā kāu kauka i kou holomua ma o nā hoʻokolohua koko maʻamau, nā loiloi hana nerve, a me nā loiloi maikaʻi o ke ola. Kōkua kēia mau nānā ʻana e hoʻoholo inā e hana pono ana ka patisiran iā ʻoe.

Hoʻomaka kekahi poʻe e ʻike i nā hoʻomaikaʻi ʻana i kā lākou mau hōʻailona i loko o kekahi mau mahina ma hope o ka hoʻomaka ʻana i ka lapaʻau, ʻoiai e lōʻihi ana ka poʻe ʻē aʻe e ʻike i nā pōmaikaʻi. Hana mālie ka lāʻau lapaʻau, no laila he mea nui ka hoʻomanawanui i ka wā o ka lapaʻau mua.

E kūkākūkā kāu hui mālama ola i nā pahuhopu lapaʻau me ʻoe a e hoʻoponopono i kāu hoʻolālā mālama e pili ana i ke ʻano o kou pane ʻana i ka lāʻau lapaʻau a me nā hopena ʻaoʻao āu e ʻike ai.

He aha nā hopena ʻaoʻao o Patisiran?

E like me nā lāʻau lapaʻau a pau, hiki i ka patisiran ke hana i nā hopena ʻaoʻao, ʻoiai ʻaʻole ʻike nā kānaka a pau iā lākou. Hiki ke mālama ʻia ka hapa nui o nā hopena ʻaoʻao, a e nānā pono kāu hui mālama ola iā ʻoe i ka wā o ka lapaʻau.

ʻO nā hopena ʻaoʻao maʻamau āu e ʻike ai, ʻo ia nā hopena i ka wā a i ʻole ma hope koke o kāu hoʻokomo ʻana. Hiki ke hopohopo kēia mau mea, akā ua mākaukau kāu hui lapaʻau e mālama iā lākou inā e hana lākou.

ʻO nā hopena ʻaoʻao maʻamau e komo pū ana:

• Ka pehu ʻana i kou mau lima a i ʻole nā wāwae (peripheral edema)
• Nā hopena e pili ana i ka hoʻokomo ʻana e like me ka ʻulaʻula, ka ʻeha hope, a i ʻole ka nausea
• Nā maʻi o ka ʻōnaehana hanu luna
• Nā spasms ʻiʻo a i ʻole nā ʻūhā
• ʻEha hui
• ʻO ka luhi a i ʻole ka manaʻo e luhi pinepine ana ma mua o ka maʻamau
• ʻO ka ʻūlū a i ʻole ka māmā
• ʻO ka ʻulaʻula ʻili a i ʻole ka ʻōpala

ʻO kēia mau hopena ʻaoʻao maʻamau he maʻalahi a he kūpono pinepine a hoʻomaikaʻi pinepine i ka wā e hoʻoponopono ai kou kino i ka lāʻau lapaʻau. Hiki i kāu hui mālama ola ke hāʻawi i nā hoʻolālā e kōkua i ka mālama ʻana i kēia mau hōʻailona.

Hiki i nā hopena ʻaoʻao koʻikoʻi akā kakaʻikahi ke komo i nā hopena maʻi maʻi koʻikoʻi i ka wā o ka hoʻokomo ʻana, nā hāʻule nui i ke koko, a i ʻole ke kahe koko ʻano ʻē. ʻOiai ʻaʻole maʻamau kēia mau mea, nānā kāu hui lapaʻau iā lākou i kēlā me kēia lāʻau lapaʻau.

I kekahi manawa, hiki i kekahi poʻe ke ʻike i ka emi ʻana o nā pae o ka huaora A i loko o ko lākou koko, hiki ke hoʻopilikia i ka ʻike pō. E nānā kāu kauka i kāu mau pae huaora A a hiki ke ʻōlelo i nā mea hoʻohui inā pono.

ʻO wai ka mea ʻaʻole pono e lawe iā Patisiran?

ʻAʻole kūpono ʻo Patisiran no nā mea a pau, ʻoiai ka poʻe me ka hATTR amyloidosis. E loiloi pono kāu kauka inā kūpono kēia lāʻau lapaʻau no kāu kūlana kikoʻī.

ʻAʻole pono ʻoe e loaʻa iā patisiran inā ua loaʻa iā ʻoe kahi hopena maʻi maʻi koʻikoʻi i ka lāʻau lapaʻau a i ʻole kekahi o kāna mau mea i ka wā i hala. Aia kēia i nā hopena e like me ka pilikia o ka hanu ʻana, ka pehu nui ʻana, a i ʻole nā hāʻule weliweli i ke koko.

Pono paha ka poʻe me kekahi mau maʻi lapaʻau i ka nānā ʻana kūikawā a i ʻole ʻaʻole paha he moho no ka lāʻau lapaʻau patisiran. Pono kāu kauka e ʻike e pili ana i kāu mōʻaukala olakino piha ma mua o ka hoʻomaka ʻana i ka lāʻau lapaʻau.

ʻO nā kūlana e pono ai ka noʻonoʻo pono e komo pū ana:

• Nā pilikia o ka naʻau koʻikoʻi a i ʻole nā puʻuwai puʻuwai hou
• Nā maʻi hana a i ʻole ka ʻōnaehana pale i hoʻopilikia ʻia
• Maʻi ate ma mua o ka mea i hana ʻia e hATTR
• Nā maʻi kahe koko a i ʻole ka hoʻohana ʻana i nā mea lahilahi koko
• Hāpai a i ʻole nā hoʻolālā e hāpai
• Hānai ʻana i ka waiū

Inā hāpai ʻoe a i ʻole e hoʻolālā ana e hāpai, e kūkākūkā i kēia me kāu kauka i kēia manawa. ʻAʻole ʻike piha ʻia nā hopena o ka patisiran i ka hāpai ʻana, no laila pono ka noʻonoʻo pono.

Nā inoa inoa Patisiran

Loaʻa ʻo Patisiran ma lalo o ka inoa inoa ʻo Onpattro. ʻO kēia wale nō ka inoa inoa i ʻae ʻia no kēia lāʻau lapaʻau ma ka hapa nui o nā ʻāina, me ʻAmelika Hui Pū ʻIa a me ʻEulopa.

Ke loaʻa iā ʻoe kāu lapaʻau, e hōʻailona ʻia ka hue lāʻau lapaʻau ʻo Onpattro, a ʻo kēia ka inoa āu e ʻike ai ma kāu moʻolelo lapaʻau a me nā palapala ʻinikua. ʻAʻohe mana generic o patisiran i loaʻa i kēia manawa.

Inā ʻoe e hele ana a i ʻole e loaʻa ana ka mālama ʻana ma nā keʻena like ʻole, e haʻi mau i ka inoa generic (patisiran) a me ka inoa inoa (Onpattro) e hōʻoia i ka kamaʻilio maopopo me kāu poʻe mālama ola.

Nā Koho ʻē aʻe o Patisiran

ʻOiai he mea maikaʻi loa ʻo patisiran no ka hATTR amyloidosis, aia nā koho lapaʻau ʻē aʻe i loaʻa. Hiki i kāu kauka ke noʻonoʻo i nā koho ʻē aʻe e pili ana i kāu mau hōʻailona kikoʻī, ka holomua o ka maʻi, a me nā kūlana pilikino.

ʻO Inotersen kekahi lāʻau lapaʻau i hoʻokumu ʻia i ka RNA i hāʻawi ʻia ma ke ʻano he injection i kēlā me kēia pule ma lalo o ka ʻili. Hana like ia me patisiran akā he ʻokoʻa ka hopena ʻaoʻao a me ka papa hana hoʻokele.

ʻO Tafamidis kahi lāʻau lapaʻau waha e kōkua ana i ka hoʻopaʻa ʻana i nā protein transthyretin, e pale ana iā lākou mai ka misfolding. He mea pono loa ia no ka poʻe i pili i ka naʻau mai hATTR.

ʻO nā lapaʻau kākoʻo ʻē aʻe paha:

• Nā lāʻau lapaʻau no ka ʻeha o ke aʻalolo a me nā hōʻailona
• Ka lāʻau lapaʻau kino a me ka lāʻau lapaʻau hana
• Kākoʻo meaʻai a me nā mea hoʻohui
• Nā lāʻau lapaʻau puʻuwai inā loaʻa ka pilina cardiac
• Hoʻololi ate i nā hihia koʻikoʻi loa

E hana pū kāu hui mālama ola me ʻoe e hoʻoholo i ke ala lapaʻau maikaʻi loa e pili ana i kāu ʻano kikoʻī o hATTR, ka paʻakikī o nā hōʻailona, ​​a me ke kūlana olakino holoʻokoʻa.

Ua ʻoi aku ka maikaʻi o Patisiran ma mua o Inotersen?

He mea maikaʻi ʻo Patisiran a me inotersen no ka mālama ʻana i ka hATTR amyloidosis, akā he mau mea maikaʻi a me nā mea e noʻonoʻo ai. ʻO ke koho "maikaʻi" e hilinaʻi ana i kou kūlana a me nā makemake.

Hāʻawi ʻia ʻo Patisiran i kēlā me kēia ʻekolu pule ma o ka IV infusion ma kahi hale mālama ola. ʻO ia hoʻi, ʻoi aku ka liʻiliʻi o nā lā lapaʻau i kēlā me kēia makahiki, akā pono ʻoe e kipa i kahi keʻena lapaʻau no kēlā me kēia mahele. Ua hōʻike ka lāʻau lapaʻau i nā hopena maikaʻi loa i nā hoʻokolohua lapaʻau me ka hōʻemi nui ʻana i ka holomua o ka maʻi.

Hāʻawi ʻia ʻo Inotersen ma ke ʻano he injection i kēlā me kēia pule ma lalo o ka ʻili hiki iā ʻoe ke aʻo e hāʻawi iā ʻoe iho ma ka home. Hāʻawi kēia i ka maʻalahi a me ka maʻalahi, akā pono ka nui o ka dosing a me ka nānā mau ʻana i ke koko ma muli o nā hopena i nā platelets a me ka hana o ka puʻupaʻa.

Ma ke ʻano o ka pono, hoʻemi nui nā lāʻau lapaʻau ʻelua i ka hana ʻana o ka protein transthyretin ʻino. Hōʻike nā haʻawina he mea maikaʻi loa lākou, ʻoiai ua kaupalena ʻia nā haʻawina hoʻohālikelike pololei.

E kōkua kāu kauka iā ʻoe e noʻonoʻo i nā mea e like me kou ʻoluʻolu me nā ʻano hana like ʻole, nā koi huakaʻi, ka papa hana, a me nā mea pilikia no nā hopena ʻaoʻao kikoʻī i ke koho ʻana ma waena o kēia mau koho.

Nā Nīnau i Nīnau Pinepine ʻia e pili ana iā Patisiran

He palekana ʻo Patisiran no ka poʻe me nā pilikia o ka naʻau?

Hiki ke hoʻohana ʻia ʻo Patisiran i ka poʻe me ka pilikia o ka naʻau mai ka hATTR amyloidosis, akā pono ia i ka nānā pono ʻana. Nui ka poʻe me ka hATTR e hoʻomohala i nā pilikia o ka naʻau ma ke ʻano o ko lākou maʻi, no laila he mea maʻamau kēia.

E hana pū kāu cardiologist a me ka loea hATTR e nānā i ka hana o kou naʻau i ka wā o ka mālama ʻana. E hoʻohana lākou i nā hoʻokolohua e like me nā echocardiograms a me nā hōʻailona koko e nānā i ka pane ʻana o kou naʻau i ka lāʻau lapaʻau.

ʻIke maoli kekahi poʻe i ka hoʻomaikaʻi ʻana i ka hana o ko lākou naʻau ma hope o ka hoʻomaka ʻana iā patisiran, no ka mea, ʻo ka hoʻemi ʻana i nā pae protein ʻino hiki ke lohi i ka pōʻino o ka naʻau. Eia naʻe, ʻaʻole hiki ke hoʻihoʻi ʻia ka pōʻino o ka naʻau.

He aha kaʻu e hana ai inā loaʻa iaʻu ka nui o Patisiran?

ʻAʻole hiki ke loaʻa ka overdose patisiran no ka mea ua hoʻomākaukau ʻia a hāʻawi ʻia ka lāʻau lapaʻau e nā poʻe loea mālama ola i aʻo ʻia i kahi hoʻonohonoho i mālama ʻia. Hoʻohālikelike ʻia ka dosage me ke akahele e pili ana i kou kaumaha kino a hāʻawi mālie ʻia ma kahi o 80 mau minuke.

Inā hopohopo ʻoe e pili ana i kāu mahele a i ʻole e ʻike i nā hōʻailona maʻamau ma hope o ka hoʻokomo ʻana, e hoʻokaʻaʻike koke i kāu hui mālama ola. Hiki iā lākou ke loiloi i kou kūlana a hāʻawi i ka nānā pono a i ʻole ka mālama ʻana inā pono.

E loaʻa i ka hale mālama ola kahi e loaʻa ai iā ʻoe ka mālama ʻana i nā protocols e mālama ai i nā hewa lāʻau lapaʻau a i ʻole nā hopena maikaʻi ʻole e hiki ke hana i ka wā o kāu hoʻokomo ʻana.

He aha kaʻu e hana ai inā hala iaʻu kahi mahele o Patisiran?

Inā hala ʻoe i kāu hoʻokomo ʻana i ka patisiran i hoʻonohonoho ʻia, e hoʻokaʻaʻike i kāu hui mālama ola i ka hiki wawe e hoʻonohonoho hou. He mea nui e mālama i nā manawa lapaʻau kūlike i ka wā hiki ke mālama i kāu mau pae protein transthyretin i kāohi ʻia.

Hiki i kāu kauka ke hoʻoponopono i kāu papa hana lapaʻau i kahi liʻiliʻi e hoʻokō i ka mahele i hala, akā e hoʻāʻo e hoʻi i ke ala me kāu mau manawa ʻekolu pule maʻamau. Mai hopohopo inā pono ʻoe e hala i kahi mahele i kekahi manawa ma muli o ka maʻi a i ʻole nā kūlana ʻē aʻe.

Inā hala pinepine ʻoe i nā mahele ma muli o nā paio hoʻonohonoho a i ʻole nā pilikia ʻē aʻe, e kūkākūkā i kēia me kāu hui mālama ola. Hiki iā lākou ke kōkua iā ʻoe e ʻimi i nā hoʻonā a i ʻole e noʻonoʻo i nā koho lapaʻau ʻē aʻe.

I ka manawa hea e hiki ai iaʻu ke hooki i ka lawe ʻana iā Patisiran?

ʻO ka hoʻoholo e hooki i ka patisiran e hana mau ʻia ma ke kūkākūkā ʻana me kāu kauka nāna i loea i ka hATTR amyloidosis. Pono ka hapa nui o nā kānaka e hoʻomau i ka mālama lōʻihi e mālama i nā pōmaikaʻi a pale i ka holomua o ka maʻi.

E loiloi mau kāu kauka i kou pane ʻana i ka lāʻau lapaʻau ma o nā hoʻokolohua hana o nā aʻalolo, nā loiloi maikaʻi o ke ola, a me ka nānā ʻana i nā pae protein transthyretin. Kōkua kēia mau loiloi i ka hoʻoholo ʻana inā he mea pono ka hoʻomau ʻana i ka lāʻau lapaʻau.

ʻO kekahi mau kumu e hiki ai iā ʻoe ke hooki i ka patisiran, ʻo ia nā hopena ʻaoʻao koʻikoʻi ʻaʻole e hoʻomaikaʻi me ka mālama ʻana, ka nele o ka pōmaikaʻi ma hope o kahi manawa hoʻokolokolo kūpono, a i ʻole inā e loli nui kou kūlana olakino holoʻokoʻa.

Inā e hooki ʻoe i ka patisiran, e hoʻi paha nā pae protein transthyretin i nā pae mua i loko o kekahi mau mahina, a e hoʻomaka hou paha kāu mau hōʻailona hATTR.

Hiki iaʻu ke hele i ka wā e lawe ana i ka Patisiran?

ʻAe, hiki iā ʻoe ke hele i ka wā e loaʻa ana ka lāʻau patisiran, akā pono ia i kekahi hoʻolālā. Ma muli o ka pono o nā infusions i kēlā me kēia ʻekolu pule, pono ʻoe e hoʻonohonoho i kāu papa huakaʻi e pili ana i kāu mau manawa lapaʻau.

Inā ʻoe e hoʻolālā ana i ka huakaʻi lōʻihi, e kūkākūkā i kēia me kāu hui mālama ola ma mua. Hiki iā lākou ke kōkua iā ʻoe e hoʻonohonoho i ka lāʻau lapaʻau manawa ma kahi kokoke i kāu huakaʻi, ʻoiai he paʻakikī kēia ma muli o ke ʻano kūikawā o ka lāʻau.

No nā huakaʻi pōkole, hiki iā ʻoe ke hoʻoponopono iki i kāu papa hana lapaʻau e hoʻokō i kāu mau hoʻolālā huakaʻi. E lawe mau i nā palapala e pili ana i kou maʻi a me ka lāʻau lapaʻau i ka wā e hele ai, ʻoiai inā e hele ana i nā palena honua.

E hoʻomanaʻo e pono ka patisiran i ka hoʻomaʻalili ʻana a me ka mālama kūikawā, no laila ʻaʻole hiki iā ʻoe ke lawe iā ʻoe iho. Pono e hana ʻia nā hoʻonohonoho a pau ma o nā keʻena mālama ola i hōʻoia ʻia.