He aha ʻo Inotersen: Nā hoʻohana, ka nui o ka lāʻau, nā hopena ʻaoʻao a me nā mea hou aku

He lāʻau lapaʻau i kauoha ʻia ʻo Inotersen i hoʻolālā ʻia e mālama i ka hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis), he maʻi genetic kakaʻikahi e pili ana i kou mau aʻalolo a me nā ʻōkana. Hana kēia lāʻau lapaʻau ma ka hoʻemi ʻana i ka hana ʻana o kahi protein pilikia i kapa ʻia ʻo transthyretin, hiki ke kūkulu i loko o kou kino a hoʻoulu i nā pilikia koʻikoʻi.

Inā ua ʻike ʻia ʻoe a i ʻole kekahi āu e mālama nei me hATTR amyloidosis, e manaʻo paha ʻoe i ka nui o ka ʻike lapaʻau. E hele kākou i nā mea āu e pono ai e ʻike e pili ana i kēia lāʻau lapaʻau ma nā huaʻōlelo maʻalahi a maopopo.

He aha ʻo Inotersen?

ʻO Inotersen kahi lāʻau lapaʻau antisense oligonucleotide āu e hoʻokomo ai ma lalo o kou ʻili i hoʻokahi manawa i ka pule. No kahi papa lāʻau lapaʻau hou e hana ana ma ka pae genetic e hōʻemi i ka holomua o ka maʻi.

Hoʻopili pololei kēia lāʻau lapaʻau i nā ʻōlelo RNA e haʻi i kou ate e hana i ka protein transthyretin. Ma ka ālai ʻana i kēia mau ʻōlelo, kōkua ʻo inotersen i ka hōʻemi ʻana i ka nui o ka protein abnormal e hana ai kou kino. E noʻonoʻo e like me ka hopu ʻana i nā memo hewa ma mua o ka hiki iā lākou ke hana i ka pōʻino hou aku.

Hāʻawi ʻia ka lāʻau lapaʻau ma ke ʻano he injection subcutaneous, ʻo ia hoʻi, komo ia i loko o ka ʻiʻo momona ma lalo pono o kou ʻili. Hiki i kēia ke komo mālie ka lāʻau lapaʻau i loko o kou kahe koko a hana i loko o kou kino.

No ke aha e hoʻohana ʻia ai ʻo Inotersen?

Hoʻomaʻamaʻa ʻo Inotersen i ka hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) i nā pākeke. Hana ʻia kēia maʻi genetic kakaʻikahi ke hana kou kino i nā protein transthyretin abnormal e hui pū a hōʻino i kou mau aʻalolo a me nā ʻōkana.

He maʻi e pili nui ana i nā aʻalolo o kou kino, e hana ana i nā hōʻailona e like me ka ʻūlū, ka ʻūlū, ka ʻeha, a me ka nāwaliwali o kou mau lima a me nā wāwae. Ma hope o ka manawa, hiki nō ke pili i kou puʻuwai, nā puʻupaʻa, a me ka ʻōnaehana digestive. Inā ʻaʻole mālama ʻia, e ʻoi aku ka maʻi hATTR amyloidosis a hiki ke lilo i mea weliweli i ke ola.

E kauoha wale kāu kauka i ka inotersen inā he maʻi genetic hōʻoia ʻoe no ka hATTR amyloidosis. Ua hoʻolālā kūikawā ʻia ka lāʻau lapaʻau no kēia maʻi a ʻaʻole ia e kōkua i nā ʻano pilikia aʻalolo ʻē aʻe a i ʻole amyloidosis.

Pehea e Hana ai ʻo Inotersen?

Hana ʻo Inotersen ma ka hoʻohana ʻana i nā mīkini cellular kūlohelohe o kou kino e hōʻemi i ka hana ʻana o ka protein transthyretin. Ua manaʻo ʻia he lāʻau lapaʻau ikaika e hiki ke hoʻolohi nui i ka holomua o ka maʻi ke hoʻohana pono ʻia.

Hoʻopaʻa ka lāʻau lapaʻau i ka messenger RNA (mRNA) e lawe ana i nā ʻōlelo aʻo mai kou mau genes i kāu mīkini hana protein. Ma ka hoʻopaʻa ʻana i kēia mRNA, pale ʻo inotersen i kou ate mai ka hana ʻana i ka nui o ka protein transthyretin. Hoʻemi kēia i ka nui o ka protein abnormal i loaʻa e hana i nā waihona pōʻino i kou kino.

Hōʻike nā haʻawina e hiki i ka inotersen ke hōʻemi i ka hana transthyretin ma kahi o 75-80% i ka hapa nui o nā maʻi. Kōkua kēia hōʻemi nui i ka hoʻolohi ʻana i ka pōʻino aʻalolo a hiki ke hoʻopaʻa a i ʻole e hoʻomaikaʻi i kekahi mau hōʻailona. Eia naʻe, pono nā mahina he nui o ka mālama ʻana e ʻike i nā pōmaikaʻi.

Pehea e Lawe ai au i ka Inotersen?

E hoʻokomo ʻoe i ka inotersen i hoʻokahi manawa i ka pule i ka lā like i kēlā me kēia pule, e hoʻololi ana ma waena o nā wahi injection like ʻole. Hele mai ka lāʻau lapaʻau i loko o nā syringes i hoʻopiha mua ʻia āu e mālama ai i loko o kāu pahu hau a hiki i ka hoʻohana ʻana.

Ma mua o kāu injection, e ʻae i ka lāʻau lapaʻau e mahana i ka lumi wela no kahi o 30 mau minuke. E hoʻomaʻemaʻe i kāu wahi injection me kahi swab waiʻona a hoʻokomo i ka lāʻau lapaʻau i loko o ka ʻiʻo momona o kou ʻūhā, lima luna, a i ʻole ka ʻōpū. E aʻo kāu hui mālama ola iā ʻoe i ke ʻano injection kūpono a me nā wahi e hoʻololi ai.

Hiki iā ʻoe ke lawe i ka inotersen me ka ʻole o ka meaʻai no ka mea ua hoʻokomo ʻia ma mua o ka lawe ʻia ʻana e ka waha. Eia naʻe, ʻo ka noho ʻana i ka wai a me ka mālama ʻana i ka meaʻai maikaʻi hiki ke kōkua i kou kino e mālama maikaʻi i ka lāʻau lapaʻau. Manaʻo kekahi poʻe he mea kōkua ke hoʻokomo i ka manawa like i kēlā me kēia pule e hoʻokumu i kahi hana maʻamau.

Pehea ka lōʻihi e lawe ai au i ka Inotersen?

ʻO ka inotersen ka mea maʻamau he lapaʻau lōʻihi e hoʻomau ʻoe i ka wā pau ʻole inā e kōkua ana a ke ʻae maikaʻi nei ʻoe. No ka mea he maʻi genetic holomua ka hATTR amyloidosis, ʻo ka hoʻōki ʻana i ka lāʻau lapaʻau e ʻae maʻamau i ka maʻi e holomua hou.

E nānā kāu kauka i kāu pane ʻana i ka lāʻau lapaʻau ma o nā hoʻokolohua koko maʻamau a me nā hoʻokolohua neurological. Hoʻomaka ka hapa nui o nā kānaka e ʻike i ka paʻa ʻana o kā lākou mau hōʻailona i loko o 6-12 mau mahina, ʻoiai e lōʻihi paha ka manawa e ʻike ʻia ai kekahi mau hoʻomaikaʻi.

ʻO ka hoʻoholo e hoʻomau a hoʻōki paha i ka inotersen e hilinaʻi ana i nā mea he nui me ka maikaʻi o kāna hana ʻana, nā hopena ʻaoʻao āu e ʻike nei, a me kou kūlana olakino holoʻokoʻa. E hana pū kāu hui lapaʻau me ʻoe e hoʻoholo i ka hoʻolālā lōʻihi maikaʻi loa no kāu kūlana kikoʻī.

He aha nā hopena ʻaoʻao o Inotersen?

E like me nā lāʻau lapaʻau a pau, hiki i ka inotersen ke hana i nā hopena ʻaoʻao, ʻoiai ʻaʻole ʻike nā kānaka a pau iā lākou. ʻO nā hopena ʻaoʻao maʻamau ka mea maʻamau e mālama ʻia me ka nānā pono a me ka mālama.

Eia nā hopena ʻaoʻao āu e ʻike ai, e hoʻomaka ana me nā mea maʻamau:

• Nā hopena o ka pūnaewele injection e like me ka ʻulaʻula, ka pehu, a i ʻole ka ʻeha
• Nausea a me ka luaʻi
• Poʻo
• Luhi
• Fiva
• ʻEha ʻiʻo
• Haʻalulu

Hoʻomaikaʻi pinepine kēia mau hopena ʻaoʻao maʻamau i ka wā e hoʻoponopono ai kou kino i ka lāʻau lapaʻau i nā pule mua o ka mālama ʻana.

Pono nā hopena ʻaoʻao koʻikoʻi i ka lāʻau lapaʻau koke a me:

• He ʻuʻuku loa o nā platelets (thrombocytopenia) e hoʻoulu ana i ka ʻeha a i ʻole ke kahe koko
• Nā pilikia o ka puʻupaʻa me ka glomerulonephritis
• Nā hopena koʻikoʻi ma ke kahua o ka pīkī ʻana me ka pehu nui a i ʻole ka maʻi
• Nā hōʻailona o nā maʻi mumū

E nānā pono kāu kauka iā ʻoe me nā hoʻokolohua koko maʻamau e ʻike koke i nā hopena ʻino. Hiki i ka hapa nui o nā kānaka ke hoʻomau i ka lāʻau lapaʻau me ka palekana me ka nānā pono a me nā hoʻoponopono ʻana i ka lāʻau ke pono.

ʻO wai ka mea pono ʻole e lawe iā Inotersen?

ʻAʻole pono kekahi poʻe e lawe iā inotersen ma muli o ka piʻi ʻana o ka pilikia o nā pilikia koʻikoʻi. E loiloi pono kāu kauka inā palekana kēia lāʻau lapaʻau iā ʻoe ma mua o ke kau ʻana.

ʻAʻole pono ʻoe e lawe iā inotersen inā loaʻa iā ʻoe:

• Maʻi puʻupaʻa koʻikoʻi a i ʻole e hana ana i ka dialysis
• Nā maʻi kahe koko ikaika, ʻaʻole i mālama ʻia
• He haʻahaʻa loa o nā helu platelet
• Nā maʻi ikaika ʻaʻole hiki i kāu ʻōnaehana pale ke mālama
• Nā maʻi maʻi i ʻike ʻia iā inotersen a i ʻole kāna mau mea

Pono ka mālama kūikawā inā loaʻa iā ʻoe nā pilikia puʻupaʻa māmā a i ʻole kaulike, ka mōʻaukala o nā maʻi kahe koko, a i ʻole e lawe ana i nā lāʻau hoʻoheheʻe koko. Hiki nō i kāu kauka ke kau iā inotersen akā e nānā pono iā ʻoe a e hoʻoponopono paha i kāu hoʻolālā lapaʻau.

Pono ka noʻonoʻo kūikawā i ka wā e hāpai ana a me ka hānai ʻana i ka waiū, no ka mea, ʻaʻole i ʻike piha ʻia nā hopena o inotersen i nā pēpē e ulu ana. E kūkākūkā kāu kauka i nā pilikia a me nā pōmaikaʻi inā ʻoe e hoʻolālā ana e hāpai a i ʻole e hāpai ana.

Nā inoa inoa inoa inoa o Inotersen

Kūʻai ʻia ʻo Inotersen ma lalo o ka inoa inoa Tegsedi ma ka hapa nui o nā ʻāina i loaʻa. ʻO kēia wale nō ka inoa inoa i ʻae ʻia no kēia lāʻau.

Hana ʻia ʻo Tegsedi e Akcea Therapeutics a loaʻa ma kekahi mau ʻāina me ʻAmelika Hui Pū ʻIa, Kanada, a me nā lāhui ʻEulopa he nui. Hele mai ka lāʻau lapaʻau i loko o nā syringes i hoʻopiha mua ʻia i loaʻa iā 284 mg o inotersen sodium.

Mai ka mea he lāʻau lapaʻau kūikawā kēia no kahi maʻi laha ʻole, loaʻa wale nō ia ma o nā hale kūʻai lāʻau lapaʻau kūikawā a pono e ʻae mua ʻia mai kāu ʻoihana ʻinikua. E kōkua kāu hui mālama ola e hoʻonohonoho i ke komo ʻana i ka lāʻau lapaʻau ma o nā ala kūpono.

Nā Koho ʻē aʻe no Inotersen

Nui nā lāʻau lapaʻau ʻē aʻe e hiki ke mālama i ka hATTR amyloidosis, akā hana kēlā me kēia mea i nā mea like ʻole a ʻoi aku paha ka maikaʻi no nā maʻi like ʻole. E kōkua kāu kauka e hoʻoholo i ke koho maikaʻi loa no kāu kūlana kikoʻī.

ʻO nā koho nui e komo pū ana:

• Patisiran (Onpattro) - kekahi lāʻau lapaʻau e kuhikuhi ana i ka RNA i hāʻawi ʻia e ka IV infusion
• Tafamidis (Vyndaqel) - he lāʻau lapaʻau e hoʻopaʻa i ka protein transthyretin
• Diflunisal - he lāʻau lapaʻau kahiko e hiki ke hoʻopaʻa i ka protein

Loaʻa i kēlā me kēia koho ʻē aʻe nā pōmaikaʻi like ʻole a me nā hopena ʻaoʻao. Hana ʻo Patisiran e like me inotersen akā pono e hoʻokomo ʻia ka IV i kēlā me kēia mahina ma kahi o nā injections i kēlā me kēia pule. Hana ʻo Tafamidis ma ka pale ʻana i ka protein transthyretin mai ka wehe ʻana a me ka hana ʻana i nā waihona pōʻino.

ʻO ke koho ʻana ma waena o kēia mau lāʻau lapaʻau e hilinaʻi ana i nā mea e like me ke kahua o kou maʻi, nā kūlana olakino ʻē aʻe, ka hiki ke ʻae i nā injections a i ʻole nā infusions, a me kāu mau makemake pilikino. Pono paha kekahi poʻe e hoʻāʻo i nā koho like ʻole e ʻike i ka mea e hana maikaʻi ai no lākou.

ʻOi aku ka maikaʻi o Inotersen ma mua o Patisiran?

He mau lāʻau lapaʻau maikaʻi ʻo Inotersen a me patisiran no ka hATTR amyloidosis, akā he mau mea maikaʻi a me nā mea maikaʻi ʻole. ʻAʻole maikaʻi kekahi ma mua o kekahi - ʻo ke koho maikaʻi loa e hilinaʻi ana i kāu mau kūlana pilikino.

Hāʻawi ʻo Inotersen i ka maʻalahi o nā injections ponoʻī i kēlā me kēia pule ma ka home, kahi e makemake ai ka poʻe he nui ma mua o nā huakaʻi i kēlā me kēia mahina i kahi kikowaena infusion no patisiran. Eia naʻe, pono ʻo inotersen i ka nānā pinepine ʻana no nā hopena ʻaoʻao, ʻo ia hoʻi nā pilikia o ka puʻupaʻa a me ka platelet.

He ʻoi aku ka maikaʻi o ka hopena ʻaoʻao o Patisiran i kekahi mau haʻawina, akā pono ia i nā infusions IV i kēlā me kēia mahina e lawe ana i kekahi mau hola. He mea paʻakikī kēia no ka poʻe e noho mamao ana mai nā kikowaena infusion a i ʻole he pilikia me ke komo ʻana o IV.

Hoʻemi maikaʻi nā lāʻau ʻelua i ka hana transthyretin ma nā ʻano like a hiki ke hoʻolohi i ka holomua o ka maʻi. E noʻonoʻo kāu kauka i nā mea e like me kou ola, nā kūlana olakino ʻē aʻe, a me nā makemake pilikino i ke kōkua ʻana iā ʻoe e koho ma waena o kēia mau koho.

Nā Nīnau i Nīnau Pinepine ʻia e pili ana iā Inotersen

Q1. He palekana anei ʻo Inotersen no ka poʻe me nā pilikia o ka naʻau?

Hiki ke hoʻohana ʻia ʻo Inotersen i ka poʻe me ka pilikia o ka naʻau mai ka hATTR amyloidosis, akā pono ka nānā pono ʻana. ʻAʻole pili pololei ka lāʻau i ka hana o ka naʻau, akā pili pinepine ke kūlana ma lalo i ka naʻau.

E hana pū kāu cardiologist a me ka neurologist e nānā i kou olakino naʻau i ka wā o ka mālama ʻana. Pono paha kekahi poʻe me ka maʻi puʻuwai koʻikoʻi i nā hana mālama hou aʻe a i ʻole ka nānā ʻana. ʻO nā pōmaikaʻi hiki ke hoʻolohi i ka holomua o ka maʻi pinepine i nā pilikia no ka poʻe me ka pilikia o ka naʻau.

Q2. He aha kaʻu e hana ai inā ua hoʻohana hewa au i ka nui o Inotersen?

Inā ʻoe e hoʻokomo hewa i ka nui o ka inotersen, e hoʻokaʻaʻike koke i kāu kauka a i ʻole nā ​​lawelawe pilikia. Mai kali e ʻike inā e hoʻomohala ʻoe i nā hōʻailona - hiki i ka overdose ke kumu i nā hopena ʻaoʻao koʻikoʻi e pono ai ka lāʻau lapaʻau wikiwiki.

E lawe mai i ka pūʻolo lāʻau me ʻoe i ka haukapila i ʻike pono nā mea mālama ola i kāu mea i lawe ai. Pono paha lākou e nānā i kāu helu koko a me ka hana o ka puʻupaʻa a hāʻawi i ke kākoʻo e like me ka mea e pono ai.

Q3. He aha kaʻu e hana ai inā ua hala au i kahi mahele o Inotersen?

Inā hala ʻoe i kāu mahele i kēlā me kēia pule, e lawe iā ia i ka wā e hoʻomanaʻo ai ʻoe, a laila e hoʻi i kāu papa maʻamau. Mai lawe i ʻelua mau mahele kokoke i ka hui e hana i kahi mahele i hala.

Inā ua hala nā lā ma mua o 2 mai kāu lā i hoʻonohonoho ʻia, e kāhea i kāu kauka no ke alakaʻi. Hiki iā lākou ke ʻōlelo e lawe i ka lā i hala a i ʻole e kali a hiki i kāu injection i hoʻonohonoho ʻia, ma muli o kou kūlana kikoʻī.

Q4. I ka manawa hea e hiki ai iaʻu ke hooki i ka lawe ʻana iā Inotersen?

Pono ʻoe e hooki i ka lawe ʻana iā inotersen ma hope o ke kūkākūkā ʻana me kāu kauka. No ka mea he holomua ka hATTR amyloidosis, ʻo ka hooki ʻana i ka lāʻau lapaʻau e ʻae pinepine i ka maʻi e holomua hou.

Hiki i kāu kauka ke ʻōlelo e hooki inā loaʻa iā ʻoe nā hopena ʻaoʻao koʻikoʻi ʻaʻole hiki ke mālama ʻia, inā ʻaʻole kōkua ka lāʻau lapaʻau ma hope o kahi manawa hoʻokolokolo kūpono, a i ʻole inā loli nui kou kūlana olakino holoʻokoʻa. Mai hooki koke i ka lawe ʻana iā inotersen me ka nānā ʻole ʻana i ka lāʻau lapaʻau.

Q5. Hiki iaʻu ke hele i ka wā e lawe ana iā Inotersen?

ʻAe, hiki iā ʻoe ke hele i ka wā e lawe ana iā inotersen, akā pono ia i kekahi hoʻolālā. Pono e hoʻomaʻalili ʻia ka lāʻau lapaʻau a pono ʻoe e mālama i kāu papa hana injection i kēlā me kēia pule me ka nānā ʻole i nā loli o ka manawa.

E kiʻi i kahi palapala huakaʻi mai kāu kauka e wehewehe ana i kou maʻi a me nā pono lāʻau lapaʻau. E hoʻopili i nā lako hou i ka hihia o nā lohi, a e noiʻi i nā keʻena lapaʻau ma kāu huakaʻi inā pono ʻoe i ke kōkua. Nui ka poʻe e hele maikaʻi ana i ka wā e inotersen me ka hoʻomākaukau kūpono.