Inā loaʻa iā ʻoe a i ʻole kekahi o kou ʻohana kahi maʻi i kapa ʻia ʻo Congenital Adrenal Hyperplasia (CAH), he nūhou koʻikoʻi loa kēia nou. Ma hope o 70 mau makahiki, ua ʻae ʻia kahi lāʻau lapaʻau hou no kēia maʻi. He manaʻolana nui kēia no ka poʻe e noho nei me CAH. No laila, e nānā kākou i ke ʻano o kēia lāʻau hou, pehea e hana ai, a me nā pono.
ʻO ka mea mua, e hoʻomaopopo kākou, he aha ka CAH?
I ka ʻōlelo maʻalahi, he maʻi hoʻoilina ʻo CAH e hoʻopilikia ana i ko kākou mau kelepa adrenal. Aia kēia mau kelepa ma luna o ko kākou mau puʻupaʻa. Hoʻopuka lākou i kekahi mau hormones e pono ai no ko kākou kino.
E noʻonoʻo i ke kelepa adrenal e like me kahi hale hana. ʻO ka huahana nui o kēia hale hana he hormone i kapa ʻia ʻo cortisol. He hormone koʻikoʻi loa kēia e kōkua i ko mākou kino e hoʻomanawanui i ke kaumaha, ka maʻi, a me ka pōʻino.
Ke hāʻule nā pae cortisol i loko o ke kanaka olakino, hoʻouna ka lolo (e like me ka luna o ka hale hana) i kahi hōʻailona (kauoha) i kapa ʻia ʻo ACTH i ka adrenal gland e "hana cortisol." Akā i loko o kekahi me CAH, nele ka adrenal gland i kahi enzyme koʻikoʻi, a i ʻole 'mea hana', e pono ai e hana i ka cortisol. No laila, ʻaʻohe mea nui ka nui o ka ACTH a ka lolo e hoʻouna ai, ʻaʻole hiki i ka hale hana ke hana i ka cortisol.
Ke hāpai nei kēia i ʻelua mau nīnau:
1. ʻAʻole hana ke kino i ka cortisol e pono ai .
2. Ke hoʻomau nei nā hōʻailona ACTH mai ka lolo e kahe, ua hana ʻia kekahi ʻano hormone ʻē aʻe i hana ʻia e ka adrenal gland , i kapa ʻia ʻo androgens, me ka nui loa.
He mau hormones kāne kēia mau androgens (e.g. testosterone). Pono nā kāne a me nā wahine i kekahi o lākou, akā i ka CAH, lilo lākou i kiʻekiʻe loa. Hiki i kēia ke hana i ka wā ʻōpiopio mua, ka hānau ʻole ʻana, a me nā pilikia ʻē aʻe me ka ulu ʻana o ke keiki .
No nā makahiki he ʻumi, ʻo ke ʻano nui o ka lāʻau lapaʻau no kēia, ʻo ia ka hāʻawi ʻana i nā steroids (glucocorticoids) e hana like me ka cortisol ma waho. ʻAʻole wale kēia e pani i ka cortisol i nele i ke kino, akā e kāohi pū i ka hōʻailona ACTH mai ka lolo, e hōʻemi ana i ka hana ʻana o nā androgens. Eia naʻe, ʻo ka pilikia, pono kēia e lawe i nā dosis steroids kiʻekiʻe ma mua o ka maʻamau no ka manawa lōʻihi. Hiki i kēia ke hana i nā hopena ʻaoʻao he nui, e like me ka piʻi ʻana o ke kaumaha, ka lahilahi o nā iwi, a me nā pilikia hiamoe .
Pehea ʻoe i loaʻa ai kēia lāʻau lapaʻau hou?
Ua noʻonoʻo nā kānaka ʻepekema inā he hopena ʻē aʻe i kēia pilikia steroid. Ma kahi o ka hāʻawi ʻana i ka nui o nā steroids ma waho, pehea inā loaʻa iā mākou kahi ala e hōʻemi pololei ai i ka hōʻailona ACTH e hele mai ana mai ka lolo?
ʻO ia kahi i loaʻa ai iā lākou kahi pahuhopu hou. He mea loaʻa ia i loko o ka lolo i kapa ʻia ʻo CRF1. Ke hāʻule nā pae cortisol, hoʻāla ʻia ka hōʻailona ACTH e CRF1. I CAH, ua hoʻopilikia ʻia kēia kaʻina hana.
No laila, ua manaʻo nā kānaka ʻepekema, inā mākou e ālai i kēia CRF1, hiki iā mākou ke hōʻemi i ka hana ʻana o ACTH, ʻeā? Inā hana ʻia kēlā, e kāohi ʻia hoʻi ka hana ʻana o nā androgens, a e hoʻemi ʻia hoʻi ka nui o nā steroids e pono ai ka mea maʻi e lawe.
ʻO kēia manaʻo hou ke kumu i alakaʻi ai i ka hoʻomohala ʻana o kahi lāʻau lapaʻau hou i kapa ʻia ʻo Crinecerfont. He lāʻau lapaʻau kēia i hoʻolālā ʻia e hoʻoponopono i kahi pilikia nui o CAH ma ke kāohi ʻana iā CRF1.
Nā hoʻokolohua i hana ʻia ma mua o ka hāʻawi ʻana i ka lāʻau lapaʻau i nā kānaka
ʻAʻole hiki ke hāʻawi koke ʻia kahi lāʻau lapaʻau hou i nā kānaka ke ʻike ʻia. Pono ia e hana i kekahi mau hoʻāʻo e hōʻoia i ka palekana a me ka pono. Ua hala ka lāʻau Crinecerfont i kēia mau ʻanuʻu a pau.
1. Hoʻāʻo ʻana ma ke keʻena hoʻokolohua a me nā holoholona: ʻO ka hana mua, ʻo ia ke hoʻāʻo ʻana i ka lāʻau lapaʻau ma nā pūnaewele a me nā holoholona i loko o ka hale hoʻokolohua. ʻO kēia ke hoʻoholo inā he mea ʻawahia ka lāʻau lapaʻau a inā paha e hōʻino ia i nā ʻōpū. E noʻonoʻo wale, hoʻokahi wale nō i loko o 10,000 mau lāʻau lapaʻau hou e hala i kēia mau pilikia a pau a ʻae ʻia.
2. Nā Hoʻokolohua Lapaʻau: Ma hope o ka holomua o ka hoʻāʻo ʻana i nā holoholona, hana ʻia nā hoʻokolohua kanaka ma kekahi mau pae.
Pehea nā hopena hoʻāʻo no Crinecerfont?
Ua loaʻa nā hopena maikaʻi loa i nā hoʻokolohua i mālama ʻia ma kēia lāʻau. ʻOi loa aku ka nui o nā hoʻokolohua Phase II a me Phase III.
Māhele II - Nā hopena o nā keiki ʻōpio
Maanei, ua hāʻawi ʻia kahi hui liʻiliʻi o nā keiki ʻōpio (14-17 mau makahiki) me CAH i ka lāʻau no 14 mau lā a ua ʻike ʻia nā loli i ko lākou pae hormone. He mea kupanaha nā hopena.
- Ua emi ka ACTH (hōʻailona lolo) ma 57% .
- Ua emi ka 17-OHP (he hōʻailona o ka hana ʻana o ka androgen) ma 69% .
- Ua emi iho ʻo Androstenedione (kahi ʻano androgen) ma 58% .
Māhele III - Nā hopena o nā hui nui
Ua hoʻoikaika ʻia e kēia mau hopena, ua hana nā kānaka ʻepekema i ʻelua mau hoʻokolohua nui e hoʻohana ana i nā pākeke a me nā keiki, kahi i hāʻawi ʻia ai kekahi i ka lāʻau Crinecerfont a ua hāʻawi ʻia kekahi i kahi placebo i like me ka lāʻau akā ʻaʻohe hopena.
| Hui | Ka hopena nui | Wehewehena |
|---|---|---|
| Nā mākua (e lawe ana iā Crinecerfont) | Hoʻemi i ka nui o ka steroid | Ma ka kaohi ʻana i nā pae hormone, ua hiki ke hōʻemi i ka nui o nā steroid i kēlā me kēia lā ma 27% . |
| Nā mākua (i lawe i ka placebo) | Hoʻemi i ka nui o ka steroid | Ua hoʻemi wale ʻia ka nui o ka steroid ma ka 10%. |
| Nā keiki (e lawe ana iā Crinecerfont) | Nā pae Androgen a me ka nui o ka steroid | Ma hope o 4 mau pule, ua emi nui nā pae androgen. I ka hopena o ka hoʻāʻo ʻana, ua hoʻemi ʻia ka nui o ka steroid ma 18% . |
| Nā keiki (ua lawe i ka placebo) | Nā pae Androgen a me ka nui o ka steroid | Ua piʻi aʻe nā pae Androgen. Pono e hoʻonui ʻia ka nui o ka steroid ma kahi o 6%. |
Hōʻike maopopo kēia mau hopena e kōkua ana ʻo Crinecerfont i ka kaohi ʻana i nā pae androgen, ʻoiai e hōʻemi ana i ka nui o nā steroids i hiki ke hoʻopōʻino i nā maʻi i ka wā lōʻihi.
Pehea ka palekana o kēia lāʻau lapaʻau?
Ua hōʻike ʻia nā noiʻi ʻaʻohe hopena koʻikoʻi o kēia lāʻau. ʻO ka hapa nui o nā hopena ʻaoʻao i hōʻike ʻia he liʻiliʻi a no ka manawa pōkole.
- Luhi
- ʻEha poʻo
- Ihu anu a i ʻole ka ihu paʻa
- ʻEha ʻiʻo a ʻeha paha o ka ʻōpū
- ʻEha hui
ʻO ka ʻōlelo aʻo nui wale nō e hele mai me kēia lāʻau lapaʻau, inā ʻaʻole ʻoe e lawe i ka nui pololei o nā steroids i kuhikuhi ʻia e kāu kauka , hiki i kāu mau pae cortisol ke hāʻule haʻahaʻa loa, e alakaʻi ana i kahi maʻi hoʻoweliweli ola i kapa ʻia ʻo ka adrenal insufficiency. Eia nō naʻe, ʻaʻohe mea nāna i lawe pono i ka lāʻau lapaʻau i loko o nā haʻawina i ʻike i kēia ʻano . No laila, he mea nui loa ia e hahai i nā ʻōlelo a kāu kauka.
Manaʻolana hou no ka mālama ʻana iā CAH
Ma muli o kēia mau hopena holomua, ua hāʻawi ka FDA (ke keʻena nui e ʻāpono ana i nā lāʻau lapaʻau) ma ʻAmelika Hui Pū ʻIa i ka ʻae kūikawā no kēia lāʻau lapaʻau. ʻAʻole ʻo Crinecerfont (inoa inoa ʻo Crenessity) he lāʻau lapaʻau no CAH. Eia nō naʻe, he ʻanuʻu nui ia i mua i ka mālama ʻana iā CAH.
Hiki i nā kauka ke kuhikuhi i kēia lāʻau lapaʻau ma waho aʻe o kāu lāʻau lapaʻau steroid o kēia manawa (hoʻohui lāʻau lapaʻau) . Hiki i kēia ke kōkua i ka hōʻemi ʻana i kāu mahele steroid. ʻO ke ʻano kēia e ʻike paha ʻoe i nā hopena ʻaoʻao liʻiliʻi mai nā steroids, kaohi maikaʻi ʻana i kāu mau pae hormone, a loaʻa iā ʻoe kahi ola maikaʻi aʻe.
Leka Lawe-Home
- ʻO CAH (Congenital Adrenal Hyperplasia) kahi maʻi genetic e hoʻopilikia i nā kelepa adrenal a hoʻoulu i ke kaulike ʻole o ka hormonal.
- He lāʻau lapaʻau hou i ʻāpono ʻia no CAH ʻo Crinecerfont (Crenessity), i hāʻawi ʻia ma waho aʻe o ka lāʻau steroid.
- Hoʻemi kēia i nā kiʻekiʻe o ka hormone androgen kiʻekiʻe i loko o ke kino a hiki ke kōkua i ka hoʻemi ʻana i ka nui o nā steroid i kēlā me kēia lā.
- Ma ka hoʻemi ʻana i ka nui o nā steroids, aia kahi manawa kūpono e hōʻemi ai i nā hopena ʻaoʻao e pili ana i ka hoʻohana ʻana i ka steroid i ka wā lōʻihi.
- ʻOiai ʻaʻole kēia he lāʻau lapaʻau no ka maʻi, he ʻanuʻu nui ia i mua i ka hoʻomaikaʻi ʻana i ke ʻano o ke ola no nā poʻe maʻi CAH.
- Inā loaʻa iā ʻoe a i ʻole kāu keiki ʻo CAH, he mea nui e kamaʻilio me kāu endocrinologist a kauka ʻohana paha e ʻike inā kūpono kēia lāʻau hou iā ʻoe .
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