Ngati inu kapena wina m'banja mwanu ali ndi vuto lotchedwa Congenital Adrenal Hyperplasia (CAH), izi zidzakhala nkhani yofunika kwambiri kwa inu. Pambuyo pa zaka 70, chithandizo chatsopano cha matendawa chavomerezedwa. Ichi ndi chiyembekezo chachikulu kwa anthu omwe ali ndi CAH. Tiyeni tiwone chomwe mankhwala atsopanowa ndi, momwe amagwirira ntchito, komanso ubwino wake.
Choyamba, tiyeni timvetse, kodi CAH ndi chiyani?
Mwachidule, CAH ndi matenda a majini omwe amakhudza ma adrenal glands athu. Ma adrenal glands amenewa ali pamwamba pa impso zathu. Amapanga mahomoni angapo ofunikira m'thupi lathu.
Ganizirani za adrenal gland ngati fakitale. Chinthu chachikulu chomwe chimapezeka mu fakitale iyi ndi hormone yotchedwa cortisol. Iyi ndi hormone yofunika kwambiri yomwe imathandiza thupi lathu kuthana ndi nkhawa, matenda, komanso zoopsa.
Pamene kuchuluka kwa cortisol mwa munthu wathanzi kutsika, ubongo (monga manejala wa fakitale) umatumiza chizindikiro (order) chotchedwa ACTH ku adrenal gland kuti "chipange cortisol." Koma mwa munthu amene ali ndi CAH, adrenal gland ilibe enzyme yofunika, kapena 'wogwira ntchito', yofunikira kuti ipange cortisol. Chifukwa chake ngakhale ubongo utumize ACTH yochuluka bwanji, fakitaleyo singathe kupanga cortisol.
Izi zikubweretsa mafunso awiri:
1. Thupi silipanga cortisol yofunikira .
2. Pamene zizindikiro za ACTH kuchokera muubongo zikupitirira kuyenda, mtundu wina wa mahomoni opangidwa ndi adrenal gland , wotchedwa androgens, umapangidwa mopitirira muyeso .
Ma androgens awa (monga testosterone) ndi mahomoni achimuna. Amuna ndi akazi onse amafunikira ena mwa iwo, koma mu CAH, amakwera kwambiri. Izi zingayambitse kutha msinkhu msanga, kusabereka, ndi mavuto ena pakukula kwa mwana .
Kwa zaka zambiri, chithandizo chachikulu cha izi chakhala kupereka ma steroids (glucocorticoids) omwe amagwira ntchito ngati cortisol kunja. Izi sizimangolowa m'malo mwa cortisol yomwe thupi silinapeze, komanso zimawongolera chizindikiro cha ACTH kuchokera ku ubongo, kuchepetsa kupanga kwa androgens. Komabe, vuto ndilakuti izi zimafuna kumwa ma steroids ambiri kuposa masiku onse kwa nthawi yayitali. Izi zingayambitse mavuto ambiri, monga kunenepa, mafupa ochepa, komanso mavuto ogona .
Kodi mwapeza bwanji mankhwala atsopanowa?
Asayansi anadzifunsa ngati pali njira ina yothetsera vutoli. M'malo mopereka ma steroid ambiri kunja, bwanji ngati titapeza njira yochepetsera mwachindunji chizindikiro cha ACTH chochokera muubongo?
Apa ndi pomwe adapeza cholinga chatsopano. Chinali cholandirira muubongo chotchedwa CRF1. Pamene kuchuluka kwa cortisol kumatsika, chizindikiro cha ACTH chimayambitsidwa ndi CRF1. Mu CAH, njirayi imasokonekera.
Choncho asayansi anaganiza kuti, ngati tiletsa CRF1 iyi, tingachepetse kupanga kwa ACTH, sichoncho? Ngati zimenezo zitachitika, kupanga kwa androgens kudzawongoleredwanso, ndipo mlingo wa ma steroids omwe wodwalayo ayenera kumwa nawonso udzachepetsedwa.
Lingaliro latsopanoli ndi lomwe linapangitsa kuti pakhale mankhwala atsopano otchedwa Crinecerfont. Uwu ndi mankhwala omwe adapangidwa kuti athetse vuto lalikulu la CAH poletsa CRF1.
Mayeso ochitidwa musanapereke mankhwala kwa anthu
Mankhwala atsopano sangapatsidwe anthu akangopezeka. Ayenera kuyesedwa kangapo kuti atsimikizire ngati ndi otetezeka komanso ogwira ntchito. Mankhwala a Crinecerfont adadutsa njira zonsezi.
1. Kuyesa kwa Laboratory ndi nyama: Gawo loyamba ndikuyesa mankhwalawo pa maselo ndi nyama zomwe zili mu laboratory. Izi ndi kudziwa ngati mankhwalawa ndi oopsa kapena ngati angavulaze ziwalo. Tangoganizirani, pafupifupi mankhwala amodzi mwa 10,000 atsopano ndi omwe amapambana zovuta zonsezi ndipo amavomerezedwa.
2. Mayeso a Zachipatala: Pambuyo poti mayeso a nyama apambana, mayesero a anthu amachitidwa m'magawo angapo.
Kodi zotsatira za mayeso a Crinecerfont zili bwanji?
Mayeso omwe anachitika pa mankhwalawa apereka zotsatira zabwino kwambiri. Makamaka mayeso a Gawo Lachiwiri ndi Lachitatu ndi ofunikira kwambiri.
Gawo Lachiwiri - Zotsatira za ana aang'ono
Apa, gulu laling'ono la ana aang'ono (azaka 14-17) omwe ali ndi CAH adapatsidwa mankhwalawa kwa masiku 14 ndipo kusintha kwa mahomoni awo kunawonedwa. Zotsatira zake zinali zodabwitsa.
- ACTH (chizindikiro cha ubongo) chatsika ndi 57% .
- 17-OHP (chizindikiro cha kupanga kwa androgen) chatsika ndi 69% .
- Androstenedione (mtundu wa androgen) yatsika ndi 58% .
Gawo Lachitatu - Zotsatira za magulu akuluakulu
Atalimbikitsidwa ndi zotsatirazi, asayansi adachita mayeso awiri akuluakulu pogwiritsa ntchito akuluakulu ndi ana, pomwe ena adapatsidwa mankhwala a Crinecerfont ndipo ena adapatsidwa placebo omwe amawoneka ngati mankhwala koma osagwira ntchito.
| Gulu | Zotsatira zazikulu | Kufotokozera |
|---|---|---|
| Akuluakulu (akutenga Crinecerfont) | Kuchepetsa mlingo wa steroid | Mwa kuwongolera kuchuluka kwa mahomoni, zinali zotheka kuchepetsa mlingo wa steroid tsiku lililonse ndi 27% . |
| Akuluakulu (adatenga placebo) | Kuchepetsa mlingo wa steroid | Mlingo wa steroid unachepetsedwa ndi 10%. |
| Ana (akutenga Crinecerfont) | Kuchuluka kwa Androgen ndi Mlingo wa Steroid | Pambuyo pa masabata anayi, kuchuluka kwa androgen kunachepa kwambiri. Pamapeto pa mayeso, mlingo wa steroid unachepetsedwa ndi 18% . |
| Ana (anatenga placebo) | Kuchuluka kwa Androgen ndi Mlingo wa Steroid | Mlingo wa Androgen unakwera. Mlingo wa steroid unayenera kuwonjezeredwa ndi pafupifupi 6%. |
Zotsatirazi zikusonyeza bwino kuti Crinecerfont imathandiza kuwongolera kuchuluka kwa androgen, komanso kuchepetsa mlingo wa ma steroids omwe angakhale oopsa kwa odwala kwa nthawi yayitali.
Kodi mankhwalawa ndi otetezeka bwanji?
Kafukufuku wasonyeza kuti mankhwalawa alibe zotsatirapo zoyipa kwambiri. Zotsatirapo zambiri zomwe zanenedwazo ndi zazing'ono komanso zosakhalitsa.
- Kutopa
- Mutu
- Mphuno yozizira kapena yodzaza
- Kupweteka kwa minofu kapena m'mimba
- Kupweteka kwa mafupa
Chenjezo lalikulu lokhalo lomwe limabwera ndi mankhwalawa ndilakuti ngati simutenga mlingo weniweni wa ma steroid omwe dokotala wanu wakupatsani , kuchuluka kwa cortisol m'magazi anu kumatha kutsika kwambiri, zomwe zimapangitsa kuti mukhale ndi vuto loopsa lotchedwa adrenal insufficiency. Komabe, palibe amene adamwa mankhwalawo moyenera mu kafukufukuyu amene adakumanapo ndi vutoli . Chifukwa chake, ndikofunikira kwambiri kutsatira malangizo a dokotala wanu.
Chiyembekezo chatsopano cha chithandizo cha CAH
Chifukwa cha zotsatira zabwinozi, FDA (bungwe lalikulu lomwe limavomereza mankhwala) ku United States lapereka chilolezo chapadera cha mankhwalawa. Crinecerfont (dzina lodziwika kuti Crenessity) si mankhwala a CAH. Komabe, ndi sitepe yofunika kwambiri pochiza CAH.
Madokotala tsopano akhoza kukupatsani mankhwalawa kuwonjezera pa mankhwala omwe mukumwa panopa a steroid (mankhwala owonjezera) . Izi zingathandize kuchepetsa mlingo wa steroid womwe mukumwa. Izi zikutanthauza kuti simungakhale ndi zotsatirapo zoyipa zambiri kuchokera ku steroid, kuwongolera bwino kuchuluka kwa mahomoni anu, komanso kukhala ndi moyo wabwino.
Uthenga Wopita Nawo Kunyumba
- CAH (Congenital Adrenal Hyperplasia) ndi matenda a majini omwe amakhudza ma adrenal glands ndipo amachititsa kusalinganika kwa mahomoni.
- Crinecerfont (Crenessity) ndi mankhwala atsopano ovomerezeka a CAH, operekedwa kuwonjezera pa chithandizo cha steroid.
- Izi zimachepetsa kuchuluka kwa mahomoni a androgen m'thupi ndipo zingathandize kuchepetsa mlingo wa steroid tsiku lililonse.
- Mwa kuchepetsa mlingo wa ma steroid, pali mwayi wochepetsa zotsatirapo zoyipa zomwe zimachitika chifukwa chogwiritsa ntchito ma steroid kwa nthawi yayitali.
- Ngakhale kuti iyi si njira yothetsera matendawa, ndi sitepe yofunika kwambiri pakukweza moyo wa odwala a CAH.
- Ngati inu kapena mwana wanu muli ndi CAH, ndikofunikira kulankhula ndi dokotala wanu wa endocrinologist kapena dokotala wa banja kuti muwone ngati chithandizo chatsopanochi chili choyenera kwa inu .
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