Idan kai ko wani a cikin iyalinka yana da wata cuta da ake kira Congenital Adrenal Hyperplasia (CAH), wannan zai zama labari mai matuƙar muhimmanci a gare ku. Bayan shekaru 70, an amince da sabon magani musamman ga wannan cutar. Wannan babban bege ne ga mutanen da ke zaune tare da CAH. Don haka bari mu dubi menene wannan sabon magani, yadda yake aiki, da kuma fa'idodinsa.
Da farko, bari mu fahimci, menene CAH?
A taƙaice dai, CAH cuta ce ta kwayoyin halitta da ke shafar glandar adrenal ɗinmu. Waɗannan gland ɗin suna saman kodanmu. Suna samar da wasu hormones da yawa waɗanda suke da mahimmanci ga jikinmu.
Ka yi tunanin glandar adrenal a matsayin masana'anta. Babban samfurin wannan masana'anta shine hormone da ake kira cortisol. Wannan wani muhimmin hormone ne wanda ke taimaka wa jikinmu ya jure wa damuwa, rashin lafiya, da haɗari.
Idan matakan cortisol a cikin mutum mai lafiya suka ragu, kwakwalwa (kamar manajan masana'anta) tana aika sigina (oda) da ake kira ACTH zuwa ga glandar adrenal don "yin cortisol." Amma a cikin wanda ke da CAH, glandar adrenal ba ta da babban enzyme, ko 'ma'aikaci', da ake buƙata don yin cortisol. Don haka komai yawan ACTH da kwakwalwa ke aikawa, masana'antar ba za ta iya yin cortisol ba.
Wannan ya haifar da tambayoyi biyu:
1. Jiki ba ya samar da sinadarin cortisol da ake buƙata .
2. Yayin da siginar ACTH daga kwakwalwa ke ci gaba da gudana, wani nau'in hormone da glandar adrenal ke samarwa , wanda ake kira androgens, ana samar da shi fiye da kima .
Waɗannan androgens (misali testosterone) hormones ne na maza. Maza da mata suna buƙatar wasu daga cikinsu, amma a cikin CAH, suna ƙaruwa da yawa. Wannan na iya haifar da balaga da wuri, rashin haihuwa, da sauran matsaloli tare da ci gaban yaro .
Shekaru da dama, babban maganin wannan shine shan magungunan steroid (glucocorticoids) waɗanda ke aiki kamar cortisol a waje. Wannan ba wai kawai yana maye gurbin cortisol da jiki ba shi da shi ba, har ma yana sarrafa siginar ACTH daga kwakwalwa, yana rage samar da androgens. Duk da haka, matsalar ita ce wannan yana buƙatar shan magungunan steroid fiye da yadda aka saba na dogon lokaci. Wannan na iya haifar da illoli da yawa, kamar ƙara nauyi, rage ƙashi, da matsalolin barci .
Ta yaya kuka sami wannan sabon maganin?
Masana kimiyya sun yi mamakin ko akwai wata mafita ga wannan matsalar steroid. Maimakon ba da magungunan steroid da yawa a waje, me zai faru idan muka sami hanyar rage siginar ACTH da ke fitowa daga kwakwalwa kai tsaye?
A nan ne suka sami wani sabon hari. Wani mai karɓa ne a cikin kwakwalwa da ake kira CRF1. Idan matakan cortisol suka ragu, siginar ACTH tana tasowa ne ta hanyar CRF1. A cikin CAH, wannan tsari yana lalacewa.
Don haka masana kimiyya suka yi tunani, idan muka toshe wannan CRF1, za mu iya rage samar da ACTH, ko ba haka ba? Idan hakan ta faru, za a kuma sarrafa samar da androgens, kuma za a rage yawan steroids da majiyyaci ke buƙatar sha.
Wannan sabon ra'ayi shine abin da ya haifar da ƙirƙirar wani sabon magani mai suna Crinecerfont. Wannan magani ne da aka tsara don magance matsalar CAH ta asali ta hanyar toshe CRF1.
An yi gwaje-gwaje kafin a ba wa mutane magani
Ba za a iya ba wa mutane sabon magani da zarar an gano shi ba. Yana buƙatar yin gwaje-gwaje da dama don tabbatar da ko yana da aminci da tasiri. Maganin Crinecerfont ya yi duk waɗannan matakan.
1. Gwajin Dakunan Gwaji da na Dabbobi: Mataki na farko shine a gwada maganin a kan ƙwayoyin halitta da dabbobi a dakin gwaje-gwaje. Wannan shine don tantance ko maganin yana da guba ko kuma zai cutar da gabobi. Ka yi tunanin, kusan ɗaya ne kawai cikin sabbin magunguna 10,000 da ke wuce duk waɗannan ƙalubalen kuma an amince da su.
2. Gwaje-gwajen Asibiti: Bayan gwajin dabbobi ya yi nasara, ana gudanar da gwaje-gwajen mutane a matakai da dama.
Yaya sakamakon gwajin Crinecerfont yake?
Gwaje-gwajen da aka gudanar kan wannan magani sun haifar da sakamako mai kyau. Musamman gwaje-gwajen Mataki na II da Mataki na III suna da matuƙar muhimmanci.
Mataki na II - Sakamakon ƙananan yara
A nan, an ba wa ƙaramin rukuni na yara ƙanana (masu shekaru 14-17) waɗanda ke ɗauke da CAH maganin na tsawon kwanaki 14 kuma an lura da canje-canje a matakan hormones ɗinsu. Sakamakon ya ba da mamaki.
- ACTH (siginar kwakwalwa) ya ragu da kashi 57% .
- 17-OHP (alamar samar da androgen) ya ragu da kashi 69% .
- Androstenedione (wani nau'in androgen) ya ragu da kashi 58% .
Mataki na III - Sakamakon manyan ƙungiyoyi
Sakamakon waɗannan sakamakon ya ƙarfafa gwiwar masana kimiyya, sun gudanar da manyan gwaje-gwaje guda biyu ta amfani da manya da yara, inda aka bai wa wasu maganin Crinecerfont wasu kuma aka ba su placebo wanda yayi kama da magani amma bai yi wani tasiri ba.
| Ƙungiyar | Babban sakamako | Bayani |
|---|---|---|
| Manya (suna shan Crinecerfont) | Rage yawan maganin steroid | Ta hanyar sarrafa matakan hormones, ya yiwu a rage yawan shan steroids a kullum da kashi 27% . |
| Manya (sun ɗauki placebo) | Rage yawan maganin steroid | An rage yawan maganin steroid da kashi 10% kawai. |
| Yara (suna shan Crinecerfont) | Matakan Androgen da kuma yawan steroids | Bayan makonni 4, matakan androgen sun ragu sosai. A ƙarshen gwajin, an rage yawan maganin steroid da kashi 18% . |
| Yara (sun ɗauki placebo) | Matakan Androgen da kuma yawan steroids | Matakan Androgen sun ƙaru. Dole ne a ƙara yawan maganin steroid da kusan kashi 6%. |
Waɗannan sakamakon sun nuna a sarari cewa Crinecerfont yana taimakawa wajen sarrafa matakan androgen, yayin da kuma rage yawan steroids da ka iya zama illa ga marasa lafiya a cikin dogon lokaci.
Yaya wannan maganin yake da aminci?
Bincike ya nuna cewa wannan maganin ba shi da wasu illoli masu tsanani. Yawancin illolin da aka ruwaito ba su da yawa kuma na ɗan lokaci ne.
- Gajiya
- Ciwon kai
- Sanyi ko toshewar hanci
- Ciwon tsoka ko ciki
- Ciwon gaɓɓai
Babban gargaɗin da ke tare da wannan maganin shine idan ba ku ɗauki ainihin adadin steroids da likitanku ya rubuta ba , matakan cortisol ɗinku na iya raguwa sosai, wanda ke haifar da wata cuta mai barazana ga rayuwa da ake kira rashin isasshen adrenal. Duk da haka, babu wanda ya sha maganin yadda ya kamata a cikin binciken da ya taɓa fuskantar wannan yanayin . Saboda haka, yana da matuƙar muhimmanci a bi umarnin likitanku.
Sabuwar fata ga maganin CAH
Saboda waɗannan nasarorin da aka samu, FDA (babban hukumar da ke amincewa da magunguna) a Amurka ta ba da izini na musamman ga wannan maganin. Crinecerfont (sunan alamar Crenessity) ba magani bane ga CAH. Duk da haka, babban ci gaba ne a cikin maganin CAH.
Likitoci yanzu za su iya rubuta wannan maganin baya ga maganin steroid da kuke amfani da shi a yanzu (ƙarin magani) . Wannan zai iya taimakawa wajen rage yawan shan steroid. Wannan yana nufin cewa kuna iya fuskantar ƙarancin illa daga steroids, ku kula da matakan hormones ɗinku da kyau, kuma ku sami ingantacciyar rayuwa.
Saƙon Ɗauka Gida
- CAH (Congenital Adrenal Hyperplasia) cuta ce ta kwayoyin halitta da ke shafar glandar adrenal kuma tana haifar da rashin daidaiton hormonal.
- Crinecerfont (Crenessity) magani ne da aka amince da shi don CAH, ban da maganin steroid.
- Wannan yana rage yawan sinadarin androgen a jiki kuma yana iya taimakawa wajen rage yawan sinadarin steroids da ake sha a kullum.
- Ta hanyar rage yawan shan magungunan steroid, akwai damar rage illar da ke tattare da amfani da maganin steroid na dogon lokaci.
- Duk da cewa wannan ba maganin cutar ba ne, babban ci gaba ne wajen inganta rayuwar marasa lafiya na CAH.
- Idan kai ko ɗanka yana da CAH, yana da mahimmanci ka yi magana da likitan ilimin halittar jikinka ko likitan iyali don ganin ko wannan sabon maganin ya dace da kai .
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