Casgevy
Ukuthombisa kwe-Exagamglogene autotemcel kusetshenziswa ukwelapha isifo se-sickle cell (isifo sama-red blood cell) kubantu abanesimo sokuvalekisa i-vaso-occlusive njalo. Kusetshenziswa futhi ukwelapha i-beta thalassemia kubantu abadinga ukuthola igazi njalo. Lena imithi yenziwe ngama-stem cells egazi lomuntu. Isetshenziswa ukwandisa ukukhiqizwa kohlobo oluhlukile lwe-hemoglobin oluthiwa yi-hemoglobin F (i-hemoglobin yengane) esiza ekuthuthukiseni ukukhiqizwa nokwakhiwa kwama-red blood cells. Lena imithi kufanele inikezwe kuphela ngudokotela wakho noma ngaphansi kokunakekelwa kwakhe. Lo mkhiqizo uyatholakala ngezindlela zokulinganisela ezilandelayo:
Ekucineni kokusebenzisa imithi, izingozi zokuthatha imithi kufanele ziqhathaniswe nezinzuzo ezizoletha. Lo ngumqondo owenzayo wena nodokotela wakho. Kulo muthi, okulandelayo kufanele kucabangelwe: Tshela udokotela wakho uma uke waba nanoma yiluphi uhlobo oluqondile noma oluhambelana nalolu hlobo lomuthi noma eminye imithi. Tshela futhi ochwepheshe bakho bezempilo uma unanoma yiluphi olunye uhlobo lokuqabukela, njengokudla, izibhebhe, izigcini, noma izilwane. Ngomkhiqizo ongenayo iresiphi, funda ilebula noma izithako zephakheji ngokucophelela. Izifundo ezifanele azikwenzelwanga ubudlelwano bokuphila nokuthinta kokubeka i-exagamglogene autotemcel kubantwana abaneminyaka engaphansi kwengu-12. Ukuphepha nokuphumelela akukaqiniswa. Izifundo ezifanele ezihlobene nobudlelwano bokuphila nokuthinta kokubeka i-exagamglogene autotemcel azikwenzelwanga kubantu abadala. Ayikho izifundo ezanele kwabesifazane zokunquma ingozi yengane uma usebenzisa lo muthi ngesikhathi sokuncelisa. Linganisa izinzuzo ezingaba khona nezingozi ezingaba khona ngaphambi kokuthatha lo muthi ngesikhathi sokuncelisa. Nakuba ezinye izidakamizwa kufanele zingasetshenziswa ndawonye, kwezinye izimo imithi emibili ehlukene ingasetshenziswa ndawonye ngisho noma kungenzeka ukuhlangana. Kulezi zimo, udokotela wakho angase afune ukushintsha umthamo, noma ezinye izinyathelo zokuphepha zingadingeka. Uma uthola lo muthi, kubalulekile kakhulu ukuthi ochwepheshe bakho bezempilo bazi uma uthatha noma yimiphi imithi ehlongozwayo ngezansi. Ukuhlangana okulandelayo kukhethwe ngokusekelwe ekubalulekeni kwabo okungenzeka futhi akuyona yonke into. Ukusebenzisa lo muthi nanoma yimiphi imithi elandelayo ngokuvamile akunconywa, kodwa kungadingeka kwezinye izimo. Uma kokubili imithi ilotshiwe ndawonye, udokotela wakho angase ashintshe umthamo noma ukuthi uyayisebenzisa kangakanani enye noma zombili imithi. Ezinye izidakamizwa akufanele zisetshenziswe ngesikhathi sokudla noma ngesikhathi sokudla izinhlobo ezithile zokudla ngoba kungenzeka ukuhlangana. Ukusebenzisa utshwala noma utshako nanoma yimiphi imithi kungabangela ukuhlangana. Xoxa nochwepheshe bakho bezempilo ngokusetshenziswa komuthi wakho ngokudla, utshwala, noma utshako. Ukuba khona kwezinye izinkinga zezokwelapha kungathinta ukusetshenziswa kwalo muthi. Qiniseka ukuthi utshela udokotela wakho uma unanoma yiziphi ezinye izinkinga zezokwelapha, ikakhulukazi:
Udokotela noma omunye ochwepheshe bezempilo oqeqeshwe ngokucophelela uzokunika le miyekezo esibhedlela. Inikezwa nge-IV ebekwe emithanjeni. Le miyekezo kufanele ifike ne-Medication Guide nemiyalo yokuqondisa. Funda bese ulandela imiyalo ngokucophelela bese uqiniseka ukuthi uyayiqonda ngaphambi kokuthola le miyekezo. Ungase futhi uthole ezinye izidakamizwa (isib. imithi yokunciphisa ubushushu bomzimba noma imithi yokwelapha ukwanda kwesisu) ngaphambi kokuthola le miyekezo. Le miyekezo yenziwe ngokukhethekile kumuntu ngamunye, usebenzisa amangqamuzana egazi lomuntu. Udokotela wakho uzoqoqa igazi lakho ngenqubo ebizwa ngokuthi ukunyakaza nokuhlukaniswa kwamagagasi. Xoxa nodokotela wakho uma uneminye imibuzo ngalokhu. Udokotela wakho uzofuna futhi ukuthi uhlale esibhedlela noma esikhungweni okungenani amasonto angu-4 kuya kwangu-6 ngemuva kokufakwa kwakho. Ungasebenzisi i-crizanlizumab, i-hydroxyurea, ne-voxelotor okungenani amasonto angu-8 kanye ne-iron chelator okungenani izinsuku ezingu-7 ngaphambi kokuba wenze inqubo yokuqoqa igazi lakho.
Umshwana wokuzihlangula: I-August iyinkundla yolwazi lwezempilo futhi izimpendulo zayo azihlanganisi iseluleko sezokwelapha. Njalo thintana nochwepheshe bezokwelapha onelayisensi eduze nawe ngaphambi kokwenza noma yiluphi ushintsho.