Izifo ZamaKrabbe Izimpawu, Ukuxilongwa, Nokunakekelwa

Isifo se-Krabbe yisimo esibuhlungu esithinta isimiso sezinzwa

Isifo se-Krabbe, esaziwa nangokuthi i-globoid cell leukodystrophy, isifo esiwumzimba esiyingozi esithinta isistimu yezinzwa. Abantu abanesifo se-Krabbe banezakhi zofuzo emthonjeni wabo we-GALC. Ngenxa yalezi zinguquko, azikhiqizi ngokwanele i-enzyme galactosylceramidase. Lokhu kwehluleka kubangela ukulahlekelwa okuqhubekayo kwendwangu yokuzivikela ehlanganisa amangqamuzana omzimba, okuthiwa i- myelin .

Ngaphandle kwalolu cingo oluvikelayo, izinzwa zethu azikwazi ukusebenza kahle, zilimaza ubuchopho bethu nesistimu yethu yezinzwa.

Isifo se-Krabbe sithinta abantu abangaba ngu-1 kubantu abayi-100,000 emhlabeni jikelele, okwenzekayo okushiwo ezindaweni ezithile kwa-Israyeli.

Izimpawu ze-Krabbe Disease

Kunezinhlobo ezine zesifo se-Krabbe, ngasinye sinokuqala kwazo kanye nezimpawu zabo.

Thayipha 1 Infantile Iqala phakathi kwezinyanga ezingu-3 kuya kwezi-6 ubudala
Thayipha 2 I-Early Childhood Iqala phakathi kwezinyanga ezingu-6 no-3 ubudala
Thayipha u-3 Izingane Iqala phakathi kweminyaka engu-3 kuya kwangu-8 ubudala
Thayipha 4 Ukuqala kwabantu abadala Iqala noma kunini ngemva kweminyaka engu-8 ubudala

Njengoba isifo se-Krabbe sithinta amangqamuzana omzimba, eziningi zezimpawu ezibangelwa yizinzwa ze-neurological. Thayipha u-1, ifomu elincane, libala amacala angu-85 kuya kwangu-90 wamacala. Thayipha 1 ithuthuka ngezigaba ezintathu:

Ezinye izinhlobo zesifo sikaKrabbe ziqala emva kwesikhathi sokuthuthukiswa okuvamile. Lezi zinhlobo nazo ziyaqhubeka zihamba kancane kunokuba u-Type 1 lwenze. Izingane ngokuvamile aziphili eminyakeni engaphezu kwemibili ngemuva kokuthi Uhlobo 2 luqala. Isikhathi sokuphila sezinhlobo ezingu-3 no-4 ziyahlukahluka, futhi izimpawu kungenzeka ukuthi azibi kakhulu.

Yeka ukuthi izifo zakwaKrabbe zixilongwa kanjani

Uma izimpawu zezingane zakho zikhomba isifo se-Krabbe, ukuhlolwa kwegazi kungenziwa ukuze ubone ukuthi banesifo se-galactosylceramidase, okuqinisekisiwe ukuxilongwa kwesifo se-Krabbe. Ukukhishwa kwe-lumbar kungenziwa ukusampula uketshezi lwe-cerebrospinal. Amazinga amaprotheni aphakeme kakhulu angabonisa lesi sifo. Ukuze ingane izalwe nesimo, bobabili abazali kufanele baphathe isakhi se-mutated - esitholakala ku-chromosome 14. Uma abazali bephethe isakhi, ingane yabo engakazalwa ingahlolwa i-galactosylceramidase. Ezinye izifunda zinikeza isifo esiseKrabbe esanda kuzalwa.

Ukwelapha

Akukho ukwelashwa kwesifo se-Krabbe. Ucwaningo lwamuva luye lwabhekisela ku-hematopoietic stem cell transplantation ( HSCT ), amangqamuzana amancane avela abe amangqamuzana egazi, njengendlela yokwelapha okungase kube khona. I-HSCT isebenza kangcono uma inikezwa iziguli ezingakaze zibonise izimpawu noma zinezimpawu ezibonisa ukuthi zinezimpawu ezibonakalayo.Ukwelapha kusebenza kahle uma kunikezwa ngenyanga yokuqala yokuphila.

I-HCST ingabazuzisa labo abanezifo ezincane noma izinkinga eziqhubekayo kancane kancane kanye nezingane ezinesifo esincane sokuqala uma sinikezwa masinyane ngokwanele. Ngenkathi i-HCST ingelapheki lesi sifo, inganikeza ikhwalithi engcono yokuphila - ukulibazisa ukukhula kwesifo nokukhulisa isikhathi sokuphila. Kodwa-ke, i-HCST iza nezingozi zayo futhi inezinga lokufa ngamaphesenti angu-15.

Ucwaningo lwamanje luphenyisisa ukwelashwa okubhekiswe emakhonini okuvuvukala, ukwelapha okushintshwa kwe-enzyme, ukwelashwa kwegciwane, nokuguqulwa kwe-neural stem cell cell. Lezi zindlela zokwelashwa zikhona zonke izigaba zabo zokuqala futhi azikakabi ukuhlolwa kokuhlolwa kwemitholampilo.

Imininingwane yalesi sihloko ithathwe kusuka:
I-Tegay, i-DH (2014). Isifo se-Krabbe. eMedicine.