I-Jakafi imelela i-paradigm yentuthuko kwezokwelapha.
Siphila ezikhathini ezimangalisayo lapho singayiqondi khona kangcono izindlela zezifo kodwa nokuthi singabhekisela kanjani lezi zinqubo ngezidakamizwa esanda kutholakala. Isibonelo, i-Jakafi (i-ruxolitinib) yaba yisidakamizwa sokuqala se-FDA esamukelekile ukuphatha i- polycythemia vera , futhi isebenza ngokuvimbela ama-enzymes Janus Associated Kinase 1 (JAK-1) noJanus Associated Kinase 2 (JAK-2).
Kanye nezinye izinguquko zamangqamuzana, la ma-enzyme aya ku-haywire kubantu abane polycythemia vera.
Yini I-Polycythemia Vera?
I-Polycythemia vera iyisifo esingavamile segazi. Lesi sifo esiyinkimbinkimbi esivame ukuveza esikhathini esizayo empilweni (abantu abaneminyaka engama-60) futhi ekugcineni kubangele i-thrombosis (cabanga ukuphazamiseka) kwesinye kwesithathu sabo bonke abantu abathintekayo. Njengoba sonke sazi, ukushaywa yisifo kungaba yingozi ngakho ukuxilongwa kwe-PV kuyinto enhle kakhulu.
Indaba yokuthi ukusebenza kwe-PV kuqala kanjani emangeni yethambo. Umnkantsha wethu wethambo unesibopho sokwenza amangqamuzana egazi lethu. Izinhlobo ezahlukene zamangqamuzana egazi emzimbeni wethu zinendima ehlukile. Amangqamuzana egazi elibomvu ahambisa oksijini ezithombeni nasezigungwini zethu, amaseli egazi amhlophe asiza ukulwa nokutheleleka kanye namaplatelets ayeke ukuphuma. Kubantu abane-PV, kukhona ukuguqulwa kwamaseli amaningi we-hematopoietic okuholela ekukhiqizeni ngokweqile kwamangqamuzana egazi abomvu, amaseli egazi amhlophe, namaplatelets. Ngamanye amazwi, ku-PV, amangqamuzana angama-progenitor, ahlukanisa amangqamuzana egazi abomvu, amangqamuzana egazi amhlophe, namaplatelets, aphonswa ekudleni okuningi.
Okuncane kakhulu kunoma yini engalungile, futhi uma kwenzeka ku-PV, amangqamuzana amaningi egazi angakwazi ukugcoba imithwalo yethu yegazi eyenza zonke izinhlobo zezinkinga zemitholampilo kuhlanganise okulandelayo:
- ikhanda
- ubuthakathaka
- i-pruritis (itching okuyinto ngokwezifiso iveza emva eshaweni eshisayo noma ibhuku)
- isiyezi
- ukuthuthumela
- i-thrombosis noma i-clotting ngokweqile yegazi (ama-clots wegazi angagubuza imishanguzo futhi abange isifo, isifo se-heart, ne-pulmary embolism, noma imivimbo ye-clog efana ne-veal portal edla isibindi esabangela ukulimala kwesibindi.)
- ukuphuma kwegazi (Amakhomikhali amaningi kakhulu egazi-amaningi awo angamaplatelets angalungile-angabangela ukuphuma kwegazi)
- i-splenomegaly (I-spleen, ehlunga amangqamuzana egazi abomvu afile, evuvukala ngenxa yenani elikhulayo lamaseli egazi ku-PV.)
- I-erythromelalgia (Ubuhlungu nokufudumala kumadijithali kubangelwa amapleletthi amaningi kakhulu avimbela ukugeleza kwegazi eminwe nasezinzwaneni ezingaholela ekufakeni idijithi yokufa nokuxoshwa.)
I-PV ingabuye ihlanganise nezinye izifo ezifana nesifo se-coronary and pulpertension pulmonary kokubili ngenxa yenani elikhudlwana lamaseli egazi eligeleza ukujikeleza nokuhamba kahle kwe-muscle hyperplasia noma ngokweqile okuqhubeka nokugxila kwegazi. (Imisipha enomsindo yenza izindonga zemithambo yethu yegazi, futhi inani elikhulayo lamaseli egazi cishe likhulula izici eziningi zokukhula ezenza ukuba imisipha evulekile ikhule.)
Abambalwa abantu abane-PV bayaqhubeka nokuthuthukisa i-myelofibrosis (lapho umthambo wamathambo uchithelwa khona noma "uchithekile" futhi ugcwele ama-fibroblasts angasebenzi namagciwane agcwalisa i-anemia) futhi ekugcineni ngisho ne-leukemia ephawulekayo. Khumbula ukuthi i-PV ivame ukubizwa ngokuthi i- myeloproliferative neoplasm noma umdlavuza ngoba njengamanye amagciwane aholela ekukhuleni kwamathambo ezinombolo zamaselula. Ngeshwa, kwabanye abantu abane-PV, i-leukemia imele ukuphela komugqa ngokuqhubeka komdlavuza.
I-Jakafi: I-Drug Ephikisana Ne-Polycythemia Vera
Abantu esigabeni se-PV noma esigabeni esibonakala ngenani elikhulayo lamaseli egazi liphathwa ngezindlela zokwelapha ezinciphisa izimpawu nokuthuthukisa ikhwalithi yokuphila. Okudume kakhulu kulezi zonyango kungenzeka ukuthi i- phlebotomy noma ukuphuma kwegazi ukunciphisa izibalo zegazi.
Abachwepheshe baphinde baphathe i-PV nge-myelosuppressive (cabanga ngamakhemikhalirapeutic) ama-agent-hydroxyurea, busulfan, 32p futhi, kamuva nje, i-interferon-okuvimbela ukukhiqizwa ngokweqile kwamangqamuzana egazi. Ukwelashwa kwe-myelosuppressive kwandisa umuzwa womuthi wesiguli futhi kucatshangwa ukusiza abantu abanePV baphile isikhathi eside.
Ngeshwa, ezinye zalezi zidakamizwa ezifana ne-chlorambucil zithwala ingozi yokudala i-leukemia.
Kubantu abane-PV abanenkinga yokubekezelela noma abangayitholi i-hydroxyurea, i-agent ye-myelosuppressive, i-Jakafi yamukelwa yi-FDA ngoDisemba 2014. I-Jakafi isebenza ngokuvimbela i-JAK-1 ne-JAK-2 enzyme ethintwa kubantu abaningi nge-PV. La ma-enzyme abandakanyeka egazini nasekusebenzeni kwe-immunological, izinqubo ezingavamile kubantu abane-PV.
Ezingamaphesenti angu-21 zabantu abangenakubekezelelana noma abangathobeli i-hydroxyurea, ucwaningo lubonisa ukuthi i-Jakafi inciphisa usayizi we-spleen (iyanciphisa i-splenomegaly) futhi inciphisa isidingo se-phlebotomy. Ucwaningo lubonisa ukuthi ngisho nangokwelashwa okunye okungcono kakhulu okutholakala, amaphesenti angu-1 kuphela abantu abanjalo bebengase bahlangabezane nokuzuza okunjalo. Ngokweqile, i-Jakafi yayivunyelwe ngaphambilini yi-FDA yokwelashwa kwe-myelofibrosis ngo-2011. Imiphumela embi kakhulu e-Jakafi (okuyinto i-FDA ikhuluma ngokuthi "imiphumela emibi") ifaka i-anemia, ukubalwa kweplatelet ephansi yegazi, isizungu, ukuqothulwa, nokukhwabanisa.
Kumele kuqashelwe ukuthi njengoba kunjalo nangezinye izindlela zokwelapha izidakamizwa, akucaci ukuthi ngabe uJakafi uzosiza abantu baphile isikhathi eside.
Uma wena noma umuntu omthandayo ane-PV engaphenduli i-hydroxyurea, i-Jakafi imelela ukwelashwa okusha okuthembisayo. Konke thina, i-Jakafi imelela umbono omkhulu wokuthi izidakamizwa eziningi zizothuthukiswa kanjani. Abacwaningi bayaphumelela ekuqaliseni ukuthi yiziphi izindlela ezihlukunyezwa yizifo futhi zibhekiswe kulokhu kuphila.
Imithombo:
"Ukwelashwa kwama-myeloproliferative neoplasms: nini, yiyiphi i-agent, nokuthi kanjani?" ngu-HL Geyer no-RA Mesa eshicilelwe ngegazi ngo-12/4/2014.
I-Prchal JT, i-Prchal JF. Isahluko 86. Polycythemia Vera. Ku: Lichtman MA, Kipps TJ, Seligsohn U, Kaushansky K, Prchal JT. ama-eds. Williams Hematology, 8e . ENew York, NY: McGraw-Hill; 2010.