{"id":26758,"date":"2023-04-22T23:12:30","date_gmt":"2023-04-22T16:12:30","guid":{"rendered":"http:\/\/noithathoaphat.in\/ctv\/?p=26758"},"modified":"2023-04-22T23:12:30","modified_gmt":"2023-04-22T16:12:30","slug":"leniolisib-phuong-phap-moi-trong-dieu-tri-apds","status":"publish","type":"post","link":"http:\/\/noithathoaphat.in\/ctv\/index.php\/2023\/04\/22\/leniolisib-phuong-phap-moi-trong-dieu-tri-apds\/","title":{"rendered":"Leniolisib: ph\u01b0\u01a1ng ph\u00e1p m\u1edbi trong \u0111i\u1ec1u tr\u1ecb APDS"},"content":{"rendered":"

Leniolisib l\u00e0 m\u1ed9t lo\u1ea1i thu\u1ed1c \u1ee9c ch\u1ebf phosphoinositide 3-kinase (PI3K) \u0111\u01b0\u1ee3c s\u1eed d\u1ee5ng trong \u0111i\u1ec1u tr\u1ecb APDS – m\u1ed9t lo\u1ea1i b\u1ec7nh mi\u1ec5n d\u1ecbch\u00a0 li\u00ean quan \u0111\u1ebfn t\u1ebf b\u00e0o T v\u00e0 B. APDS c\u00f3 th\u1ec3 g\u00e2y ra c\u00e1c tri\u1ec7u ch\u1ee9ng nh\u01b0 vi\u00eam xoang, vi\u00eam \u0111\u01b0\u1eddng h\u00f4 h\u1ea5p, nhi\u1ec5m tr\u00f9ng v\u00e0 t\u1ed5n th\u01b0\u01a1ng t\u1ebf b\u00e0o th\u1ea7n kinh. Leniolisib l\u00e0 m\u1ed9t ph\u01b0\u01a1ng ph\u00e1p m\u1edbi trong \u0111i\u1ec1u tr\u1ecb APDS v\u00e0 \u0111\u01b0\u1ee3c ph\u00e1t tri\u1ec3n v\u00e0 ch\u1ea5p thu\u1eadn \u0111\u1ec3 gi\u1ea3m thi\u1ec3u c\u00e1c tri\u1ec7u ch\u1ee9ng v\u00e0 c\u1ea3i thi\u1ec7n ch\u1ea5t l\u01b0\u1ee3ng cu\u1ed9c s\u1ed1ng c\u1ee7a b\u1ec7nh nh\u00e2n.<\/strong><\/p>\n

1.T\u00ecm hi\u1ec3u v\u1ec1 h\u1ed9i ch\u1ee9ng phosphoinositide 3-kinase delta (APDS)<\/strong><\/h2>\n

1.1 Gi\u1edbi thi\u1ec7u chung\u00a0<\/span><\/h3>\n

H\u1ed9i ch\u1ee9ng PI3K-delta k\u00edch ho\u1ea1t l\u00e0 m\u1ed9t b\u1ec7nh suy gi\u1ea3m mi\u1ec5n d\u1ecbch nguy\u00ean ph\u00e1t. B\u1ec7nh nh\u00e2n m\u1eafc h\u1ed9i ch\u1ee9ng APDS c\u00f3 c\u00e1c bi\u1ec3u hi\u1ec7n t\u1ef1 mi\u1ec5n d\u1ecbch v\u00e0 t\u0103ng sinh lympho \u0111\u00e1ng k\u1ec3. M\u1ed9t \u0111\u1eb7c \u0111i\u1ec3m kh\u00e1c c\u00f3 th\u1ec3 c\u00f3 c\u1ee7a h\u1ed9i ch\u1ee9ng PI3K-delta \u0111\u01b0\u1ee3c k\u00edch ho\u1ea1t (APDS) l\u00e0 s\u1ef1 k\u1ebft d\u00ednh b\u1ea5t th\u01b0\u1eddng c\u1ee7a c\u00e1c t\u1ebf b\u00e0o b\u1ea1ch c\u1ea7u t\u1ea1o kh\u1ed1i u d\u1eabn \u0111\u1ebfn h\u1ea1ch b\u1ea1ch huy\u1ebft to ra (b\u1ec7nh h\u1ea1ch b\u1ea1ch huy\u1ebft) ho\u1eb7c c\u00e1c t\u1ebf b\u00e0o b\u1ea1ch c\u1ea7u c\u00f3 th\u1ec3 t\u00edch t\u1ee5 th\u00e0nh kh\u1ed1i r\u1eafn (t\u0103ng s\u1ea3n h\u1ea1ch b\u1ea1ch huy\u1ebft d\u1ea1ng n\u1ed1t) th\u01b0\u1eddng \u1edf l\u1edbp ni\u00eam m\u1ea1c \u1edf \u0111\u01b0\u1eddng h\u00f4 h\u1ea5p ho\u1eb7c \u0111\u01b0\u1eddng ti\u00eau h\u00f3a. M\u1eb7c d\u00f9 b\u1ec7nh h\u1ea1ch b\u1ea1ch huy\u1ebft v\u00e0 t\u0103ng s\u1ea3n b\u1ea1ch huy\u1ebft d\u1ea1ng n\u1ed1t kh\u00f4ng ph\u1ea3i ung th\u01b0 nh\u01b0ng APDS c\u0169ng l\u00e0m t\u0103ng nguy c\u01a1 ph\u00e1t tri\u1ec3n m\u1ed9t d\u1ea1ng ung th\u01b0 g\u1ecdi l\u00e0 u lympho t\u1ebf b\u00e0o B<\/span><\/p>\n

1.2 Sinh l\u00fd b\u1ec7nh v\u00e0 di truy\u1ec1n c\u1ee7a APDS<\/span><\/h3>\n

H\u1ed9i ch\u1ee9ng PI3K-delta \u0111\u01b0\u1ee3c k\u00edch ho\u1ea1t l\u00e0 do \u0111\u1ed9t bi\u1ebfn gen PIK3CD , gen n\u00e0y cung c\u1ea5p h\u01b0\u1edbng d\u1eabn \u0111\u1ec3 t\u1ea1o ra m\u1ed9t lo\u1ea1i protein g\u1ecdi l\u00e0 p110 delta (p110\u03b4). Protein n\u00e0y l\u00e0 m\u1ed9t ph\u1ea7n (ti\u1ec3u \u0111\u01a1n v\u1ecb) c\u1ee7a enzym c\u00f3 t\u00ean l\u00e0 phosphatidylinositol 3-kinase (PI3K), enzym n\u00e0y k\u00edch ho\u1ea1t c\u00e1c \u0111\u01b0\u1eddng truy\u1ec1n t\u00edn hi\u1ec7u trong t\u1ebf b\u00e0o. Phi\u00ean b\u1ea3n PI3K ch\u1ee9a ti\u1ec3u \u0111\u01a1n v\u1ecb p110\u03b4, \u0111\u01b0\u1ee3c g\u1ecdi l\u00e0 PI3K-delta, \u0111\u01b0\u1ee3c t\u00ecm th\u1ea5y \u0111\u1eb7c bi\u1ec7t trong c\u00e1c t\u1ebf b\u00e0o b\u1ea1ch c\u1ea7u, bao g\u1ed3m t\u1ebf b\u00e0o B v\u00e0 t\u1ebf b\u00e0o T. T\u00edn hi\u1ec7u PI3K-delta li\u00ean quan \u0111\u1ebfn s\u1ef1 ph\u00e1t tri\u1ec3n v\u00e0 ph\u00e2n chia (sinh s\u00f4i n\u1ea3y n\u1edf) c\u1ee7a c\u00e1c t\u1ebf b\u00e0o b\u1ea1ch c\u1ea7u, \u0111\u1ed3ng th\u1eddi gi\u00fap \u0111\u1ecbnh h\u01b0\u1edbng c\u00e1c t\u1ebf b\u00e0o B v\u00e0 t\u1ebf b\u00e0o T tr\u01b0\u1edfng th\u00e0nh (bi\u1ec7t h\u00f3a) th\u00e0nh c\u00e1c lo\u1ea1i kh\u00e1c nhau, m\u1ed7i lo\u1ea1i c\u00f3 m\u1ed9t ch\u1ee9c n\u0103ng ri\u00eang bi\u1ec7t trong h\u1ec7 th\u1ed1ng mi\u1ec5n d\u1ecbch.<\/span><\/p>\n

\"Sinh
Sinh l\u00fd b\u1ec7nh APDS<\/figcaption><\/figure>\n

PIK3CA \u0111\u1ed9t bi\u1ebfn gen li\u00ean quan \u0111\u1ebfn h\u1ed9i ch\u1ee9ng PI3K-delta \u0111\u00e3 ho\u1ea1t h\u00f3a d\u1eabn \u0111\u1ebfn s\u1ea3n xu\u1ea5t protein p110\u03b4 b\u1ecb bi\u1ebfn \u0111\u1ed5i. Enzyme PI3K-delta ch\u1ee9a ti\u1ec3u \u0111\u01a1n v\u1ecb b\u1ecb bi\u1ebfn \u0111\u1ed5i \u0111\u01b0\u1ee3c b\u1eadt (k\u00edch ho\u1ea1t) m\u1ed9t c\u00e1ch b\u1ea5t th\u01b0\u1eddng. C\u00e1c nghi\u00ean c\u1ee9u ch\u1ec9 ra r\u1eb1ng t\u00edn hi\u1ec7u PI3K-delta ho\u1ea1t \u0111\u1ed9ng qu\u00e1 m\u1ee9c l\u00e0m thay \u0111\u1ed5i s\u1ef1 bi\u1ec7t h\u00f3a c\u1ee7a t\u1ebf b\u00e0o B v\u00e0 t\u1ebf b\u00e0o T, d\u1eabn \u0111\u1ebfn vi\u1ec7c s\u1ea3n xu\u1ea5t c\u00e1c t\u1ebf b\u00e0o kh\u00f4ng th\u1ec3 ph\u1ea3n \u1ee9ng v\u1edbi nhi\u1ec5m tr\u00f9ng v\u00e0 ch\u1ebft s\u1edbm h\u01a1n b\u00ecnh th\u01b0\u1eddng. Thi\u1ebfu t\u1ebf b\u00e0o B v\u00e0 t\u1ebf b\u00e0o T ho\u1ea1t \u0111\u1ed9ng khi\u1ebfn nh\u1eefng ng\u01b0\u1eddi m\u1eafc ch\u1ee9ng r\u1ed1i lo\u1ea1n n\u00e0y kh\u00f3 ch\u1ed1ng l\u1ea1i nhi\u1ec5m tr\u00f9ng do vi khu\u1ea9n v\u00e0 vi r\u00fat. K\u00edch ho\u1ea1t qu\u00e1 m\u1ee9c t\u00edn hi\u1ec7u PI3K-delta c\u0169ng c\u00f3 th\u1ec3 k\u00edch th\u00edch s\u1ef1 t\u0103ng sinh b\u1ea5t th\u01b0\u1eddng c\u1ee7a c\u00e1c t\u1ebf b\u00e0o b\u1ea1ch c\u1ea7u, d\u1eabn \u0111\u1ebfn b\u1ec7nh h\u1ea1ch b\u1ea1ch huy\u1ebft v\u00e0 t\u0103ng s\u1ea3n b\u1ea1ch huy\u1ebft d\u1ea1ng n\u1ed1t \u1edf m\u1ed9t s\u1ed1 ng\u01b0\u1eddi b\u1ecb \u1ea3nh h\u01b0\u1edfng. S\u1ef1 gia t\u0103ng t\u0103ng sinh t\u1ebf b\u00e0o B k\u1ebft h\u1ee3p v\u1edbi suy gi\u1ea3m ch\u1ee9c n\u0103ng h\u1ec7 th\u1ed1ng mi\u1ec5n d\u1ecbch c\u00f3 th\u1ec3 g\u00f3p ph\u1ea7n ph\u00e1t tri\u1ec3n u lympho t\u1ebf b\u00e0o B. C\u00e1c t\u1ebf b\u00e0o B v\u00e0 t\u1ebf b\u00e0o T b\u1ea5t th\u01b0\u1eddng c\u0169ng c\u00f3 th\u1ec3 t\u1ea5n c\u00f4ng nh\u1ea7m c\u00e1c t\u1ebf b\u00e0o c\u01a1 th\u1ec3 b\u00ecnh th\u01b0\u1eddng (\u0111i\u1ec1u n\u00e0y \u0111\u01b0\u1ee3c g\u1ecdi l\u00e0 t\u1ef1 mi\u1ec5n d\u1ecbch), c\u00f3 th\u1ec3 d\u1eabn \u0111\u1ebfn c\u00e1c t\u00ecnh tr\u1ea1ng nh\u01b0 thi\u1ebfu m\u00e1u ho\u1eb7c s\u1ed1 l\u01b0\u1ee3ng ti\u1ec3u c\u1ea7u th\u1ea5p khi\u1ebfn c\u01a1 th\u1ec3 kh\u00f4ng th\u1ec3 h\u00ecnh th\u00e0nh c\u1ee5c m\u00e1u \u0111\u00f4ng v\u00e0 c\u00f3 th\u1ec3 g\u00e2y ch\u1ea3y m\u00e1u nghi\u00eam tr\u1ecdng.<\/span><\/p>\n

1.3 Ch\u1ea9n \u0111o\u00e1n APDS<\/span><\/h3>\n

Vi\u1ec7c ch\u1ea9n \u0111o\u00e1n APDS d\u1ef1a tr\u00ean c\u00e1c d\u1ea5u hi\u1ec7u ho\u1eb7c tri\u1ec7u ch\u1ee9ng m\u00e0 m\u1ed9t c\u00e1 nh\u00e2n bi\u1ec3u hi\u1ec7n, <\/span>c\u00e1c x\u00e9t nghi\u1ec7m trong ph\u00f2ng th\u00ed nghi\u1ec7m (\u0111\u1ebfm s\u1ed1 l\u01b0\u1ee3ng c\u00e1c lo\u1ea1i t\u1ebf b\u00e0o lympho kh\u00e1c nhau <\/span>nh\u01b0 t\u1ebf b\u00e0o B v\u00e0 t\u1ebf b\u00e0o T) v\u00e0 x\u00e9t nghi\u1ec7m di truy\u1ec1n (x\u00e1c \u0111\u1ecbnh ki\u1ec3u gen)<\/span><\/p>\n

Bi\u1ec3u hi\u1ec7n l\u00e2m s\u00e0ng: B\u1ec7nh nh\u00e2n m\u1eafc APDS c\u00f3 th\u1ec3 xu\u1ea5t hi\u1ec7n \u1edf th\u1eddi th\u01a1 \u1ea5u ho\u1eb7c sau n\u00e0y <\/span>khi l\u1edbn l\u00ean v\u1edbi t\u00ecnh tr\u1ea1ng nhi\u1ec5m vi khu\u1ea9n v\u00e0 virus nghi\u00eam tr\u1ecdng, dai d\u1eb3ng v\u00e0 t\u00e1i <\/span>ph\u00e1t, th\u01b0\u1eddng \u1edf ph\u1ed5i, m\u0169i ho\u1eb7c tai, ho dai d\u1eb3ng th\u01b0\u1eddng c\u00f3 \u0111\u1eddm, h\u1ea1ch b\u1ea1ch huy\u1ebft s\u01b0ng <\/span>to v\u00e0 th\u01b0\u1eddng xuy\u00ean v\u1edbi m\u1ed9t l\u00e1 l\u00e1ch v\u00e0 gan m\u1edf r\u1ed9ng. B\u1ec7nh nh\u00e2n tr\u1ebb tu\u1ed5i c\u00f3 th\u1ec3 b\u1ecb ch\u1eadm <\/span>ph\u00e1t tri\u1ec3n v\u00e0 t\u1ea5t c\u1ea3 b\u1ec7nh nh\u00e2n c\u00f3 th\u1ec3 c\u00f3 d\u1ea5u hi\u1ec7u t\u1ef1 mi\u1ec5n d\u1ecbch v\u00e0 t\u00ecnh tr\u1ea1ng vi\u00eam <\/span>nhi\u1ec5m. Ch\u00fang c\u00f3 th\u1ec3 bao g\u1ed3m thi\u1ebfu m\u00e1u, s\u1ed1 l\u01b0\u1ee3ng ti\u1ec3u c\u1ea7u th\u1ea5p, vi\u00eam ru\u1ed9t k\u1ebft (vi\u00eam \u0111\u1ea1i <\/span>tr\u00e0ng, th\u01b0\u1eddng th\u1ea5y l\u00e0 ti\u00eau ch\u1ea3y dai d\u1eb3ng v\u00e0 \u0111au b\u1ee5ng k\u00e9o d\u00e0i) v\u00e0 vi\u00eam th\u1eadn (vi\u00eam c\u1ea7u <\/span>th\u1eadn, n\u01b0\u1edbc ti\u1ec3u c\u00f3 m\u00e0u h\u1ed3ng ho\u1eb7c n\u00e2u v\u00e0 s\u01b0ng tay, ch\u00e2n , m\u1eb7t v\u00e0 b\u1ee5ng)<\/span><\/p>\n

C\u00e1c x\u00e9t nghi\u1ec7m trong ph\u00f2ng th\u00ed nghi\u1ec7m: Do s\u1ef1 s\u1ea3n xu\u1ea5t b\u1ea5t th\u01b0\u1eddng c\u1ee7a c\u00e1c t\u1ebf b\u00e0o b\u1ea1ch <\/span>c\u1ea7u, n\u1ed3ng \u0111\u1ed9 t\u1ebf b\u00e0o T t\u0103ng l\u00ean. M\u1eb7c d\u00f9 c\u00f3 s\u1ed1 l\u01b0\u1ee3ng l\u1edbn c\u00e1c t\u1ebf b\u00e0o b\u1ea1ch c\u1ea7u, nh\u01b0ng c\u00e1c t\u1ebf b\u00e0o n\u00e0y kh\u00f4ng ho\u1ea1t \u0111\u1ed9ng b\u00ecnh th\u01b0\u1eddng v\u00e0 \u0111i\u1ec1u n\u00e0y d\u1eabn \u0111\u1ebfn vi\u1ec7c gi\u1ea3m s\u1ea3n xu\u1ea5t c\u00e1c kh\u00e1ng th\u1ec3 g\u1ecdi l\u00e0 Globulin mi\u1ec5n d\u1ecbch trong huy\u1ebft t\u01b0\u01a1ng<\/span><\/p>\n

X\u00e9t nghi\u1ec7m di truy\u1ec1n: Gi\u1ea3i tr\u00ecnh t\u1ef1 c\u00e1c gen \u0111\u01b0\u1ee3c bi\u1ebft l\u00e0 c\u00f3 li\u00ean quan \u0111\u1ebfn APDS cung c\u1ea5p <\/span>ch\u1ea9n \u0111o\u00e1n x\u00e1c \u0111\u1ecbnh b\u1ec7nh<\/span><\/p>\n

1.4 Ph\u01b0\u01a1ng ph\u00e1p \u0111i\u1ec1u tr\u1ecb \u0111\u01b0\u1ee3c \u0111\u1ec1 xu\u1ea5t cho APDS<\/span><\/h3>\n

Nhi\u1ec5m tr\u00f9ng n\u00ean \u0111\u01b0\u1ee3c ki\u1ec3m so\u00e1t nhanh ch\u00f3ng b\u1eb1ng thu\u1ed1c kh\u00e1ng sinh (nhi\u1ec5m vi khu\u1ea9n) <\/span>ho\u1eb7c thu\u1ed1c kh\u00e1ng virus (nhi\u1ec5m virus). \u0110\u1ed1i v\u1edbi nh\u1eefng b\u1ec7nh nh\u00e2n s\u1ea3n xu\u1ea5t kh\u00e1ng th\u1ec3 k\u00e9m, c\u00f3 th\u1ec3 \u00e1p d\u1ee5ng ph\u01b0\u01a1ng ph\u00e1p \u0111i\u1ec1u tr\u1ecb thay th\u1ebf b\u1eb1ng Globulin mi\u1ec5n d\u1ecbch. Gh\u00e9p t\u1ebf b\u00e0o g\u1ed1c t\u1ea1o m\u00e1u (HSCT) l\u00e0 l\u1ef1a ch\u1ecdn ch\u1eefa b\u1ec7nh duy nh\u1ea5t v\u00e0 c\u00f3 th\u1ec3 xem x\u00e9t r\u1ee7i ro cho nh\u1eefng b\u1ec7nh nh\u00e2n m\u1eafc APDS n\u1eb7ng. M\u1ed9t s\u1ed1 lo\u1ea1i thu\u1ed1c (ch\u1eb3ng h\u1ea1n nh\u01b0 steroid, sirolimus ho\u1eb7c kh\u00e1ng th\u1ec3 \u0111\u01a1n d\u00f2ng Rituximab) c\u00f3 th\u1ec3 thay \u0111\u1ed5i ph\u1ea3n \u1ee9ng c\u1ee7a h\u1ec7 th\u1ed1ng mi\u1ec5n d\u1ecbch \u0111\u1ec3 ch\u00fang ph\u00f9 h\u1ee3p v\u1edbi nh\u1eefng b\u1ec7nh nh\u00e2n c\u00f3 c\u00e1c \u0111\u1eb7c \u0111i\u1ec3m t\u1ef1 mi\u1ec5n d\u1ecbch nh\u01b0 s\u1ed1 l\u01b0\u1ee3ng t\u1ebf b\u00e0o m\u00e1u th\u1ea5p, b\u1ec7nh th\u1eadn, vi\u00eam kh\u1edbp,…<\/span><\/p>\n

2.Th\u00f4ng tin v\u1ec1 thu\u1ed1c m\u1edbi Leniolisib trong \u0111i\u1ec1u tr\u1ecb APDS<\/strong><\/h2>\n

M\u1edbi \u0111\u00e2y nh\u1ea5t, FDA \u0111\u00e3 ph\u00ea duy\u1ec7t Leniolisib d\u01b0\u1edbi d\u1ea1ng vi\u00ean n\u00e9n Joenja l\u00e0 ph\u01b0\u01a1ng ph\u00e1p \u0111i\u1ec1u tr\u1ecb \u0111\u1ea7u ti\u00ean cho h\u1ed9i ch\u1ee9ng delta 3-kinase phosphoinositide ho\u1ea1t h\u00f3a (APDS) \u1edf b\u1ec7nh nh\u00e2n ng\u01b0\u1eddi l\u1edbn v\u00e0 tr\u1ebb em t\u1eeb 12 tu\u1ed5i tr\u1edf l\u00ean. Hi\u1ec7u qu\u1ea3 c\u1ee7a Joenja \u0111\u01b0\u1ee3c \u0111\u00e1nh gi\u00e1 th\u00f4ng qua m\u1ed9t nghi\u00ean c\u1ee9u ng\u1eabu nhi\u00ean, m\u00f9, c\u00f3 \u0111\u1ed1i ch\u1ee9ng v\u1edbi gi\u1ea3 d\u01b0\u1ee3c trong 12 tu\u1ea7n tr\u00ean 31 b\u1ec7nh nh\u00e2n ng\u01b0\u1eddi l\u1edbn v\u00e0 tr\u1ebb em t\u1eeb 12 tu\u1ed5i tr\u1edf l\u00ean m\u1eafc ph\u1ea3i PI3K\u03b4 di truy\u1ec1n li\u00ean quan \u0111\u1ebfn APDS \u0111\u00e3 \u0111\u01b0\u1ee3c x\u00e1c nh\u1eadn \u0111\u1ed9t bi\u1ebfn, v\u1edbi m\u1ed9t bi\u1ebfn th\u1ec3 \u0111\u01b0\u1ee3c ghi nh\u1eadn l\u00e0 PIK3CD ho\u1eb7c PIK3R1. Trong qu\u00e1 tr\u00ecnh nghi\u00ean c\u1ee9u, 21 b\u1ec7nh nh\u00e2n \u0111\u01b0\u1ee3c d\u00f9ng 70mg Joenja v\u00e0 10 b\u1ec7nh nh\u00e2n d\u00f9ng gi\u1ea3 d\u01b0\u1ee3c hai l\u1ea7n m\u1ed9t ng\u00e0y trong 12 tu\u1ea7n. Sau k\u1ebft th\u00fac th\u1eed nghi\u1ec7m, nh\u1eefng b\u1ec7nh nh\u00e2n d\u00f9ng Joenja c\u00f3 d\u1ea5u hi\u1ec7u gi\u1ea3m k\u00edch th\u01b0\u1edbc h\u1ea1ch b\u1ea1ch huy\u1ebft v\u00e0 c\u1ea3i thi\u1ec7n 37% s\u1ed1 l\u01b0\u1ee3ng t\u1ebf b\u00e0o B<\/span><\/p>\n

\"Leniolisib\"
Leniolisib<\/figcaption><\/figure>\n

2.1 Nh\u00f3m d\u01b0\u1ee3c l\u00fd v\u00e0 d\u01b0\u1ee3c l\u1ef1c h\u1ecdc<\/span><\/h3>\n

Leniolisib l\u00e0 m\u1ed9t ch\u1ea5t \u1ee9c ch\u1ebf m\u1ea1nh v\u00e0 ch\u1ecdn l\u1ecdc phosphoinositide 3-kinase \u03b4 (PI3K\u03b4) \u0111\u01b0\u1ee3c ch\u1ec9 \u0111\u1ecbnh \u0111\u1ec3 \u0111i\u1ec1u tr\u1ecb h\u1ed9i ch\u1ee9ng phosphoinositide 3-kinase delta (PI3K\u03b4) ho\u1ea1t h\u00f3a (APDS) \u1edf b\u1ec7nh nh\u00e2n ng\u01b0\u1eddi l\u1edbn v\u00e0 tr\u1ebb em t\u1eeb 12 tu\u1ed5i tr\u1edf l\u00ean.\u00a0<\/span><\/p>\n

Leniolisib ho\u1ea1t \u0111\u1ed9ng \u0111\u1ec3 ng\u0103n ch\u1eb7n c\u00e1c \u0111\u01b0\u1eddng truy\u1ec1n t\u00edn hi\u1ec7u b\u1ea5t th\u01b0\u1eddng ph\u1ee5 thu\u1ed9c v\u00e0o PI3K\u03b4 c\u1ee7a c\u00e1c t\u1ebf b\u00e0o mi\u1ec5n d\u1ecbch, ch\u1eb3ng h\u1ea1n nh\u01b0 t\u1ebf b\u00e0o B v\u00e0 T, b\u1ea1ch c\u1ea7u trung t\u00ednh, b\u1ea1ch c\u1ea7u \u0111\u01a1n nh\u00e2n, basophils, t\u1ebf b\u00e0o \u0111u\u00f4i gai plasmacytoid v\u00e0 t\u1ebf b\u00e0o mast<\/span><\/p>\n

2.2 C\u01a1 ch\u1ebf ho\u1ea1t \u0111\u1ed9ng c\u1ee7a Leniolisib tr\u00ean APDS<\/span><\/h3>\n

Leniolisib \u1ee9c ch\u1ebf PI3K\u03b4<\/a> b\u1eb1ng c\u00e1ch ch\u1eb7n v\u1ecb tr\u00ed li\u00ean k\u1ebft ho\u1ea1t \u0111\u1ed9ng trong ti\u1ec3u \u0111\u01a1n v\u1ecb p110\u03b4. Trong c\u00e1c th\u1eed nghi\u1ec7m enzyme ph\u00e2n l\u1eadp kh\u00f4ng c\u00f3 t\u1ebf b\u00e0o, \u0111\u1ed9 ch\u1ecdn l\u1ecdc c\u1ee7a PI3K-delta cao h\u01a1n PI3K-alpha (28 l\u1ea7n), PI3K-beta (43 l\u1ea7n) v\u00e0 PI3K-gamma (257 l\u1ea7n). Leniolisib l\u00e0m gi\u1ea3m ho\u1ea1t \u0111\u1ed9ng c\u1ee7a con \u0111\u01b0\u1eddng pAKT v\u00e0 \u1ee9c ch\u1ebf s\u1ef1 t\u0103ng sinh c\u0169ng nh\u01b0 k\u00edch ho\u1ea1t c\u00e1c t\u1eadp h\u1ee3p con t\u1ebf b\u00e0o B v\u00e0 T v\u00e0 \u1ee9c ch\u1ebf c\u00e1c con \u0111\u01b0\u1eddng truy\u1ec1n t\u00edn hi\u1ec7u d\u1eabn \u0111\u1ebfn t\u0103ng s\u1ea3n xu\u1ea5t PIP3, t\u0103ng ho\u1ea1t \u0111\u1ed9ng c\u1ee7a con \u0111\u01b0\u1eddng mTOR\/AKT theo chi\u1ec1u thu\u1eadn<\/span><\/p>\n

2.3 D\u01b0\u1ee3c \u0111\u1ed9ng h\u1ecdc\u00a0<\/span><\/h3>\n

H\u1ea5p thu: N\u1ed3ng \u0111\u1ed9 thu\u1ed1c \u1edf tr\u1ea1ng th\u00e1i \u1ed5n \u0111\u1ecbnh c\u00f3 th\u1ec3 \u0111\u1ea1t \u0111\u01b0\u1ee3c sau kho\u1ea3ng 2-3 ng\u00e0y. T max l\u00e0 kho\u1ea3ng m\u1ed9t gi\u1edd<\/span><\/p>\n

Ph\u00e2n b\u1ed1: N\u1ed3ng \u0111\u1ed9 Leniolisib trong huy\u1ebft t\u01b0\u01a1ng t\u0103ng theo c\u1ea5p s\u1ed1 nh\u00e2n theo th\u1eddi gian. Th\u1ec3 t\u00edch ph\u00e2n b\u1ed1 (Vd) c\u1ee7a Leniolisib \u01b0\u1edbc t\u00ednh l\u00e0 28,5L. Leniolisib li\u00ean k\u1ebft 94,5% v\u1edbi protein huy\u1ebft t\u01b0\u01a1ng<\/span><\/p>\n

Chuy\u1ec3n h\u00f3a: Kho\u1ea3ng 60% Leniolisib \u0111\u01b0\u1ee3c chuy\u1ec3n h\u00f3a \u1edf gan v\u00e0 tr\u1ea3i qua qu\u00e1 tr\u00ecnh chuy\u1ec3n h\u00f3a oxy h\u00f3a, ch\u1ee7 y\u1ebfu qua trung gian CYP3A4 (94,5%), m\u1ed9t ph\u1ea7n nh\u1ecf qua c\u00e1c enzyme CYP3A5 (3,5%), CYP1A2 (0,7%) v\u00e0 CYP2D6 (0,7%). C\u00e1c con \u0111\u01b0\u1eddng chuy\u1ec3n h\u00f3a kh\u00e1c bao g\u1ed3m kh\u1eed alkyl, kh\u1eed metyl v\u00e0 hydroxyl h\u00f3a. Th\u1eddi gian b\u00e1n h\u1ee7y (T1\/2)\u00a0 hi\u1ec7u qu\u1ea3 l\u00e0 kho\u1ea3ng 7 gi\u1edd<\/span><\/p>\n

2.4 T\u00e1c d\u1ee5ng ph\u1ee5\u00a0<\/span><\/h3>\n

C\u00e1c t\u00e1c d\u1ee5ng ph\u1ee5 th\u01b0\u1eddng g\u1eb7p c\u1ee7a Leniolisib bao g\u1ed3m: \u0111au \u0111\u1ea7u, vi\u00eam xoang, vi\u00eam da d\u1ecb \u1ee9ng, ti\u00eau ch\u1ea3y, r\u1ee5ng t\u00f3c, \u0111au l\u01b0ng, \u0111au c\u1ed5<\/span><\/p>\n

Nhi\u1ec1u t\u00e1c d\u1ee5ng ph\u1ee5 nghi\u00eam tr\u1ecdng c\u1ee7a Leniolisib kh\u00e1c nh\u01b0: tim \u0111\u1eadp nhanh, s\u1ed1t, d\u1ea5u hi\u1ec7u c\u1ee7a ph\u1ea3n \u1ee9ng d\u1ecb \u1ee9ng (ph\u00e1t ban, n\u1ed5i m\u1ec1 \u0111ay, ng\u1ee9a, da \u0111\u1ecf, s\u01b0ng, ph\u1ed3ng r\u1ed9p, kh\u00f2 kh\u00e8, t\u1ee9c ng\u1ef1c, kh\u00e0n gi\u1ecdng, s\u01b0ng mi\u1ec7ng, kh\u00f3 nu\u1ed1t ho\u1eb7c n\u00f3i chuy\u1ec7n)<\/span><\/p>\n

2.5 Li\u1ec1u l\u01b0\u1ee3ng c\u1ee7a Leniolisib<\/span><\/h3>\n

Li\u1ec1u d\u00f9ng cho ng\u01b0\u1eddi l\u1edbn v\u00e0 tr\u1ebb em: vi\u00ean thu\u1ed1c 70mg<\/span><\/p>\n

\u0110i\u1ec1u tr\u1ecb h\u1ed9i ch\u1ee9ng phosphoinositide 3-kinase delta (APDS)<\/span><\/p>\n

    \n
  • Li\u1ec1u l\u01b0\u1ee3ng cho ng\u01b0\u1eddi l\u1edbn: (1) Tr\u00ean 45kg u\u1ed1ng 70mg m\u1ed7i 12 gi\u1edd (2) D\u01b0\u1edbi 45kg kh\u00f4ng c\u00f3 li\u1ec1u khuy\u1ebfn c\u00e1o<\/span><\/li>\n
  • Li\u1ec1u l\u01b0\u1ee3ng cho tr\u1ebb em: (1) 12 tu\u1ed5i tr\u1edf l\u00ean v\u00e0 c\u00e2n n\u1eb7ng t\u1eeb 45kg tr\u1edf l\u00ean u\u1ed1ng 70mg m\u1ed7i 12 gi\u1edd (2) D\u01b0\u1edbi 45kg kh\u00f4ng c\u00f3 li\u1ec1u khuy\u1ebfn c\u00e1o<\/span><\/li>\n<\/ul>\n

    3.K\u1ebft lu\u1eadn\u00a0<\/strong><\/h2>\n

    Leniolisib l\u00e0m gi\u1ea3m \u0111\u00e1ng k\u1ec3 k\u00edch th\u01b0\u1edbc h\u1ea1ch b\u1ea1ch huy\u1ebft v\u00e0 \u0111\u01b0\u1ee3c hy v\u1ecdng s\u1ebd gi\u00fap c\u1ea3i thi\u1ec7n t\u00ecnh tr\u1ea1ng b\u1ec7nh \u1edf nh\u1eefng ng\u01b0\u1eddi m\u1eafc APDS sau khi \u0111\u01b0\u1ee3c cung c\u1ea5p v\u00e0o gi\u1eefa th\u00e1ng 4 n\u0103m 2023<\/span><\/p>\n","protected":false},"excerpt":{"rendered":"

    Leniolisib l\u00e0 m\u1ed9t lo\u1ea1i thu\u1ed1c \u1ee9c ch\u1ebf phosphoinositide 3-kinase (PI3K) \u0111\u01b0\u1ee3c s\u1eed d\u1ee5ng trong \u0111i\u1ec1u tr\u1ecb APDS – m\u1ed9t lo\u1ea1i b\u1ec7nh mi\u1ec5n d\u1ecbch\u00a0 li\u00ean quan \u0111\u1ebfn t\u1ebf b\u00e0o T v\u00e0 B. APDS c\u00f3 th\u1ec3 g\u00e2y ra c\u00e1c tri\u1ec7u ch\u1ee9ng nh\u01b0 vi\u00eam xoang, vi\u00eam \u0111\u01b0\u1eddng h\u00f4 h\u1ea5p, nhi\u1ec5m tr\u00f9ng v\u00e0 t\u1ed5n th\u01b0\u01a1ng t\u1ebf b\u00e0o th\u1ea7n kinh. […]<\/p>\n","protected":false},"author":54,"featured_media":0,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":[],"categories":[12],"tags":[],"yoast_head":"\nLeniolisib: ph\u01b0\u01a1ng ph\u00e1p m\u1edbi trong \u0111i\u1ec1u tr\u1ecb APDS<\/title>\n<meta name=\"description\" content=\"Leniolisib l\u00e0 m\u1ed9t lo\u1ea1i thu\u1ed1c \u1ee9c ch\u1ebf phosphoinositide 3-kinase (PI3K) \u0111\u01b0\u1ee3c s\u1eed d\u1ee5ng trong \u0111i\u1ec1u tr\u1ecb APDS v\u00e0 \u0111\u01b0\u1ee3c FDA ch\u1ea5p thu\u1eadn v\u00ec t\u00ednh hi\u1ec7u qu\u1ea3\" \/>\n<meta name=\"robots\" content=\"noindex, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Leniolisib: ph\u01b0\u01a1ng ph\u00e1p m\u1edbi trong \u0111i\u1ec1u tr\u1ecb APDS\" \/>\n<meta property=\"og:description\" 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