Ua ʻae ʻia ʻo Ryoncil no ka mālama ʻana i ka maʻi steroid-refractory acute graft-versus-host maʻi (SR-aGVHD), kahi maʻi weliweli i ke ola e hiki ke hopena mai ka hoʻololi ʻana i ke ʻano o ke koko i hana ʻia e hoʻololi i nā pūnaewele hemahema o ka mea loaʻa i kekahi mau ʻano maʻi maʻi koko. , nā maʻi koko a i ʻole nā maʻi ʻōnaehana pale. Hoʻolilo kēia iā Ryoncil i ka lāʻau lapaʻau MSC (Mesenchymal Stem Cell) i ʻae ʻia e FDA. Loaʻa iā Ryoncil nā MSC i hoʻokaʻawale ʻia mai ka iwi iwi o nā mea hāʻawi kanaka makua olakino e hiki ke hoʻokaʻawale i nā ʻano ʻano pūnaewele ʻē aʻe. Hāʻawi ʻia nā pono o ke ola ʻana i nā infusions intravenous o nā mesenchymal stromal cell (MSCs).
Ma ka 18 Kekemapa 2024, FDA ʻae ʻia ʻo Ryoncil (remestemcel-L-rknd), he allogeneic (donor) iwi iwi i loaʻa i ka mesenchymal stromal cell (MSC) therapy no ka mālama ʻana i ka maʻi steroid-refractory acute graft-versus-host (SR-aGVHD) i nā maʻi pediatric 2 mahina o ka makahiki a oi. Hoʻolilo kēia iā Ryoncil i ka lāʻau lapaʻau MSC (Mesenchymal Stem Cell) mua i ʻae ʻia e FDA.
Loaʻa iā Ryoncil nā MSC i hoʻokaʻawale ʻia mai ka iwi iwi o nā mea hāʻawi kanaka makua olakino e hiki ke hoʻokaʻawale i nā ʻano ʻano pūnaewele ʻē aʻe.
ʻO ka Steroid-refractory acute graft-versus-host maʻi (SR-aGVHD) he maʻi koʻikoʻi a weliweli i ke ola e hiki ke loaʻa i nā hopena olakino koʻikoʻi, ākea ākea, me ka pōʻino i nā kino he nui, hoʻemi ʻia ka maikaʻi o ke ola a me ka pilikia o ka make i ka maʻi. nā maʻi. Hiki ke hana kēia ma ke ʻano he hoʻopiʻi o ka allogeneic hematopoietic (koko) stem cell transplantation (allo-HSCT).
Ma ka allo-HSCT, loaʻa i ka mea maʻi nā pūnaewele hematopoietic mai kahi mea hāʻawi olakino e pani i kā lākou mau pūnaewele ponoʻī a hana i nā ʻāpana koko hou, kahi kaʻina hana i hana pinepine ʻia ma ke ʻano o ka mālama ʻana i kekahi ʻano o nā maʻi maʻi koko, nā maʻi koko a i ʻole nā maʻi ʻōnaehana pale.
ʻO ka ʻae ʻana o Ryoncil ma hope o ka loiloi ʻana i kona palekana a me ka maikaʻi ma kahi noiʻi multicentre, hoʻokahi lima e pili ana i 54 mau keiki pediatric me SR-aGVHD i loaʻa i ka allo-HSCT. Ua loaʻa i ka poʻe i komo he ʻewalu intravenous infusions o Ryoncil i ʻelua pule i kēlā me kēia pule no ʻehā mau pule. Hoʻokumu ʻia ka loiloi o ka hopena ma ka helu a me ka lōʻihi o ka pane ʻana i ka mālama ʻana i nā lā 28 ma hope o ka hoʻomaka ʻana iā Ryoncil.
Ma kekahi ho'āʻo'ē aʻe no SR-aGVHD ma hope o ka allo-HSCT, uaʻikeʻiaʻo ka hui pūʻana o nā MSC a me ka basiliximab (kahi antibody monoclonal i hoʻohanaʻia no ka paleʻana i ka transplantation) ke alakaʻi i kahi pane piha maikaʻi loa (CR) ma mua o basiliximab wale nō.
Ua ʻike ʻia i loko o nā hoʻokolohua lapaʻau ʻo IV infusions o nā mesenchymal stromal cell (MSCs) hāʻawi i nā pono ola.
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E hoʻomaopopo ':
- Hoʻokuʻu ʻia ka Nūhou FDA - Ua ʻae ʻo FDA i ka Mesenchymal Stromal Cell Therapy e mālama i ka maʻi Steroid-refractory Acute Graft-versus-host. Hoʻopuka ʻia 18 Dekemaba 2024. Loaʻa ma https://www.fda.gov/news-events/press-announcements/fda-approves-first-mesenchymal-stromal-cell-therapy-treat-steroid-refractory-acute-graft-versus-host
- Fu, H., Lā, X., Lin, R. et al. ʻO nā pūnaewele stromal Mesenchymal a me ka basiliximab e hoʻomaikaʻi i ka pane o ka maʻi steroid-refractory acute graft-versus-host ma ke ʻano he lāʻau lapaʻau lua: he multicentre, randomized, controlled trial. BMC Med 22, 85 (2024). https://doi.org/10.1186/s12916-024-03275-5
- Kelly, K., Bloor, AJC, Griffin, JE et al. ʻElua mau hopena palekana o ka iPS cell-derived mesenchymal stromal cell i loko o ka maʻi maʻi graft-versus-host. Nat Med 30, 1556–1558 (2024). https://doi.org/10.1038/s41591-024-02990-z
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