Ua loaʻa iā Qfitlia (Fitusiran), kahi lāʻau lapaʻau e pili ana i ka siRNA no ka hemophilia. ʻO ia he RNA liʻiliʻi liʻiliʻi (siRNA) ma muli o ka therapeutic hoʻopilikia i nā anticoagulants kūlohelohe e like me ka antithrombin (AT) a me ka tissue factor pathway inhibitor (TFPI). Hoʻopaʻa ʻo ia i ka AT mRNA i loko o ke ake a hoʻopaʻa i ka unuhi AT ma laila e hōʻemi ai i ka antithrombin a hoʻomaikaʻi i ka hana thrombin. Hāʻawi ʻia ia ma ke ʻano he subcutaneous injection e hoʻomaka ana i hoʻokahi manawa i kēlā me kēia ʻelua mahina. Hoʻoponopono ʻia ka nui a me ka pinepine o nā injections me ka hoʻohana ʻana i ka diagnostics hoa Antithrombin INNOVANCE e hōʻoia i ka hana antithrombin i ka pae i hoʻopaʻa ʻia. ʻAʻole ʻae ʻia ka dosis paʻa. He mea koʻikoʻi ka lāʻau lapaʻau hou no nā maʻi no ka mea ʻoi aku ka liʻiliʻi o ka lawelawe ʻana ma mua o nā koho ʻē aʻe.
Ua ʻae ʻia ʻo Qfitlia (fitusiran) ma ʻAmelika Hui Pū ʻIa (ma ka 28 Malaki 2025) no ka prophylaxis maʻamau e pale a hōʻemi paha i ka pinepine o ke kahe koko i nā pākeke a me nā keiki he 12 mau makahiki a ʻoi aku me ka hemophilia A a i ʻole hemophilia B, me ka ʻole o ka factor VIII a i ʻole IX inhibitors (neutralizing antibodies). He mea koʻikoʻi ka lāʻau lapaʻau hou no ka mea ʻoi aku ka liʻiliʻi o ka lawelawe ʻana (e hoʻomaka ana i hoʻokahi manawa i kēlā me kēia ʻelua mahina) ma mua o nā koho ʻē aʻe.
Hoʻokumu ʻia nā maʻi koko i ka hemophilia ma muli o ka lawa ʻole o nā kumu clotting. Hoʻokumu ʻia ka maʻi hemophilia A ma muli o ka hemahema o ke kumu clotting VIII (FVIII), aʻo ka hemophilia B ma muli o nā haʻahaʻa haʻahaʻa o ka helu IX (FIX). ʻO ka nele o ka mea hana XI ke kuleana no ka hemophilia C. Hoʻomaʻamaʻa ʻia kēia mau maʻi ma o ka hoʻokomo ʻana i ka mea hoʻohui i hoʻomākaukau ʻia ma ke kālepa a i ʻole kahi huahana kumu ʻole ma ke ʻano he hana pani o ka mea i nalowale.
ʻO Octocog alfa (Advate), ʻo ia ka 'genetically engineered me ka hoʻohana ʻana i ka ʻenehana DNA' version of clotting factor VIII, i hoʻohana mau ʻia no ka pale a me ka mālama ʻana i ka hemophilia A. No ka hemophilia B, nonacog alfa (BeneFix), ka mea. he mana hoʻohana ʻia o ka clotting factor IX i hoʻohana mau ʻia.
Ua ʻae ʻia ʻo Hympavzi (marstacimab-hncq) ma ʻAmelika Hui Pū ʻIa (ma 11 ʻOkakopa 2024) a ma EU (ma 19 Kepakemapa 2024) ma ke ʻano he lāʻau lapaʻau hou no ka pale ʻana i nā ʻāpana koko i nā poʻe me ka hemophilia A a i ʻole hemophilia B. Hoʻemi i kāna hana anticoagulation a laila hoʻonui i ka nui o ka thrombin. ʻO kēia ka lāʻau lapaʻau mua, kumu ʻole a hoʻokahi pule no ka hemophilia B.
ʻO kekahi antibody monoclonal, ʻo Concizumab (Alhemo) i ʻae ʻia ma USA (ma 20 Dekemaba 2024) a ma EU (ma 16 Dekemaba 2024) no ka pale ʻana i nā ʻāpana koko i nā mea maʻi me ka hemophilia A me nā mea paʻa VIII a i ʻole hemophilia B me nā mea paʻa IX. ʻO kekahi poʻe maʻi hemophilia ma "nā lāʻau clotting factor" no ka mālama ʻana i ko lākou maʻi maʻi koko e hoʻomohala i nā antibodies (kūʻē i nā lāʻau clotting factor). ʻO nā antibodies i hoʻokumu ʻia ke kāohi i ka hana o nā "lāʻau clotting factor" i emi iho ka hopena. ʻO ka Concizumab (Alhemo), i lawelawe ʻia i kēlā me kēia lā ma ke ʻano he subcutaneous injection, i manaʻo ʻia e mālama i kēia maʻi i mālama ʻia ma ke ʻano maʻamau e ka hoʻoulu ʻana i ka hoʻomanawanui ʻole ma o nā injections i kēlā me kēia lā o nā kumu clotting.
ʻOiai ʻo mpavzi (marstacimab-hncq) a me Concizumab (Alhemo) he monoclonal antibodies, ʻo ka lāʻau hou Qfitlia (fitusiran) he mea liʻiliʻi liʻiliʻi liʻiliʻi RNA (siRNA) i hoʻokumu ʻia i ka therapeutic e hoʻopilikia i nā anticoagulants kūlohelohe e like me antithrombin (AT) a me ka tissue factor pathway inhibitor (TFPI). Hoʻopili ia i ka AT mRNA i loko o ke ake a paʻa i ka unuhi AT no laila e hoʻemi i ka antithrombin a hoʻomaikaʻi i ka hana thrombin.
Hoʻohana ʻia ʻo Qfitlia (fitusiran) ma ke ʻano he subcutaneous injection e hoʻomaka ana i hoʻokahi manawa i kēlā me kēia ʻelua mahina.
Hoʻoponopono ʻia ka nui a me ka pinepine o nā injections me ka hoʻohana ʻana i ka diagnostics hoa Antithrombin INNOVANCE e hōʻoia i ka hana antithrombin i ka pae i hoʻopaʻa ʻia. ʻAʻole ʻae ʻia ka dosis paʻa. Eia naʻe, he mea koʻikoʻi ka lāʻau lapaʻau hou no nā maʻi no ka mea ʻoi aku ka liʻiliʻi o ka lawelawe ʻana ma mua o nā koho ʻē aʻe.
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E hoʻomaopopo ':
- Hoʻokuʻu ʻia ka Nūhou FDA - Ua ʻae ʻo FDA i ka mālama ʻana i ka Novel no ka Hemophilia A a i ʻole B, me ka ʻole o ka Factor Inhibitors. Hoʻopuka ʻia 28 Malaki 2025. Loaʻa ma https://www.fda.gov/news-events/press-announcements/fda-approves-novel-treatment-hemophilia-or-b-or-without-factor-inhibitors
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Pākuʻi pili:
- ʻO Concizumab (Alhemo) no ka Hemophilia A a i ʻole B me nā mea pale (29 Dekemaba 2024)
- Hympavzi (marstacimab): Lapaʻau Hou no ka Hemophilia (12 ʻOkakopa 2024)
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, a novel siRNA-based treatment for haemophilia has received FDA approval. It is a small interfering RNA (siRNA) based therapeutic that interfere with natural anticoagulants such as antithrombin (AT) and tissue factor pathway inhibitor (TFPI). It binds to AT mRNA in the liver and blocks AT translation thereby reduce antithrombin and improve thrombin generation. It is administered as subcutaneous injection starting once every two months. The dose and frequency of injections are adjusted using the INNOVANCE Antithrombin companion diagnostic that ensure antithrombin activity in the target range. The fixed dose is not approved. The new treatment is significant for patients because it is administered less frequently than other existing options.){kind=link}