RISE UP
Nwebata RISE UP ELU, Ọmụmụ ọgwụ ỌHỤRỤ iji chọpụta ọgbaghara nke ihemgbu na ụkọ ọbara nke na-adị n’ọrịa sickle cell.
RISE UP (2021‑001674‑34) bụ agba nke 2/3 nke nwale ọgwụ e zoro ndị e ji eme nwale ezo, nke a na-ahọrọ na ndapụta, nhazi ihenchekwa ndụ ndị a na-enye n’ọnọdụ ọgwụ e mere n’ebe ndị dị iche iche. 1
Ọmụmụ ihe ọgwụ a ga-enyocha arụmọrụ na nchekwa nke mitapivat na ọgwụgwọ ọrịa sickle cell maka ndị nsonye ruru afọ 16 nakwa ndị gafere ya. 1
Nnyocha na-egosi na ihe karịrị ọkara nke ndị ọrịa ga-esonye na nwale ahụike ma ọ bụrụ na ha na onye na-ahụ maka ahụike ha ekwuo maka ya. 2
IHENHAZI ỌMỤMỤ ỌGWỤ NKE RISE UP1
Nkebi nke 3 (na-edebanye aha ugbu a)
Hb = hemoglobin.
EBEMGBADOỤKWỤ KACHA MKPA A NA-AGBASO N’ITINYE ETINYE1
- Onye dị afọ 16 ma ọ bụ karịa, nke ji akwụkwọ ndetu nke nchoputa ọrịa sickle cell (HbSS, HbSC, HbS / β0 thalassemia, HbS / β + thalassemia, ma ọ bụ ntuwanye ọrịa sickle cell ndị ọzọ)
- O peka mpe 2, na ihe akarịghị nsogbu 10 ihemgbu nke ọrịa sickle cell n'ime ọnwa 12 gara aga tupu nkwenye ọmụma
- A kọwara dịka oké ihe omume nke ihemgbu, oké obi mgbu, ọrịa amu nkenyi na-egbu mgbu, ma ọ bụ mkpachi imeju ma ọ bụ nke spleen
- Ọ bụrụ na ị na-aṅụ ọgwụ hydroxyurea, ị ga-aṅụ mkpụrụọgwụ ọgwụ hydroxyurea ole ahụ ị na-aṅụ esepughi aka ihe ruru o pekata mpe mkpuru ụbọchị 90 tupu ị họrọwa ya na ndapụta
EBEMGBADOỤKWỤ KACHA MKPA NA MWEPU1
- I di ime ma ọ bụ na-enye nwa ara
- Ịnata mmịnye ọbara a na-eme mgbe niile
- Ọrịa Hepatobiliary, ọrịa imeju dị ịrịba ama, ọrịa gallbladder, ma ọ bụ ọrịa akụrụ siri ike
- Mkpughe bu ụzọ tupu ọgwụgwọ ma ọ bụ mwụgharị ụmụọkpụkpụ ma ọ bụ mwụgharị seelụ site n’otu ahụ gaa na nke ọzọ
- Na-enweta ọgwụ voxelotor, crizanlizumab, ma ọ bụ L-glutamine ugbua
- Ji ihe ndi na-akpali okpukpu ọbara enweta ọgwụgwọ ugbua
- Na-aṅụ ọgwụ CYP3A4/5 siri ike ma ọ bụ ndi mgbochi siri ike nke CYP3A4
MWAKE MAỌBỤ MKPALIỌRỤ PK NA-AKWADO AHỤIKE ỌBARA UHIE
Mitapivat — ọgwụ ọmụmụ ihe dị na RISE UP— bụ ihe mwake nji-eme nchọpụta, a na-eri n’ọnụ ma nwee ike igbanwe mmiri ọgwụ PK 1,3
Ịkpali ọrụ enzaịm PK nwere ike imelite ahụike, ike aru, na ndụ nke mkpụrụ ndụ ọbara uhie (RBC) maka ndị ọrịa nwere ọrịa mgbali elu nke ụkọ ọbara1
- Ịbawanye mmepụta ATP, na-enye aka iji dakọọ na mkpa ike RBC
- Mbelata 2,3-dpg nke na-eme ka ikuku ume ndu dịkwuo elu maka hemoglobin, nwere ike ibelata ọrịa
- Ime ka antioxidants dịgide etu ọ dị, si etú ahụ belata mmebi cell
JIKERE MAKA ÀGBA RISE UP NKE ỌZỌ NA-ESOTE
Mụtakwuo ihe banyere RISE UP, ihe ndị gụnyere nkọwa ndebanye aha zuru oke, ebumnuche ọmụmụihe niile, na ihemgbadoụkwụ zuru oke maka ntinye na mwepu. Gwazia ndị ọrịa gị ruru eru banyere ọmụmụ ihe ahụ.
Ọ bụrụ na ị nwere ajụjụ, zipu ozi-ịntanetị:
medinfo@agios.com (US)
ex-usmedinfo@agios.com (na mpụga US)
Nrụtụaka gasi:
1. Data di na faịlụ. Agios Pharmaceuticals, Inc.
2. Nnyocha!America. Ntuli aka echiche ọha na eze gburu-gburu. Julaị 2017. Banyere n’Ọktoba 18, 2023. https://www.researchamerica.org/wp-content/uploads/2022/07/July2017ClinTrialMinorityOversamplesPressReleaseSlidesFINAL_0-1.pdf
3. Howard J, Kuo KHM, Oluyadi A, et al. Agba nke 2/3, nke nwale ọgwụ e zoro ndị e ji eme nwale ezo, nhazi ihe ndị a na-enye n’ọnọdụ ọgwụ nke ọmụmụ ọgwụ mitapivat maka ọrịa sickle cell. Ọbara. 2021; 138(suppl 1): 3109.
