研究筆記_藥華藥_20171120_P1101 vs.Jakafi
P1101
v.s Jakafi
l Jakafi=Ruxolitinib，是一種JAK1/JAK2 inhibitor
l Jakafi有做2個trial: RESPONSE (主要的) and RELIEF (後來追加的)。
l RESPONSE Trial並不是Jakafi vs. HU的一線用藥PK，而是針對HU不耐受/抗藥的病人，進行jakafi
vs. BAT的二線用藥PK。Incyte是先於2010年瞄準Jakafi二線用藥進行RESPONSE Trial，後來才於2012年同步增加Jakafi vs. HU的Relief試驗(後來沒達到試驗目標)。(From小林大)
RESPONSE (成功!)
1.
Target對HU不耐的二線病患
(who are
resistant to or intolerant of hydroxyurea)。
2.
有達到primary
endpoint: 達成phlebotomy independence+減少脾臟體積達35%或以上。(The primary endpoint of the trial is
the proportion of patients whose hematocrit level is controlled in absence of phlebotomy
and whose spleen volume is reduced by 35 percent or more from baseline as assessed
by imaging at 32 weeks.)
3.
安全性部份: In addition to safety, key secondary endpoints include
durable response and complete hematological remission.
RELIEF (失敗!)
1.
Target 有在服用HU的一線病患(沒抗藥性，但有副作用):
Who were generally well-controlled on hydroxyurea but
reporting continued disease-related symptoms.
(與HU PK副作用)
2.
有觀察到Jakafi有比HU好的趨勢，但可惜沒達到primary endpoint : 也就是要有一定比例的病患在醒著的時候出現指定副作用的緩解(疲倦、癢、肌肉疼痛、盜汗、流汗)，且緩解率要達到50%以上 (Proportion of Patients With a ≥50% Improvement in TSS-C and Individual
Symptoms)à採用Jakafi的有43.4%，採用HU的有29.6%的病患達成此一目標，但P值為0.139，沒有達到統計上的顯著，哭哭。(等等，HU自己就可以緩解??)
3.
安全性方面: 採用Jakafi的Adverse event並沒有比HU來的少 (各有所短)。