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Haematologica Conference: 46th congress of the italian society of hematology Italy 2017; 102(Supplement 3): 89. Available from:
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18. Cortes JE, Kim DG, Pinilla-Ibarz J, et al. Efficacy and safety of ponatinib in heavily pre-treated leukemia patients in the PACE trial: 3-year results. Journal
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20. Cortes JE, Pinilla-Ibarz J, Coutre PD, et al. Long-term efficacy and safety of ponatinib in heavily pretreated leukemia patients: 4-year results from the pivotal phase 2 pace trial. Haematologica Conference: 21st congress of the european hematology association Denmark 2016; 101: 60-61. Available from:
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21. Jabbour EJ, Cortes JE, Talpaz M, et al. Long-term follow-up of the efficacy and safety of ponatinib in philadelphia chromosome-positive leukemia patients with the T315I mutation. Blood Conference: 58th annual meeting of the
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22. Cortes JE, Kantarjian HM, Pinilla-Ibarz J, et al. 5-YR results from the pivotal phase 2 ponatinib pace trial: efficacy, safety and landmark analysis in heavily pretreated patients (PTS) with chronic-phase chronic myeloid leukemia (CP-CML). Haematologica Conference: 22th congress of the european hematology association Spain 2017; 102: 236-237. Available from:
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23. Nct. Ponatinib in Participants With Resistant Chronic Phase Chronic Myeloid Leukemia (CML) to Characterize the Efficacy and Safety of a Range of Doses.
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25. Sanford D, Kantarjian H, Skinner J, Jabbour E, Cortes J. Phase II trial of ponatinib in patients with chronic myeloid leukemia resistant to one previous tyrosine kinase inhibitor. Haematologica 2015; 100(12): e494-495.
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27. Deininger MW, Hodgson JG, Shah NP, et al. Compound mutations in BCR-ABL1 are not major drivers of primary or secondary resistance to ponatinib in CP-CML patients. Blood 2016; 127(6): 703-712.
28. Parker WT, Yeung DT, Yeoman AL, et al. The impact of multiple low-level BCR-ABL1 mutations on response to ponatinib. Blood 2016; 127(15):
1870-1880.
29. Tojo A, Kyo T, Yamamoto K, et al. Ponatinib in Japanese patients with Philadelphia chromosome-positive leukemia, a phase 1/2 study. International journal of hematology 2017; 106(3): 385-397.
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https://clinicaltrials.gov/ct2/show/NCT01207440?term=NCT01207440&rank=
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附錄
附錄一 imatinib（如 Glivec®）、dasatinib（如 Sprycel®）、nilotinib（如 Tasigna®） 及 clofarabine（如 Evoltra®）之藥品給付規定（僅節錄「慢性骨髓性白血病」及
「費城染色體陽性急性淋巴性白血病」相關內容）[8]
（一） Imatinib（如 Glivec®）： 限用於
1. 治療正值急性轉化期（blast crisis）、加速期或經 ALPHA-干擾素治療無效之 慢性期的慢性骨髓性白血病（CML）患者使用。
2. 用於治療初期診斷為慢性骨髓性白血病（CML）的病人。
3. 治療初診斷為費城染色體陽性急性淋巴性白血病（Ph+ ALL）且併用化療之 成年人。
4. 未曾使用 imatinib 治療之成人復發性或難治性費城染色體陽性急性淋巴性 白血病（Ph+ ALL）之單一療法。
（二） Dasatinib （如 Sprycel®）： 限用於
1. 第一線使用：治療新診斷的費城染色體陽性之慢性期慢性骨髓性白血病的成 人。
2. 第二線使用：
（1） 治療患有慢性、加速或急性期慢性骨髓性白血病，對先前經 imatinib 400mg（含）以上治療後有抗藥性或無耐受性的成人。
（2） 治療患有費城染色體陽性急性淋巴性白血病，且對先前經 imatinib 400mg
（含）以上治療後有抗藥性或無耐受性的成人。
（3） 需檢送病歷及對 imatinib 耐受性不良或無效的證明。
（三） Nilotinib：
1. Nilotinib 200mg（如 Tasigna® 200mg）
（1） 限用於治療對 imatinib 400MG（含）以上耐受性不良或治療無效的「慢 性期或加速期費城染色體（Philadelphia chromosome） 陽性的慢性骨髓 性白血病（CML）成年患者」。
（2） 必須檢附耐受性不良或治療無效的證明。
（3） Nilotinib 與 dasatinib 不得合併使用。
2. Nilotinib 150mg（如 Tasigna® 150mg）：限用於新確診之慢性期費城染色體陽 性的慢性骨髓性白血病。
（四） Clofarabine（如 Evoltra®）：
1. 使用於先前接受至少兩種化療療程（如 TPOG 之療程表）治療無效或已復發 第二次或以上之急性淋巴母細胞白血病（acute lymphoblastic leukemia）病，
且計畫進行造血幹細胞移植的病人（限 21 歲以下），每位病人限給付一療程。
2. 須事前審查核准後使用，申請時須檢附完整之造血幹細胞移植計畫，並詳細 記載確認捐贈者名單、確認移植之執行醫院及移植前調適治療等資料。
附錄二 療效評估文獻搜尋記錄
資料庫 查詢日期 關鍵字 篇數
Cochrane Library
107/6/14 #1 ponatinib 66 PubMed 107/6/14 #1 "ponatinib"[Supplementary Concept] OR
"ponatinib"[All Fields]
465
#2 #1 AND (Meta-Analysis[ptyp] OR systematic[sb])
17
#3 #1 AND (Randomized Controlled Trial[ptyp] OR Controlled Clinical Trial[ptyp] OR Clinical Trial, Phase
IV[ptyp] OR Clinical Trial, Phase III[ptyp]
OR Clinical Trial, Phase II[ptyp])
12
Embase 107/6/14 #1 'ponatinib'/exp OR ponatinib 2,022
#2 'chronic myeloid
leukemia'/exp/dm_dr,dm_dt
15,611
#3 'acute lymphoblastic
leukemia'/exp/dm_dr,dm_dt
16,360
#4 #2 OR #3 29,926
#5 #1 AND #4 694
#6 #5 AND ([systematic review]/lim OR [meta analysis]/lim)
21
#7 #5 AND ('phase 2 clinical trial'/de OR 'phase 2 clinical trial (topic)'/de OR 'phase 3
clinical trial (topic)'/de OR 'randomized controlled trial (topic)'/de)
159
附錄三 經濟評估文獻搜尋記錄
資料庫 查詢日期 關鍵字 篇數 篩選後篇數
PubMed 2018.06.05 #1 ponatinib
#2 (chronic myeloid leukemia) OR acute lymphocytic leukemia
#3 (((cost-effectiveness analysis) OR cost-utility analysis) OR cost-benefit analysis) OR cost-minimization analysis
#1 AND #2 AND #3
4 1
EMBASE 2018.06.05 #1 ponatinib
#2 (chronic myeloid leukemia) OR acute lymphoblastic leukemia
#3 (((cost-effectiveness analysis) OR cost-utility analysis) OR cost-benefit analysis) OR cost-minimization analysis
#1 AND #2 AND #3
21 7
Cochrane Library
2018.06.05 #1 ponatinib
#2 (chronic myeloid leukemia) OR acute lymphocytic leukemia
#3 (((cost-effectiveness analysis) OR cost-utility analysis) OR cost-benefit analysis) OR cost-minimization analysis
#1 AND #2 AND #3
0 0
CRD 2018.06.05 #1 ponatinib
#2 (chronic myeloid leukemia) OR acute lymphocytic leukemia
#3 (((cost-effectiveness analysis) OR cost-utility analysis) OR cost-benefit analysis) OR cost-minimization analysis
#1 AND #2 AND #3
0 0