Is intensive chemotherapy and allogeneic stem cell transplantation mandatory for curing Philadelphia chromosome-positive acute lymphoblastic leukemia in young patients in the era of multitarget agents?

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초록

IntroductionThe treatment outcomes for Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ALL) have improved with various tyrosine kinase inhibitors (TKIs) and bispecific T-cell engagers. Although allogeneic stem cell transplantation (allo-SCT) is the standard treatment for young patients with Ph+ALL, its role remains debatable in the era of TKIs and blinatumomab.Areas coveredThere are some issues regarding Ph+ALL. First, do young patients require intensive chemotherapy (IC) in the era of multitarget agents? Second, which TKI is preferred for frontline therapy? Third, should allo-SCT be performed in patients achieving complete remission with ponatinib and IC? Fourth, can chemo-free treatment lead to a cure without allo-SCT? We searched relevant literature from the last 30 years on PubMed; reviewed the role of chemo-free therapies and combinations of ponatinib and IC; and assessed the necessity of allo-SCT in young patients with Ph+ALL.Expert opinionAllo-SCT may not be needed, even in young patients with Ph+ALL treated with ponatinib-based IC or combined ponatinib and blinatumomab as frontline therapy. When adopting a ponatinib-based chemo-minimized regimen for induction, allo-SCT is needed with posttransplant ponatinib maintenance. Continuous exposure to ponatinib at pre- or post-transplant is regarded as one of the most important factor for the success of treatment.

키워드

acute lymphoblastic leukemia; allogeneic stem cell transplantation; intensive chemotherapy; Philadelphia chromosome; ponatinib; VON-WILLEBRAND-FACTOR; THROMBOTIC THROMBOCYTOPENIC PURPURA; FACTOR-CLEAVING PROTEASE; UPSHAW-SCHULMAN-SYNDROME; HEMOLYTIC-UREMIC SYNDROME; ADAMTS13 GENE-MUTATIONS; FACTOR-VIII; RECOMBINANT ADAMTS13; MOLECULAR CHARACTERIZATION; VONWILLEBRAND-FACTOR
제목
Is intensive chemotherapy and allogeneic stem cell transplantation mandatory for curing Philadelphia chromosome-positive acute lymphoblastic leukemia in young patients in the era of multitarget agents?
저자
Sohn, Sang Kyun; Lee, Jung Min; Jang, Youngeun; Lee, Yunji; Na, Jihyun; Cho, Hee Jeong; Moon, Joon Ho; Baek, Dong Won
DOI
10.1080/17474086.2024.2357273
발행일
2024-07
유형
Review
저널명
Expert Review of Hematology
권
17
호
7
페이지
353 ~ 359