Olsson-Strömberg Ulla, Simonsson Bengt, Ahlgren Tomas, Björkholm Magnus, Carlsson Karin, Gahrton Gösta, Hast Robert, Löfvenberg Eva, Linder Olle, Ljungman Per, Malm Claes, Paul Christer, Rödjer Stig, Turesson Ingemar, Udén Ann-Mari, Wahlin Anders, Killander Andreas, Wadman Bengt, Westin Jan, Vikrot Olle, Zettervall Olle, Oberg Gunnar
Department of Medicine, University Hospital, Uppsala, Sweden.
Hematol J. 2004;5(6):462-6. doi: 10.1038/sj.thj.6200552.
Whether busulphan-treated patients develop blastic transformation earlier than hydroxyurea treated has been a controversial issue. In a randomised prospective study, we examined the busulphan versus hydroxyurea influence on time to blast crisis and on survival. When we opened our study in 1984, the clinical benefit of allogeneic bone marrow transplantation (BMT) was not well known; to follow up the long-time outcome of this treatment was therefore of great interest.
Previously untreated CML patients were randomly started on either hydroxyurea (30 mg/kg/day) or busulphan (0.1 mg/kg/day). The end points of the study were overall survival and time to blast crisis. A total of 26 patients subsequently underwent BMT.
A total of 179 patients were randomised, 90 of hydroxyurea, and 89 to busulphan treatment. There was no significant difference in survival between hydroxyurea- and busulphan-treated patients (P = 0.46); median survival was 3.5 and 3.2 years, respectively. In all, 85 of the patients were subsequently diagnosed with blast crisis, 41 in the busulphan and 44 in the hydroxyurea group. There was no significant difference between the two groups (P = 0.91). The 26 patients who were allotransplanted survived significantly longer than those who were not transplanted (P = 0.0001). The 5-year-survival rates were 50 and 22% and the 10-year-survival rates were 46 and 2%, respectively. The median survival was 4.7 years for the transplanted and 3.3 years for the nontransplanted patients.
We did not find any difference between hydroxyurea and busulphan treatment, either in overall survival or in blast crisis-free survival; transplanted patients survived significantly longer than nontransplanted patients.
白消安治疗的患者是否比羟基脲治疗的患者更早发生急变一直是个有争议的问题。在一项随机前瞻性研究中，我们研究了白消安与羟基脲对急变时间和生存的影响。1984年我们开展这项研究时，异基因骨髓移植（BMT）的临床益处尚不为人所知；因此，随访这种治疗的长期结果非常有意义。
既往未接受治疗的慢性粒细胞白血病患者被随机开始接受羟基脲（30mg/kg/天）或白消安（0.1mg/kg/天）治疗。研究的终点是总生存期和急变时间。共有26例患者随后接受了BMT。
共有179例患者被随机分组，90例接受羟基脲治疗，89例接受白消安治疗。羟基脲治疗组和白消安治疗组患者的生存率无显著差异（P = 0.46）；中位生存期分别为3.5年和3.2年。共有85例患者随后被诊断为急变，白消安组41例，羟基脲组44例。两组之间无显著差异（P = 0.91）。接受同种异体移植的26例患者的生存期明显长于未接受移植的患者（P = 0.0001）。5年生存率分别为50%和22%，10年生存率分别为46%和2%。移植患者的中位生存期为4.7年，未移植患者为3.3年。
我们未发现羟基脲和白消安治疗在总生存期或无急变生存期方面存在任何差异；移植患者的生存期明显长于未移植患者。
