循环淋巴细胞比例在初发急性早幼粒细胞白血病死亡中的意义

目的:探索循环淋巴细胞比例(LYR)在初发急性早幼粒细胞白血病(APL)早期死亡中的意义。方法:收集2017年1月—2022年1月在血液科初诊、初治的APL患者111例，收集这些患者治疗前的临床数据，包括性别、年龄、血红蛋白(Hb)、白细胞计数(WBC)、血小板计数(PLT)、LYR、循环单核细胞比例(MOR)、骨髓及外周血早幼粒细胞比率、凝血酶原时间(PT)、活化部分凝血活酶时间(APTT)、纤维蛋白原(FIB)、D-二聚体(D-D)。根据是否发生早期死亡分为早期死亡组和非早期死亡组，分析早期死亡的相关因素，对单因素有意义的指标行受试者工作特征(ROC)曲线分析，以LYR及MOR的截断值为界，分为高LYR组和低LYR组；高MOR组和低MOR组。纳入单因素有意义的指标行多因素分析明确早期死亡的独立危险因素。结果:111例初发APL患者中18例发生早期死亡，简单关联性分析中与无早期死亡组比较，早期死亡组外周血早幼粒细胞比率、骨髓早幼粒细胞比率、WBC、MOR较高，LYR较低，PT延长。ROC分析显示预测效能较好的指标有PT、LYR、MOR的AUC分别为0.751、0.736、0.704。...     

高白细胞急性髓系白血病早期死亡临床分析

目的:探讨高白细胞急性髓系白血病(HAML)早期死亡的原因,分析其潜在危险因素。方法:回顾性分析67例初诊HAML患者(非M3型)在确诊后7d内早期死亡的病例(15例)和非早期死亡病例(52例)的临床特征。结果:15例(22.4%)早期死亡,原因分别为颅内出血(10例)、心力衰竭(2例)、肺栓塞(1例)、重度肺部感染(1例)及呼吸衰竭(1例)。早期死亡组白细胞淤滞分级评分(LGS)≥2分、外周血白细胞数、乳酸脱氢酶(LDH)及凝血功能异常发生率均高于非早期死亡组(P0.05)。2组之间性别、年龄、初诊时外周血血红蛋白水平、血小板水平、骨髓原/幼细胞比例、法美英(FAB)分型、尿酸、肌酐的差异不明显(P0.05)。Logistic回归分析表明,LDH为HAML患者早期死亡的危险因素。结论:白细胞淤滞、出血、脏器衰竭和感染是HAML早期死亡的主要原因。血LDH水平是HAML患者是否发生早期死亡的危险因素。     

黏附分子CD56、CD44、CD54、CD11a在急性髓系白血病骨髓中的表达及意义

目的:探讨黏附分子CD56、CD44、CD54、CD11a在急性髓系白血病(AML)骨髓中的表达情况及临床意义。方法:流式细胞仪检测57例AML患者和20例正常对照者骨髓单个核细胞表面CD56、CD44、CD54、CD11a的表达水平,分析其与AML患者白细胞计数、白细胞淤滞及早期病死率的关系。结果:1CD56仅在AML组表达(AML组表达率为35.1%,正常对照组0,P0.05),其在AML高白细胞(HAML)组的表达率(53.3%)高于AML非高白细胞(NHAML)组(28.6%),白细胞淤滞组(62.5%)高于无白细胞淤滞组(28.9%)(P0.05);2AML患者骨髓单个核细胞表面CD44的平均荧光强度在HAML组显著高于NHAML组(P0.05),其表达与AML患者外周血白细胞计数呈正相关(r=0.446,P0.01)。CD56、CD54、CD11a在HAML组与NHAML组、无白细胞淤滞组与白细胞淤滞组的平均荧光强度均差异无统计学意义(P0.05)。3AML患者中HAML组、白细胞淤滞组患者的早期病死率(33.3%、25.0%)分别明显高于NHAML组、无白细胞淤滞组(0、0)。结论:CD56在HAML患者和白细胞淤滞患者有更高的表达率,有可能是AML患者预后不良的因素之一;CD44在AML患者骨髓中的表达水平与外周血白细胞计数呈正相关,可能参与HAML的发生。CD54、CD11a与HAML及白细胞淤滞的关系不十分密切。     

初发APL患者接受ATRA+ATO±化疗早期死亡危险因素分析

目的:探讨初发急性早幼粒细胞白血病(APL)患者接受ATRA+ATO±化疗早期死亡的危险因素。方法:本研究回顾性分析417例初治成人APL患者,比较早期死亡与非早期死亡的患者在临床表现、白细胞计数、血小板计数、凝血指标、骨髓细胞形态学、免疫学等检查方面是否存在差异。结果:417例患者中早期死亡36例,早期死亡率为8.6%。低、中、高危患者相应组别早期死亡率分别为1.9%,8.1%,16.2%(P=0.001)。出血引起的死亡21例,心脏功能衰竭和严重肺部感染各5例,维甲酸综合征3例,脑梗死1例,还有1例原因不明。多因素分析提示,患者起病时的美国东部肿瘤协作组(ECOG)评分、危险分层、出血症状和纤维蛋白原水平是早期死亡的独立危险因素(P值分别为P0.001,P=0.007,0.031和0.022)。结论:出血,尤其是颅内出血是APL早期死亡最为常见的原因。患者起病时的体能状态、危险分层、出血症状和纤维蛋白原水平是APL早期死亡的独立危险因素。     

原发高白细胞型急性早幼粒细胞白血病66例临床特征及治疗研究

目的探讨原发高白细胞型急性早幼粒细胞白血病(APL)的临床特点及有效的治疗方法。方法回顾性分析1993年10月至2006年8月苏州大学附属第一医院收治的66例原发高白细胞型APL患者和152例非高白细胞型APL患者的临床资料,并对高白细胞型患者按治疗方案的不同进行了分组比较。结果高白细胞组APL患者早期病死率、弥散性血管内凝血(DIC)和维甲酸综合征(RAS)发生率分别为30.3%,57.6%和31.8%,均高于非高白细胞组(7.2%,38.1%和21.1%)(P<0.05),而完全缓解(CR)率较低(63.6%对88.2%)(P<0.05)。高白细胞组患者中61例接受了诱导治疗,其中31例单用维甲酸治疗,21例维甲酸联合亚砷酸治疗,9例单用亚砷酸治疗,各组早期病死率分别为27.3%,14.3%和55.6%,CR率分别为67.7%,81.0%和44.4%。61例患者中41例在接受诱导分化治疗的同时加用化疗,其CR率为80.5%,总病死率为19.5%;而未加用化疗的20例患者其CR率为45.0%,总病死率为55.0%,两组比较差异有显著性意义(P<0.05)。结论原发高白细胞型APL较非高白细胞型APLCR率低,早期病死率高,DIC、RAS发生率高。维甲酸加亚砷酸双诱导并联合小剂量化疗是治疗高白细胞型APL的最有效的方案,可明显减少早期病死率,提高CR率。     

CD56抗原表达在急性早幼粒细胞白血病中的临床意义和预后分析

目的:分析CD56抗原在初诊急性早幼粒细胞白血病(acute promyelocytic leukemia, APL)中的表达及其与临床参数之间的相关性,探究该抗原的临床意义及对预后的影响。方法:回顾性分析169例初诊APL患者的临床数据,按照CD56抗原表达状况,将其分为CD56阳性和CD56阴性组,比较2组患者性别、年龄、体能状态(ECOG)、血细胞计数、生化指标、凝血功能、骨髓及外周血早幼粒细胞比例、免疫分型、危险分层、FLT3突变、染色体核型、PML/RAR α异聚体类型及融合率、FAB分型(M3a/M3b/M3v)、弥散性血管内凝血(disseminated intravascular coagulation, DIC)积分等参数有无差异。以达分子学缓解(molecular complete remission, mCR)的时间为界,将临床事件分为早期事件(分化综合征、QT间期延长、出血、栓塞、死亡)和晚期事件(继发第二肿瘤、QT间期延长、出血、疾病复发、死亡),比较2组早晚期事件发生率的差异。探索CD56阳性表达对总生存期(OS)、无进展生存期(PFS)及无事件生存期(EF...     

高白细胞急性髓系白血病早期死亡原因分析

目的：探讨高白细胞急性髓系白血病（HAML）早期死亡的原因，分析其潜在危险因素。方法：回顾性分析67例初诊HAML患者（非M3型）在确诊后7d内早期死亡的病例（15例）和非早期死亡病例（52例）的临床特征。结果：15例（22.4%）早期死亡，原因分别为颅内出血（10例）、心力衰竭（2例）、肺栓塞（1例）、重度肺部感染（1例）及呼吸衰竭（1例）。早期死亡组白细胞淤滞分级评分（LGS）≥2、外周血白细胞数、乳酸脱氢酶（LDH）及凝血功能异常发生率均高于非早期死亡组（P<0.05）。两组之间性别、年龄、初诊时外周血血红蛋白水平、血小板水平、骨髓原/幼细胞比例、FAB分型、尿酸、肌酐的差异不明显（P>0.05）。Logistic回归分析表明，LDH为HAML患者早期死亡的危险因素。结论：出血、白细胞淤滞、脏器衰竭和感染是HAML早期死亡的主要原因。LDH水平是HAML患者是否发生早期死亡的危险因素。     

急性早幼粒细胞白血病CD56表达的临床意义

全反式维甲酸 ( ATRA)和三氧化二砷 ( As2 O3 )的应用 ,使急性早幼粒细胞白血病 ( APL )的治疗发生了根本性改变。尽管如此 ,仍有一部分患者死于治疗前的出血、治疗无效和早期复发 〔1〕。研究分析表明 ,不良的预后与外周血白细胞过高、血小板过低和存在 S型 PML- RARa融合基因有关〔2〕。近来的研究表明 ,CD56的表达与 APL预后不良亦有直接关系〔3〕。国内目前尚未见 CD56+ APL的报道。我院 1 995～ 1 999年收治 CD56+ APL患者 6例 ,现将其血液及临床特征分析如下。1　材料与方法1 .1 　 研究对象本组 46例 ,经 MIC分型符合 …     

急性早幼粒细胞白血病患者合并弥散性血管内凝血的影响因素及列线图模型构建

目的:探讨急性早幼粒细胞白血病(APL)合并弥散性血管内凝血(DIC)的影响因素，建立初诊APL患者发生DIC风险的预测模型，为临床诊治提供参考。方法:回顾性分析单中心179例初诊APL患者的临床资料，根据有无DIC进行分组，采用单因素及多因素回归分析，探讨APL合并DIC的影响因素，构建列线图预测模型，采用校正曲线、受试者工作特征曲线下的面积(AUC)及一致性指数(CI)评价模型的预测价值。结果:179例初诊APL患者平均年龄41.77岁，根据临床和实验室指标分为DIC组(95例，53.07%),非DIC组(84例，46.93%)。单因素分析显示：预后分层、骨髓早幼粒细胞比例、初诊时白细胞计数、中性粒细胞绝对值、凝血酶原时间、凝血酶时间、纤维蛋白原(FIB)、纤维蛋白降解产物(FDP)、D-二聚体、乳酸脱氢酶(LDH)水平在2组中比较，差异有统计学意义(均P<0.05)。多因素分析显示，初诊时FIB、FDP及LDH水平为APL合并DIC的独立影响因素(OR=0.418、1.009、1.005,P<0.05)。列线图模型预测APL合并DIC的AUC为0.845,CI为0.8...     

急性白血病早期死亡高危因素的回归分析

采用病例对照研究，对11年上海地区27家医院2867例急性白血病（AL）住院患者早期死亡的高危同素进行Lognistic回归分析。结果：出血、感染和多脏器衰竭是AL死亡的主要原因。全反式维甲酸（ATRA）的应用，降低了急性早幼粒细胞白血病（APL）早期病死率。早期死亡的高危因素为：年龄≥60岁或≤1岁，发病时伴有明显出血、外用血白细胞＞100X109／L、BPC＜50X109／L。结论：针对上述高危因素采取相应防治措施，降低AL的早期病死率十分必要。     

白细胞淤滞和维甲酸综合征：急性早幼粒细胞白血病早期死亡原因分析（附5例报告）

５２例初诊ＡＰＬ患者单用全反式维甲酸（ＡＴＲＡ）治疗，其中５例在治疗后１月内早期死亡。除１例因ＡＰＬ本身严重的血小板减少所致的颅内出血外，其余４例死于与ＡＴＲＡ治疗相关的白细胞淤滞或维甲酸综合征。结合有关文献复习了这些严重合并症的临床表现和发病机理，并提出初步的处理意见。     

Inclusion of hemoglobin level in prognostic score provides better prognostic stratification in patients with acute promyelocytic leukemia (APL)

The clinical outcomes of acute promyelocytic leukemia (APL) have improved greatly, but treatment failure still occurs. Identification of patients with poor prognosis is fundamental, and we propose a new clinical prognostic system (CBC-score) consisting of WBC, platelet count, and hemoglobin level. Between 1995 and 2009, 156 patients with APL from seven institutes in Korea were retrospectively reviewed. In the new CBC-score system, each of the following (WBC ≥10 × 109/L, platelet <40 × 109/L, hemoglobin <8.0 g/dL) was considered as a risk factor; the sum of each was designated as the CBC-score. With a median follow-up of 8.4 years, the complete remission (CR) rate was 81.4 % (127/156), while 24 (15.4 %) were considered as treatment failures due to early death (ED). The 5-year overall survival (OS), leukemia-free survival, and cumulative incidence of relapse were 73.8, 82.8, and 13.5 %, respectively. Compared to the individual CBC parameters, combined prognostic systems such as PETHEMA or CBC-score provided better prognostic stratification. Compared to PETHEMA stratification, the proposed prognostic CBC-score system showed better stratification of APL patients in terms of CR rates (p = 0.004), OS (p = 0.004), and ED (p = 0.008). This retrospective study suggests that the proposed CBC-score may provide better prognostic stratification of APL patients.     

Clinical bleeding events and laboratory coagulation profiles in acute promyelocytic leukemia

Background: Bleeding is the leading cause of death for patients with acute promyelocytic leukemia (APL). Blood component transfusion to correct coagulopathy is the keystone in reducing bleeding. The benefit of fresh frozen plasma transfusion is unproven. Using laboratory profiles to predict bleeding is important guidance for the determination of transfusion policies in the treatment of APL. Design and methods: For 116 patients of APL, bleeding events were collected and correlated with various hematologic and coagulation parameters, including leukemic cell percentages, white blood cell (WBC) and platelet counts, prothrombin time (PT), activated partial thromboplastin time (APTT), fibrinogen levels, and disseminated intravascular coagulation (DIC) scores. Results: Overt DIC occurred in 77.6% of patients. Severity of DIC was associated with bone marrow leukemic cell percentages but unrelated to bleeding. Patients with bleeding had significantly higher WBC counts (26.73 ± 6.18 vs. 13.03 ± 3.03 per μL, P = 0.026) and more prolonged PT (4.85 ± 0.70 vs. 2.59 ± 0.28 s, P = 0.002) and APTT (3.98 ± 1.68 vs. 0.96 ± 0.93 s, P = 0.017). Fibrinogen levels, platelet counts, and leukemia cell percentages were not significantly different between bleeding and non‐bleeding patients. PT is valuable in prediction of bleeding. Patients with PT ≧ 5 s had a relative risk of 6.14 for bleeding. Seven patients had severe bleeding before initiation of all‐trans retinoic acid (ATRA). Conclusions: Patients with APL are susceptible to DIC and subsequent bleeding events. Prompt ATRA administration is crucial in preventing hemorrhagic events. High WBC counts, prolonged PT, and APTT are associated with clinical bleeding in our series. PT is the most accurate parameter in predicting bleeding. Based on these findings, supportive care should be directed toward correction of coagulopathy to prevent bleeding complications and fresh frozen plasma appears to be indicated for coagulopathy associated with APL.     

Difference in causes and prognostic factors of early death between cohorts with de novo and relapsed acute promyelocytic leukemia

Early death (ED) remains the most critical issue in the current care of patients with acute promyelocytic leukemia (APL). Very limited data are available regarding ED in patients with relapsed APL. In this retrospective study, 285 de novo and 79 relapsed patients were included. All patients received single-agent arsenic trioxide as induction therapy. The differences in baseline clinical features, incidence, causes, and prognostic factors of ED were compared between the two patient cohorts. The relapse cohort exhibited a better overall condition than the de novo cohort upon hospital admission. The ED rate in the relapsed patients (24.1%) was somewhat higher than that in the de novo patients (17.9%), although the difference was not significant (P?=?0.219). For both cohorts, hemorrhage was the main cause of ED, followed by differentiation syndrome, infection, and other causes. Increased serum creatinine level, older age, male sex, white blood cell (WBC) count?>?10?×?10⁹/L, and fibrinogen?<?1 g/L were independently risk factors for ED in the de novo patients, whereas WBC count?>?10?×?10⁹/L, elevated serum uric acid level, and D-dimer?>?4 mg/L were independent risk factors for ED in the relapsed patients. These data furnish clinically relevant information that might be useful for designing more appropriate risk-adapted treatment protocols aimed at reducing ED rate in patients with relapsed APL.     

The global problem of early deaths in acute promyelocytic leukemia: A strategy to decrease induction mortality in the most curable leukemia

Acute promyelocytic leukemia (APL) is a hyper-acute illness and presents with profound cytopenias in most patients and disseminated intravascular coagulation (DIC). Excellent treatment options are now available with drugs such as all-trans retinoic acid (ATRA), arsenic trioxide (ATO), anthracyclines and cytarabine. The outcome in APL has improved tremendously in the last 50 years due to better understanding of the disease, development of effective targeted agents and improvement in supportive care. Carefully selected groups of patients treated in large multi-center trials on a protocol and in experienced centers have shown survival rates in excess of 85%. However population data and other studies show that approximately 30% of patients die during induction. This is an Institutional, national and global problem and remains a pressing and frustrating challenge in APL.While most APL experts are aware of the high rate of early deaths (ED), such awareness is not typically present among general hematologists and oncologists. Our area of focus over the last 7 years has been the reduction of ED in both academic and community centers; as a result we have acquired substantial experience in APL induction. Two centers have implemented population-wide prospective trials; Brazil and Georgia/South Carolina, USA with improvement in the ED rate. Both centers used standardized guidelines along with consultative support and sharing of expertise which proved effective and helped to decrease ED.Induction mortality in APL is 30% or greater. We believe ED is largely preventable and population-wide survival can be improved. An effective strategy is to utilize a set of simplified treatment guidelines coupled with support from a group of experts during induction. Treating oncologists in both academic and community hospitals should receive aggressive education about ED and be encouraged to seek advice from a core group of established APL experts. This model could be implemented nationally to improve population-wide survival in this most curable leukemia.     

Acute promyelocytic leukaemia is characterized by stable incidence and improved survival that is restricted to patients managed in leukaemia referral centres: a pan‐Canadian epidemiological study

Timely diagnosis and care are major determinants of the outcome in acute promyelocytic leukaemia (APL), a malignancy whose incidence may be increasing. The Canadian Cancer Registry (CCR) and health system represent valuable settings to study APL epidemiology. We analysed the CCR, which contains data on all Canadians with APL. To provide clinical information lacking in the CCR, we obtained data from five leukaemia referral centres during a similar time period. Between 1993 and 2007, there were 399 APL in Canada. Age‐standardized incidence was 0·083/100 000 and was stable over time. The early death (ED) rate was 21·8% (10·6% in patients <50 years old and 35·5% for those aged >50 years), with no improvement over time. Five‐year overall survival (OS) was 54·6% (73·3% in patients <50 years; 29·1% older patients). In the referral cohort, 131 patients were diagnosed between 1999 and 2010. ED was 14·6% and 2‐year OS was 76·5%. Within this cohort, ED and OS improved over time, although advanced patient age remained an adverse determinant of OS. In Canada, APL incidence is unexpectedly low and temporally stable. ED was higher than reported in clinical trials, but similar to reports from other registries. In contrast, ED was lower in referral centres and improved with time.     

Clinicobiological features and outcome of acute promyelocytic leukemia occurring as a second tumor: the GIMEMA experience

We analyzed the clinicobiological features and treatment outcome of a series of acute promyelocytic leukemias (APLs) occurring as a second tumor (APL-st's, n = 51) and compared these with a large group of de novo APL cases (n = 641), both observed by the Italian cooperative group GIMEMA. In the APL-st group, 37 patients had received radiotherapy and/or chemotherapy for their primary malignancy (PM), while 14 had been treated by surgery alone. Compared with de novo APL patients, APL-st patients were characterized by a predominance of females (P <.003), higher median age (P <.05), and worse performance status (P <.005). The median time elapsed between PM and APL-st was 36 months, with a longer latency for patients treated with surgery alone. No significant differences were found with regard to karyotypic lesions or type of promyelocytic leukemia/retinoic acid receptor alpha (PML/RARalpha) fusion in the 2 cohorts. A high prevalence of PMs of the reproductive system was observed among the female APL-st population (24 [71%] of 34 patients in this group had suffered from breast, uterine, or ovarian cancer). Thirty-one APL-st and 641 de novo APL patients received homogeneous APL therapy according to the all-trans retinoic acid (ATRA) and idarubicin regimen (the AIDA regimen). The complete remission (CR), 4-year event-free survival (EFS), and 4-year overall survival (OS) rates were 97% and 93%, 65% and 68%, and 85% and 78% in the APL-st and de novo APL groups, respectively. In spite of important clinical differences (older age and poorer performance status), the APL-st group responded as well as the de novo APL group to upfront ATRA plus chemotherapy, probably reflecting genetic similarity.     

Clinical characteristics and outcome of childhood acute promyelocitic leukemia (APL) in Saudi Arabia: a multicenter SAPHOS leukemia group study

Background: Acute promyelocytic leukemia (APL) is a rare form of acute myelogenous leukemia (AML). Survival rates exceed 80% in developed countries. Successful treatments rely on all-trans retinoic acid with anthracycline-based chemotherapy. Availability of modern care and public knowledge play important roles in pediatric APL survival.

Method: A cytogenetic diagnosis of APL was confirmed in 30 (14.5%) out of 207 children consecutively diagnosed with de novo AML between January 2005 and December 2012 at nine cancer care centers in Saudi Arabia. Patients were treated based on the standard protocol used by the center following the PETHEMA or the C9710 treatment protocols. We modeled 5-year overall survival (OS), event-free survival (EFS) and cumulative incidence of relapse (CIR) vs. treatment and potential covariates of age at diagnosis, involvement of central nervous system (CNS), and white blood cell (WBC) levels.

Results: The median age was 10.4 years with a male:female ratio of 1.9. WBC was 10?×?10⁹/l or greater in 57% and CNS involvement was confirmed in 13%. OS, EFS, and CIR were 74?±?12%, 55?±?19%, and, 36?±?17% respectively. No significant difference was found by treatment protocol. WBC levels were significantly prognostic for all negative events, but treatment with C9710 significantly ameliorated negative WBC effects. Overall outcomes were comparable to those reported in developed countries.

Conclusions: Access to modern care is likely to be a critical factor in successful and comparable outcomes of childhood APL across the globe. In the present study, utilizing a cytarabine-containing protocol improved outcome of high-risk pediatric patients with APL.     

Severe hemorrhagic complications during remission induction therapy for acute promyelocytic leukemia: incidence, risk factors, and influence on outcome

BACKGROUND: Even after the introduction of all-trans retinoic acid (ATRA), early hemorrhagic death remains a major cause of remission induction failure for acute promyelocytic leukemia (APL). METHODS: To investigate severe hemorrhagic complications during remission induction therapy with respect to incidence, risk factors, and influence on outcome. Results were analyzed for 279 patients enrolled in the APL97 study conducted by the Japan Adult Leukemia Study Group (JALSG). RESULTS: Severe hemorrhage occurred in 18 patients (6.5%). Although most of them were receiving frequent transfusions, the targeted levels of platelet counts (30 x 10(9)/L) and plasma fibrinogen (1.5 g/L) for this study were reached at the day of bleeding in only 71% and 40%, respectively. Nine of them succumbed to an early death, while the remaining nine patients eventually achieved complete remission (CR). The 5-yr event-free survival rate was 68.1% for those who did not suffer severe hemorrhage, and 31.1% for those who did (P < 0.0001). For patients who achieved CR, on the other hand, there was no difference in disease-free survival between patients with and without severe hemorrhage (P = 0.6043). Risk factor analysis identified three pretreatment variables associated with severe hemorrhage: initial fibrinogen level, white blood cell count, and performance status. Additionally, patients with severe hemorrhage were more easily prone to develop retinoic acid syndrome or pneumonia than patients without hemorrhage. CONCLUSIONS: These results indicate that fatal hemorrhage represents a major obstacle in curing APL, and that patients with such high-risk features may benefit from more aggressive supportive care.     

Successful All-<Emphasis Type="Italic">trans</Emphasis> Retinoic Acid Treatment of Acute Promyelocytic Leukemia in a Patient with <Emphasis Type="Italic">NPM/RAR</Emphasis> Fusion

Acute promyelocytic leukemia (APL) is characterized by a reciprocal chromosomal translocation involving the gene for retinoic acid receptor alpha(RAR). Most APL patients have a t(15;17) translocation that generates the PML-RAR fusion gene, and such patients respond well to treatment with all-trans retinoic acid (ATRA). Some APL cases also involve rearrangements that fuse RAR to partner genes other than PML, including nucleophosmin (NPM), promyelocytic leukemia zinc finger (PLZF), nuclear mitotic apparatus (NUMA), and Stat5b, but the clinical characteristics of APL without PML-RAR have not been fully clarified. We describe a 64-year-old man with NPM-RAR-positive APL who was receiving hemodialysis therapy for chronic uremia. Complete remission was achieved with ATRA monotherapy and was maintained for 18 months with consolidation chemotherapy. These findings suggest that ATRA can be used to treat APL patients with NPM/RAR as well as APL with PML/RAR.