Nand S, Godwin J E
Department of Medicine, Loyola University Stritch School of Medicine, Maywood, Illinois 60153.
Cancer. 1988 Sep 1;62(5):958-64. doi: 10.1002/1097-0142(19880901)62:5<958::aid-cncr2820620519>3.0.co;2-p.
Over a period of 8 years 11 of 64 patients seen at Loyola University Medical Center with the diagnosis of myelodysplastic syndrome (MDS) also exhibited bone marrow hypoplasia (marrow cellularity of 25% or less) at presentation. The other 53 had normocellular or hypercellular marrow. Clinical features, hemograms, chromosome analysis, incidence of progression to acute leukemia or aplastic anemia, and survival in each group were compared. Using the French-American-British (FAB) classification, there were seven patients with refractory anemia (RA), one refractory anemia with ringed sideroblasts (RARS), and three refractory anemia with excess blasts (RAEB) in the hypoplastic MDS group. Those with normocellular or hypercellular marrow included 22 with RA, nine with RARS, 12 with RAEB, three with chronic myelomonocytic leukemia, and four with RAEB in transformation; one had chronic diGuglielmo syndrome and two patients were not classified. Patients with hypoplastic MDS had lower hemoglobin levels (median, 8 g/dl versus 9 g/dl), more severe leucopenia (median 3100/microliter versus 4200/microliter) and thrombocytopenia (median, 28,000/microliter versus 75,000/microliter), and marked macrocytosis (mean corpuscular volume (MCV), 107 mu 3 versus 97 mu 3). Nine patients with hypoplastic MDS had a chromosome analysis of the bone marrow, and all were normal. In those with normocellular or hyperplastic bone marrow, 22 such analyses were done, and seven (23%) were abnormal. One patient (11%) from the hypoplastic group and 11 (23%) from the normocellular or hyperplastic MDS transformed into acute leukemia. None progressed to aplastic anemia. With a mean follow-up time of 33 months in the hypoplastic MDS, eight patients (72%) are alive. In the group with normal or hyperplastic MDS, the mean follow up was 47 months, and 27 patients (50%) have survived. The two groups differ significantly in leukocyte count (P less than 0.0015), platelet count (P less than 0.0001), and MCV (P less than 0.0023). There may be a possible difference between these groups related to abnormal karyotype, but it is not statistically significant (P = 0.06). Therapy with pyridoxine, folic acid, prednisone, anabolic steroids, retinoids, or low-dose cytosine arabinoside was not beneficial in hypoplastic MDS. Hypoplastic MDS appears to be a distinct clinicopathologic entity characterized by marrow hypoplasia, macrocytosis, severe leucopenia and thrombocytopenia, low incidence of progression to acute leukemia, and unresponsiveness to conventional therapy.
在8年的时间里，洛约拉大学医学中心诊治的64例骨髓增生异常综合征（MDS）患者中有11例在初诊时也表现为骨髓增生低下（骨髓细胞比例为25%或更低）。另外53例患者的骨髓细胞数量正常或增多。对两组患者的临床特征、血常规、染色体分析、进展为急性白血病或再生障碍性贫血的发生率以及生存率进行了比较。根据法美英（FAB）分类，增生低下的MDS组中有7例难治性贫血（RA）患者、1例环形铁粒幼细胞性难治性贫血（RARS）患者和3例原始细胞增多的难治性贫血（RAEB）患者。骨髓细胞数量正常或增多的患者包括22例RA患者、9例RARS患者、12例RAEB患者、3例慢性粒单核细胞白血病患者和4例转化中的RAEB患者；1例患有慢性迪古列尔莫综合征，2例未分类。增生低下的MDS患者血红蛋白水平较低（中位数，8 g/dl对9 g/dl），白细胞减少症更严重（中位数3100/微升对4200/微升）和血小板减少症（中位数，28,000/微升对75,000/微升），并且有明显的大细胞性（平均红细胞体积（MCV），107立方微米对97立方微米）。9例增生低下的MDS患者进行了骨髓染色体分析，结果均正常。在骨髓细胞数量正常或增多的患者中，进行了22次此类分析，其中7次（23%）异常。增生低下组中有1例患者（11%）和骨髓细胞数量正常或增多的MDS组中有11例患者（23%）转化为急性白血病。无人进展为再生障碍性贫血。增生低下的MDS患者平均随访时间为33个月，8例患者（72%）存活。在骨髓细胞数量正常或增多的MDS组中，平均随访时间为47个月，27例患者（50%）存活。两组在白细胞计数（P<0.0015）、血小板计数（P<0.0001）和MCV（P<0.0023）方面有显著差异。这些组之间在异常核型方面可能存在差异，但无统计学意义（P = 0.06）。使用吡哆醇、叶酸、泼尼松、同化类固醇、维甲酸或小剂量阿糖胞苷治疗增生低下的MDS并无益处。增生低下的MDS似乎是一种独特的临床病理实体，其特征为骨髓增生低下、大细胞性、严重白细胞减少和血小板减少、进展为急性白血病的发生率低以及对传统治疗无反应。
