Stem cell transplantation in patients with severe congenital neutropenia without evidence of leukemic transformation.
Stem cell transplantation in patients with severe congenital neutropenia without evidence of leukemic transformation.
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对患有严重先天性中性粒细胞减少症且无白血病转化证据的患者进行干细胞移植。
DOI：
10.1182/blood.v95.4.1195.004k36_1195_1198
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发表时间：
2000
期刊：
影响因子：
20.3
通讯作者：
J. Levine
中科院分区：
文献类型：
--
作者：
C. Zeidler;K. Welte;Y. Barak;F. Barriga;A. Bolyard;L. Boxer;G. Cornu;M. Cowan;D. Dale;T. Flood;M. Freedman;H. Gadner;H. Mandel;R. O'reilly;U. Ramenghi;A. Reiter;R. Skinner;C. Vermylen;J. Levine
严重先天性中性粒细胞减少症（CN） （Kostmann综合征）是一种血液系统疾病，其特征是早幼粒细胞/髓细胞发育阶段的骨髓生成成熟停止。这种停止导致严重的中性粒细胞减少，导致绝对中性粒细胞计数（ANC）低于0.2 x 10(9)/L，与婴儿期早期严重的细菌感染有关。从1994年开始，严重慢性中性粒细胞减少症国际登记处（SCNIR）收集了300多例CN患者的数据，表明90%以上的患者对重组人粒细胞集落刺激因子（r-HuG-CSF）治疗有应答，ANC大于1。0 × 10(9)/L。对于r-HuG-CSF治疗难治性且持续存在严重且经常危及生命的细菌感染的患者，造血干细胞移植是目前唯一可用的治疗方法。我们报告了在1976年至1998年期间，共有11例向SCNIR报告的CN患者因恶性转化以外的原因接受了移植。在这些患者中，8例对持续感染的r-HuG-CSF治疗无反应或仅显示部分反应。这些患者的结果表明，来自hla相同的同胞的干细胞移植对r-HuG-CSF难治性患者是有益的。(血。2000;95:1195 - 1198)
Severe congenital neutropenia (CN) (Kostmann syndrome) is a hematologic disorder characterized by a maturation arrest of myelopoiesis at the promyelocyte/myelocyte stage of development. This arrest results in severe neutropenia leading to absolute neutrophil counts (ANC) below 0.2 x 10(9)/L associated with severe bacterial infections from early infancy. Data on over 300 patients with CN collected by the Severe Chronic Neutropenia International Registry (SCNIR) beginning in 1994 indicate that more than 90% of these patients respond to recombinant human granulocyte-colony stimulating factor (r-HuG-CSF) treatment with an ANC greater than 1. 0 x 10(9)/L. For patients who are refractory to r-HuG-CSF treatment and continue to have severe and often life-threatening bacterial infections, hematopoietic stem cell transplantation is the only currently available treatment. We report on a total of 11 patients with CN reported to the SCNIR who underwent transplantation for reasons other than malignant transformation between 1976 and 1998. Of these patients, 8 were nonresponders or showed only partial response to r-HuG-CSF treatment with ongoing infections. Results from these patients suggest that transplantation of stem cells from an HLA-identical sibling is beneficial for patients refractory to r-HuG-CSF. (Blood. 2000;95:1195-1198)
影响因子：
158.5
作者：
BONILLA, MA;GILLIO, AP;OREILLY, RJ
通讯作者：
OREILLY, RJ
重组人粒细胞集落刺激因子（非格司亭）治疗严重慢性中性粒细胞减少症的随机对照 III 期试验。
影响因子：
20.3
作者：
Dale,DC;Bonilla,MA;Davis,MW;Nakanishi,AM;Hammond,WP;Kurtzberg,J;Wang,W;Jakubowski,A;Winton,E;Lalezari,P
通讯作者：
Lalezari,P
影响因子：
56.9
作者：
SOUZA, LM;BOONE, TC;WELTE, K
通讯作者：
WELTE, K