Stem cell transplantation for paroxysmal nocturnal haemoglobinuria in childhood

被引：16
作者：
Flotho, C
Strahm, B
Kontny, U
Duffner, U
Peters, AMJ
Dupuis, W
Niemeyer, CM
机构：
[1] Univ Freiburg, Childrens Hosp, D-7800 Freiburg, Germany
[2] Childrens Hosp, Karlsruhe, Germany
来源：
BRITISH JOURNAL OF HAEMATOLOGY | 2002年 / 118卷 / 01期
关键词：
paroxysmal nocturnal haemoglobinuria; trans-plantation; PNH; SCT; BMT;
D O I：
10.1046/j.1365-2141.2002.03543.x
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
Paroxysmal nocturnal haemoglobinuria (PNH) is a clonal haematopoietic disorder characterized by chronic or intermittent intravascular haemolysis, variable cytopenia and an increased risk of thrombosis. Stem cell transplantation (SCT) is a curative therapeutic option, but its risks must be carefully weighed against the natural course of PNH. World-wide experience with SCT for PNH in the paediatric age group is scarce. We report on two adolescents suffering from PNH with life-threatening complications who were successfully transplanted from unrelated donors. Indications and techniques of SCT in childhood PNH are discussed and an overview of the literature is given.
引用
收藏
页码：124 / 127
页数：4
相关论文
共 12 条
[1]  
ANTIN JH, 1985, BLOOD, V66, P1247
[2]   Bone marrow transplantation for paroxysmal nocturnal haemoglobinuria [J].
Bemba, M ;
Guardiola, P ;
Garderet, L ;
Devergie, A ;
Ribaud, P ;
Esperou, H ;
Noguera, MH ;
Gluckman, E ;
Socié, G .
BRITISH JOURNAL OF HAEMATOLOGY, 1999, 105 (02) :366-368
[3]   Resolution of Budd-Chiari syndrome following bone marrow transplantation for paroxysmal nocturnal haemoglobinuria [J].
Graham, ML ;
Rosse, WF ;
Halperin, EC ;
Miller, CR ;
Ware, RE .
BRITISH JOURNAL OF HAEMATOLOGY, 1996, 92 (03) :707-710
[4]   NATURAL-HISTORY OF PAROXYSMAL-NOCTURNAL HEMOGLOBINURIA [J].
HILLMEN, P ;
LEWIS, SM ;
BESSLER, M ;
LUZZATTO, L ;
DACIE, JV .
NEW ENGLAND JOURNAL OF MEDICINE, 1995, 333 (19) :1253-1258
[5]   MARROW TRANSPLANTATION FOR PAROXYSMAL-NOCTURNAL HEMOGLOBINURIA [J].
KAWAHARA, K ;
WITHERSPOON, RP ;
STORB, R .
AMERICAN JOURNAL OF HEMATOLOGY, 1992, 39 (04) :283-288
[6]  
Locatelli F, 2001, BONE MARROW TRANSPL, V27, pS69
[7]  
Raiola AM, 2000, HAEMATOLOGICA, V85, P59
[8]   Bone marrow transplants for paroxysmal nocturnal haemoglobinuria [J].
Saso, R ;
Marsh, J ;
Cevreska, L ;
Szer, J ;
Gale, RP ;
Rowlings, PA ;
Passweg, JR ;
Nugent, ML ;
Luzzatto, L ;
Horowitz, MM ;
Gordon-Smith, EC .
BRITISH JOURNAL OF HAEMATOLOGY, 1999, 104 (02) :392-396
[9]  
Socié G, 2000, SEMIN HEMATOL, V37, P91
[10]   Successful application of nonmyeloablative transplantation for paroxysmal nocturnal hemoglobinuria [J].
Suenaga, K ;
Kanda, Y ;
Niiya, H ;
Nakai, K ;
Saito, T ;
Saito, A ;
Ohnishi, M ;
Takeuchi, T ;
Tanosaki, R ;
Makimoto, A ;
Miyawaki, S ;
Ohnishi, T ;
Kanai, S ;
Tobinai, K ;
Takaue, Y ;
Mineishi, S .
EXPERIMENTAL HEMATOLOGY, 2001, 29 (05) :639-642
12