LONG-TERM SURVIVAL AND CURE AFTER MARROW TRANSPLANTATION FOR CONGENITAL HYPOPLASTIC-ANEMIA (DIAMOND-BLACKFAN SYNDROME)

被引:37
作者
GREINIX, HT
STORB, R
SANDERS, JE
DEEG, HJ
DONEY, KC
SULLIVAN, KM
WITHERSPOON, RP
机构
[1] UNIV WASHINGTON,SCH MED,FRED HUTCHINSON CANC RES CTR,DIV CLIN RES,1124 COLUMBIA ST,M318,SEATTLE,WA 98195
[2] UNIV WASHINGTON,SCH MED,DEPT MED,SEATTLE,WA 98195
关键词
D O I
10.1111/j.1365-2141.1993.tb03109.x
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
Four patients with Diamond-Blackfan syndrome (congenital hypoplastic anaemia) whose disease was resistant to corticosteroid treatment and who were red blood cell transfusion-dependent, were given marrow grafts from allogeneic human-leucocyte-antigen (HLA)-identical siblings. The patients were conditioned with regimens including cyclophosphamide and busulfan. Three of four patients had sustained and complete marrow engraftment. One patient showed early signs of haematopoietic recovery but died on day 3 5 of pulmonary toxicity. The three surviving patients are well with normal haematopoiesis and Karnofsky performance scores of 100%, 3.0, 7.4 and 10.6 years after transplantation. Congenital hypoplastic anaemia can be treated successfully by allogeneic marrow grafts.
引用
收藏
页码:515 / 520
页数:6
相关论文
共 48 条
  • [1] ABSENCE OF ABNORMALITIES OF C-KIT OR ITS LIGAND IN 2 PATIENTS WITH DIAMOND-BLACKFAN ANEMIA
    ABKOWITZ, JL
    BROUDY, VC
    BENNETT, LG
    ZSEBO, KM
    MARTIN, FH
    [J]. BLOOD, 1992, 79 (01) : 25 - 28
  • [2] ANTIN JH, 1985, BLOOD, V66, P1247
  • [3] AUGUST CS, 1976, BLOOD, V48, P491
  • [4] AUTHANOSIOU A, 1977, LANCET, V2, P616
  • [5] BAGNARA GP, 1991, BLOOD, V78, P2203
  • [6] MYASTHENIA-GRAVIS AFTER ALLOGENEIC BONE-MARROW TRANSPLANTATION - RELATIONSHIP TO CHRONIC GRAFT-VERSUS-HOST DISEASE
    BOLGER, GB
    SULLIVAN, KM
    SPENCE, AM
    APPELBAUM, FR
    JOHNSTON, R
    SANDERS, JE
    DEEG, HJ
    WITHERSPOON, RP
    DONEY, KC
    NIMS, J
    THOMAS, ED
    STORB, R
    [J]. NEUROLOGY, 1986, 36 (08) : 1087 - 1091
  • [7] DEEG HJ, 1980, BLOOD, V55, P233
  • [8] DEEG HJ, 1983, BLOOD, V61, P954
  • [9] Diamond L K, 1976, Adv Pediatr, V22, P349
  • [10] DRACHTMAN RA, 1992, BLOOD, V79, P2177