Allogeneic bone marrow transplantation for fourteen patients with severe aplastic anemia

Erh Jueng Hsueh, Wei Shou Hwang, Shyuh Huei Huang, Tsu Yi Chao, Jang Yang Chang, Chang Chung Wang

Research output: Contribution to journalArticle

Abstract

Background: Bone marrow transplantation (BMT) is the best curative approach for younger patients with severe aplastic anemia (SAA). Major obstacles to success of allogeneic BMT include graft-versus-host disease (GVHD), graft rejection and treatment related toxicities. Experience with 14 SAA patients who received BMT is reported here. Methods: From December 1986 to May 1995, 14 patients with SAA were treated with BMT; 13 were allogeneic, and 1 was syngeneic. There were nine males and five females whose average age was 24.7 years (range 15-36 years). The median pretransplant disease duration was 93 days (range 7-610 days). Five patients were nontransfused before BMT. The pre-transplant conditioning regimen consisted of 200 mg/kg cyclophosphamide (CY) intravenously, divided over four consecutive days, followed by 300 cGy total-body irradiation (TBI) on the day before BMT. Two untransfused, one transfused patient and one syngeneic transplant received CY only as preconditioning. For GVHD prophylaxis, the 13 patients were given a combination of cyclosporine and a short course of methotrexate. Results: Of the 14 patients 11 were still alive 10 to 90 months later, with functional engraftment; the median survival of 39 months. There were three deaths including one with primary graft failure with intracranial hemorrhage, and two with delayed graft rejection and sepsis. The patient who received syngeneic BMT developed late graft failure six months post-transplant, but was successfully treated with a second BMT. Acute GVHD occurred among 5 of the 13 engrafted patients, only one of whom was Grade III clinically. Chronic GVHD was observed in 2 out of 10 evaluable patients. Conclusions: The combination of CY and TBI is an effective, well-tolerated conditioning regimen for BMT in patients with SAA. The acute GVHD rate was low in our patients receiving cyclosporine. BMT is the treatment-of-choice for patients under the age of 40 with SAA, for those with human leucocyte antigen (HLA)-identical siblings or an identical twin and particularly for those patients who have not received transfusion.

Original languageEnglish
Pages (from-to)247-253
Number of pages7
JournalChinese Medical Journal (Taipei)
Volume57
Issue number4
Publication statusPublished - Apr 1996
Externally publishedYes

Fingerprint

Aplastic Anemia
Homologous Transplantation
Bone Marrow Transplantation
Graft vs Host Disease
Transplants
Cyclophosphamide
Whole-Body Irradiation
Graft Rejection
Cyclosporine
Isogeneic Transplantation
Monozygotic Twins
Intracranial Hemorrhages
HLA Antigens
Methotrexate
Siblings
Sepsis

Keywords

  • aplastic anemia
  • bone marrow transplantation
  • cyclophosphamide
  • graft-versus-host-disease
  • total body irradiation

ASJC Scopus subject areas

  • Medicine(all)

Cite this

Hsueh, E. J., Hwang, W. S., Huang, S. H., Chao, T. Y., Chang, J. Y., & Wang, C. C. (1996). Allogeneic bone marrow transplantation for fourteen patients with severe aplastic anemia. Chinese Medical Journal (Taipei), 57(4), 247-253.

Allogeneic bone marrow transplantation for fourteen patients with severe aplastic anemia. / Hsueh, Erh Jueng; Hwang, Wei Shou; Huang, Shyuh Huei; Chao, Tsu Yi; Chang, Jang Yang; Wang, Chang Chung.

In: Chinese Medical Journal (Taipei), Vol. 57, No. 4, 04.1996, p. 247-253.

Research output: Contribution to journalArticle

Hsueh, EJ, Hwang, WS, Huang, SH, Chao, TY, Chang, JY & Wang, CC 1996, 'Allogeneic bone marrow transplantation for fourteen patients with severe aplastic anemia', Chinese Medical Journal (Taipei), vol. 57, no. 4, pp. 247-253.
Hsueh, Erh Jueng ; Hwang, Wei Shou ; Huang, Shyuh Huei ; Chao, Tsu Yi ; Chang, Jang Yang ; Wang, Chang Chung. / Allogeneic bone marrow transplantation for fourteen patients with severe aplastic anemia. In: Chinese Medical Journal (Taipei). 1996 ; Vol. 57, No. 4. pp. 247-253.
@article{ade9f85198de4b26818b125b3451a49f,
title = "Allogeneic bone marrow transplantation for fourteen patients with severe aplastic anemia",
abstract = "Background: Bone marrow transplantation (BMT) is the best curative approach for younger patients with severe aplastic anemia (SAA). Major obstacles to success of allogeneic BMT include graft-versus-host disease (GVHD), graft rejection and treatment related toxicities. Experience with 14 SAA patients who received BMT is reported here. Methods: From December 1986 to May 1995, 14 patients with SAA were treated with BMT; 13 were allogeneic, and 1 was syngeneic. There were nine males and five females whose average age was 24.7 years (range 15-36 years). The median pretransplant disease duration was 93 days (range 7-610 days). Five patients were nontransfused before BMT. The pre-transplant conditioning regimen consisted of 200 mg/kg cyclophosphamide (CY) intravenously, divided over four consecutive days, followed by 300 cGy total-body irradiation (TBI) on the day before BMT. Two untransfused, one transfused patient and one syngeneic transplant received CY only as preconditioning. For GVHD prophylaxis, the 13 patients were given a combination of cyclosporine and a short course of methotrexate. Results: Of the 14 patients 11 were still alive 10 to 90 months later, with functional engraftment; the median survival of 39 months. There were three deaths including one with primary graft failure with intracranial hemorrhage, and two with delayed graft rejection and sepsis. The patient who received syngeneic BMT developed late graft failure six months post-transplant, but was successfully treated with a second BMT. Acute GVHD occurred among 5 of the 13 engrafted patients, only one of whom was Grade III clinically. Chronic GVHD was observed in 2 out of 10 evaluable patients. Conclusions: The combination of CY and TBI is an effective, well-tolerated conditioning regimen for BMT in patients with SAA. The acute GVHD rate was low in our patients receiving cyclosporine. BMT is the treatment-of-choice for patients under the age of 40 with SAA, for those with human leucocyte antigen (HLA)-identical siblings or an identical twin and particularly for those patients who have not received transfusion.",
keywords = "aplastic anemia, bone marrow transplantation, cyclophosphamide, graft-versus-host-disease, total body irradiation",
author = "Hsueh, {Erh Jueng} and Hwang, {Wei Shou} and Huang, {Shyuh Huei} and Chao, {Tsu Yi} and Chang, {Jang Yang} and Wang, {Chang Chung}",
year = "1996",
month = "4",
language = "English",
volume = "57",
pages = "247--253",
journal = "Journal of the Chinese Medical Association",
issn = "1726-4901",
publisher = "Elsevier Taiwan LLC",
number = "4",

}

TY - JOUR

T1 - Allogeneic bone marrow transplantation for fourteen patients with severe aplastic anemia

AU - Hsueh, Erh Jueng

AU - Hwang, Wei Shou

AU - Huang, Shyuh Huei

AU - Chao, Tsu Yi

AU - Chang, Jang Yang

AU - Wang, Chang Chung

PY - 1996/4

Y1 - 1996/4

N2 - Background: Bone marrow transplantation (BMT) is the best curative approach for younger patients with severe aplastic anemia (SAA). Major obstacles to success of allogeneic BMT include graft-versus-host disease (GVHD), graft rejection and treatment related toxicities. Experience with 14 SAA patients who received BMT is reported here. Methods: From December 1986 to May 1995, 14 patients with SAA were treated with BMT; 13 were allogeneic, and 1 was syngeneic. There were nine males and five females whose average age was 24.7 years (range 15-36 years). The median pretransplant disease duration was 93 days (range 7-610 days). Five patients were nontransfused before BMT. The pre-transplant conditioning regimen consisted of 200 mg/kg cyclophosphamide (CY) intravenously, divided over four consecutive days, followed by 300 cGy total-body irradiation (TBI) on the day before BMT. Two untransfused, one transfused patient and one syngeneic transplant received CY only as preconditioning. For GVHD prophylaxis, the 13 patients were given a combination of cyclosporine and a short course of methotrexate. Results: Of the 14 patients 11 were still alive 10 to 90 months later, with functional engraftment; the median survival of 39 months. There were three deaths including one with primary graft failure with intracranial hemorrhage, and two with delayed graft rejection and sepsis. The patient who received syngeneic BMT developed late graft failure six months post-transplant, but was successfully treated with a second BMT. Acute GVHD occurred among 5 of the 13 engrafted patients, only one of whom was Grade III clinically. Chronic GVHD was observed in 2 out of 10 evaluable patients. Conclusions: The combination of CY and TBI is an effective, well-tolerated conditioning regimen for BMT in patients with SAA. The acute GVHD rate was low in our patients receiving cyclosporine. BMT is the treatment-of-choice for patients under the age of 40 with SAA, for those with human leucocyte antigen (HLA)-identical siblings or an identical twin and particularly for those patients who have not received transfusion.

AB - Background: Bone marrow transplantation (BMT) is the best curative approach for younger patients with severe aplastic anemia (SAA). Major obstacles to success of allogeneic BMT include graft-versus-host disease (GVHD), graft rejection and treatment related toxicities. Experience with 14 SAA patients who received BMT is reported here. Methods: From December 1986 to May 1995, 14 patients with SAA were treated with BMT; 13 were allogeneic, and 1 was syngeneic. There were nine males and five females whose average age was 24.7 years (range 15-36 years). The median pretransplant disease duration was 93 days (range 7-610 days). Five patients were nontransfused before BMT. The pre-transplant conditioning regimen consisted of 200 mg/kg cyclophosphamide (CY) intravenously, divided over four consecutive days, followed by 300 cGy total-body irradiation (TBI) on the day before BMT. Two untransfused, one transfused patient and one syngeneic transplant received CY only as preconditioning. For GVHD prophylaxis, the 13 patients were given a combination of cyclosporine and a short course of methotrexate. Results: Of the 14 patients 11 were still alive 10 to 90 months later, with functional engraftment; the median survival of 39 months. There were three deaths including one with primary graft failure with intracranial hemorrhage, and two with delayed graft rejection and sepsis. The patient who received syngeneic BMT developed late graft failure six months post-transplant, but was successfully treated with a second BMT. Acute GVHD occurred among 5 of the 13 engrafted patients, only one of whom was Grade III clinically. Chronic GVHD was observed in 2 out of 10 evaluable patients. Conclusions: The combination of CY and TBI is an effective, well-tolerated conditioning regimen for BMT in patients with SAA. The acute GVHD rate was low in our patients receiving cyclosporine. BMT is the treatment-of-choice for patients under the age of 40 with SAA, for those with human leucocyte antigen (HLA)-identical siblings or an identical twin and particularly for those patients who have not received transfusion.

KW - aplastic anemia

KW - bone marrow transplantation

KW - cyclophosphamide

KW - graft-versus-host-disease

KW - total body irradiation

UR - http://www.scopus.com/inward/record.url?scp=0030013498&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=0030013498&partnerID=8YFLogxK

M3 - Article

C2 - 8705875

AN - SCOPUS:0030013498

VL - 57

SP - 247

EP - 253

JO - Journal of the Chinese Medical Association

JF - Journal of the Chinese Medical Association

SN - 1726-4901

IS - 4

ER -