Hematopoietic stem cell transplantation in patients with severe congenital neutropenia: An analysis of 18 Japanese cases

Koichi Oshima, Ryoji Hanada, Ryoji Kobayashi, Koji Kato, Yoshihisa Nagatoshi, Ken Tabuchi, Shunichi Kato

Research output: Contribution to journalArticle

16 Citations (Scopus)

Abstract

We studied the outcome of allogeneic HSCT in patients with SCN. Between 1989 and 2005, 18 patients with SCN in Japan received HSCT for reasons other than malignant transformation, i.e., because of the lack of or a partial response to treatment with r-HuG-CSF. The median age of the patients at the first HSCT was three and a half yr (range 0.2-16.7 yr). Nine patients received stem cells from an HLA-identical sibling donor and nine from an alternative donor. Twelve and six patients received myeloablative and non-myeloablative conditioning regimens, respectively. Engraftment occurred at the first HSCT in 12 patients, four patients received a second HSCT for graft failure, and two patients died. The cause of death was renal failure and graft failure at the first and second HSCT, respectively. The cumulative incidence of grade II-IV acute GVHD and TRM at the first transplantation was 11% and 5.6%, respectively. Of our patients, 16 are alive and in complete remission, with a median follow-up of six and a half yr. Our results suggest that HSCT is beneficial for patients with SCN refractory to r-HuG-CSF treatment.

Original languageEnglish
Pages (from-to)657-663
Number of pages7
JournalPediatric Transplantation
Volume14
Issue number5
DOIs
Publication statusPublished - Aug 1 2010
Externally publishedYes

Fingerprint

Hematopoietic Stem Cell Transplantation
Neutropenia, Severe Congenital, Autosomal Recessive 3
Tissue Donors
Transplants
Renal Insufficiency
Siblings
Cause of Death
Japan
Stem Cells
Transplantation

All Science Journal Classification (ASJC) codes

  • Pediatrics, Perinatology, and Child Health
  • Transplantation

Cite this

Hematopoietic stem cell transplantation in patients with severe congenital neutropenia : An analysis of 18 Japanese cases. / Oshima, Koichi; Hanada, Ryoji; Kobayashi, Ryoji; Kato, Koji; Nagatoshi, Yoshihisa; Tabuchi, Ken; Kato, Shunichi.

In: Pediatric Transplantation, Vol. 14, No. 5, 01.08.2010, p. 657-663.

Research output: Contribution to journalArticle

Oshima, Koichi ; Hanada, Ryoji ; Kobayashi, Ryoji ; Kato, Koji ; Nagatoshi, Yoshihisa ; Tabuchi, Ken ; Kato, Shunichi. / Hematopoietic stem cell transplantation in patients with severe congenital neutropenia : An analysis of 18 Japanese cases. In: Pediatric Transplantation. 2010 ; Vol. 14, No. 5. pp. 657-663.
@article{aea7eb874a6b460da07a8c0d5ce6d51e,
title = "Hematopoietic stem cell transplantation in patients with severe congenital neutropenia: An analysis of 18 Japanese cases",
abstract = "We studied the outcome of allogeneic HSCT in patients with SCN. Between 1989 and 2005, 18 patients with SCN in Japan received HSCT for reasons other than malignant transformation, i.e., because of the lack of or a partial response to treatment with r-HuG-CSF. The median age of the patients at the first HSCT was three and a half yr (range 0.2-16.7 yr). Nine patients received stem cells from an HLA-identical sibling donor and nine from an alternative donor. Twelve and six patients received myeloablative and non-myeloablative conditioning regimens, respectively. Engraftment occurred at the first HSCT in 12 patients, four patients received a second HSCT for graft failure, and two patients died. The cause of death was renal failure and graft failure at the first and second HSCT, respectively. The cumulative incidence of grade II-IV acute GVHD and TRM at the first transplantation was 11{\%} and 5.6{\%}, respectively. Of our patients, 16 are alive and in complete remission, with a median follow-up of six and a half yr. Our results suggest that HSCT is beneficial for patients with SCN refractory to r-HuG-CSF treatment.",
author = "Koichi Oshima and Ryoji Hanada and Ryoji Kobayashi and Koji Kato and Yoshihisa Nagatoshi and Ken Tabuchi and Shunichi Kato",
year = "2010",
month = "8",
day = "1",
doi = "10.1111/j.1399-3046.2010.01319.x",
language = "English",
volume = "14",
pages = "657--663",
journal = "Pediatric Transplantation",
issn = "1397-3142",
publisher = "Wiley-Blackwell",
number = "5",

}

TY - JOUR

T1 - Hematopoietic stem cell transplantation in patients with severe congenital neutropenia

T2 - An analysis of 18 Japanese cases

AU - Oshima, Koichi

AU - Hanada, Ryoji

AU - Kobayashi, Ryoji

AU - Kato, Koji

AU - Nagatoshi, Yoshihisa

AU - Tabuchi, Ken

AU - Kato, Shunichi

PY - 2010/8/1

Y1 - 2010/8/1

N2 - We studied the outcome of allogeneic HSCT in patients with SCN. Between 1989 and 2005, 18 patients with SCN in Japan received HSCT for reasons other than malignant transformation, i.e., because of the lack of or a partial response to treatment with r-HuG-CSF. The median age of the patients at the first HSCT was three and a half yr (range 0.2-16.7 yr). Nine patients received stem cells from an HLA-identical sibling donor and nine from an alternative donor. Twelve and six patients received myeloablative and non-myeloablative conditioning regimens, respectively. Engraftment occurred at the first HSCT in 12 patients, four patients received a second HSCT for graft failure, and two patients died. The cause of death was renal failure and graft failure at the first and second HSCT, respectively. The cumulative incidence of grade II-IV acute GVHD and TRM at the first transplantation was 11% and 5.6%, respectively. Of our patients, 16 are alive and in complete remission, with a median follow-up of six and a half yr. Our results suggest that HSCT is beneficial for patients with SCN refractory to r-HuG-CSF treatment.

AB - We studied the outcome of allogeneic HSCT in patients with SCN. Between 1989 and 2005, 18 patients with SCN in Japan received HSCT for reasons other than malignant transformation, i.e., because of the lack of or a partial response to treatment with r-HuG-CSF. The median age of the patients at the first HSCT was three and a half yr (range 0.2-16.7 yr). Nine patients received stem cells from an HLA-identical sibling donor and nine from an alternative donor. Twelve and six patients received myeloablative and non-myeloablative conditioning regimens, respectively. Engraftment occurred at the first HSCT in 12 patients, four patients received a second HSCT for graft failure, and two patients died. The cause of death was renal failure and graft failure at the first and second HSCT, respectively. The cumulative incidence of grade II-IV acute GVHD and TRM at the first transplantation was 11% and 5.6%, respectively. Of our patients, 16 are alive and in complete remission, with a median follow-up of six and a half yr. Our results suggest that HSCT is beneficial for patients with SCN refractory to r-HuG-CSF treatment.

UR - http://www.scopus.com/inward/record.url?scp=77954514634&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=77954514634&partnerID=8YFLogxK

U2 - 10.1111/j.1399-3046.2010.01319.x

DO - 10.1111/j.1399-3046.2010.01319.x

M3 - Article

C2 - 20331518

AN - SCOPUS:77954514634

VL - 14

SP - 657

EP - 663

JO - Pediatric Transplantation

JF - Pediatric Transplantation

SN - 1397-3142

IS - 5

ER -