Hematopoietic stem cell transplantation for patients with acute lymphoblastic leukemia and Down syndrome

Hiroaki Goto, Takashi Kaneko, Yoko Shioda, Michiko Kajiwara, Kazuo Sakashita, Toshiyuki Kitoh, Akira Hayakawa, Mizuka Miki, Keisuke Kato, Atsushi Ogawa, Yoshiko Hashii, Takeshi Inukai, Chiaki Kato, Hisashi Sakamaki, Hiromasa Yabe, Ritsuro Suzuki, Koji Kato

Research output: Contribution to journalArticle

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Abstract

Background: Hematopoietic stem cell transplantation (HSCT) is one curable option for high-risk acute lymphoblastic leukemia (ALL); however, transplant-related toxicities might be severe in patients with Down syndrome and ALL (DS-ALL). Procedure: HSCTs performed in patients with DS-ALL were identified in the Japan Society for Hematopoietic Cell Transplantation registry. Results: In the registry data, 11 patients with DS-ALL were identified. The median age at HSCT was 9 years (range: 6-22 years). Six patients underwent HSCT at non-remission status. Allogeneic grafts were utilized in all patients, including eight patients who received HSCT from unrelated donors. Reduced intensity conditioning regimens were used in three patients. All patients achieved neutrophil engraftment by a median of day 18 (range: day 11-61). Ten patients experienced grade 3 or more infectious episodes. Six patients experienced complications of the respiratory system. The incidences of II-IV or III-IV acute GVHD were nine (81.8%) or seven patients (63.6%), respectively. Chronic GVHD was observed in five (55.6%) out of nine evaluable patients. Seven patients died at a median of 6 months (range: 0-24 months) after HSCT. Two-year relapse-free and overall survival were 33.3% (95% CI: 2.5-64.1%) or 37.5% (95% CI: 5.9-69.1%), respectively. The causes of death were relapse (n=2), infection (n=2), bleeding (n=1), thrombotic microangiopathy (n=1), and chronic GVHD (n=1). Conclusions: Therapy-related mortality accounted for five out of seven deceased patients in this case series. Attempts to reduce toxicities should be considered in HSCT for patients with DS-ALL. Pediatr Blood Cancer 2015;62:148-152.

Original languageEnglish
Pages (from-to)148-152
Number of pages5
JournalPediatric Blood and Cancer
Volume62
Issue number1
DOIs
Publication statusPublished - Jan 1 2015
Externally publishedYes

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Hematopoietic Stem Cell Transplantation
Down Syndrome
Precursor Cell Lymphoblastic Leukemia-Lymphoma
Registries
Thrombotic Microangiopathies
Transplants
Recurrence
Unrelated Donors
Cell Transplantation
Respiratory System
Cause of Death

All Science Journal Classification (ASJC) codes

  • Pediatrics, Perinatology, and Child Health
  • Hematology
  • Oncology

Cite this

Hematopoietic stem cell transplantation for patients with acute lymphoblastic leukemia and Down syndrome. / Goto, Hiroaki; Kaneko, Takashi; Shioda, Yoko; Kajiwara, Michiko; Sakashita, Kazuo; Kitoh, Toshiyuki; Hayakawa, Akira; Miki, Mizuka; Kato, Keisuke; Ogawa, Atsushi; Hashii, Yoshiko; Inukai, Takeshi; Kato, Chiaki; Sakamaki, Hisashi; Yabe, Hiromasa; Suzuki, Ritsuro; Kato, Koji.

In: Pediatric Blood and Cancer, Vol. 62, No. 1, 01.01.2015, p. 148-152.

Research output: Contribution to journalArticle

Goto, H, Kaneko, T, Shioda, Y, Kajiwara, M, Sakashita, K, Kitoh, T, Hayakawa, A, Miki, M, Kato, K, Ogawa, A, Hashii, Y, Inukai, T, Kato, C, Sakamaki, H, Yabe, H, Suzuki, R & Kato, K 2015, 'Hematopoietic stem cell transplantation for patients with acute lymphoblastic leukemia and Down syndrome', Pediatric Blood and Cancer, vol. 62, no. 1, pp. 148-152. https://doi.org/10.1002/pbc.25245
Goto, Hiroaki ; Kaneko, Takashi ; Shioda, Yoko ; Kajiwara, Michiko ; Sakashita, Kazuo ; Kitoh, Toshiyuki ; Hayakawa, Akira ; Miki, Mizuka ; Kato, Keisuke ; Ogawa, Atsushi ; Hashii, Yoshiko ; Inukai, Takeshi ; Kato, Chiaki ; Sakamaki, Hisashi ; Yabe, Hiromasa ; Suzuki, Ritsuro ; Kato, Koji. / Hematopoietic stem cell transplantation for patients with acute lymphoblastic leukemia and Down syndrome. In: Pediatric Blood and Cancer. 2015 ; Vol. 62, No. 1. pp. 148-152.
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abstract = "Background: Hematopoietic stem cell transplantation (HSCT) is one curable option for high-risk acute lymphoblastic leukemia (ALL); however, transplant-related toxicities might be severe in patients with Down syndrome and ALL (DS-ALL). Procedure: HSCTs performed in patients with DS-ALL were identified in the Japan Society for Hematopoietic Cell Transplantation registry. Results: In the registry data, 11 patients with DS-ALL were identified. The median age at HSCT was 9 years (range: 6-22 years). Six patients underwent HSCT at non-remission status. Allogeneic grafts were utilized in all patients, including eight patients who received HSCT from unrelated donors. Reduced intensity conditioning regimens were used in three patients. All patients achieved neutrophil engraftment by a median of day 18 (range: day 11-61). Ten patients experienced grade 3 or more infectious episodes. Six patients experienced complications of the respiratory system. The incidences of II-IV or III-IV acute GVHD were nine (81.8{\%}) or seven patients (63.6{\%}), respectively. Chronic GVHD was observed in five (55.6{\%}) out of nine evaluable patients. Seven patients died at a median of 6 months (range: 0-24 months) after HSCT. Two-year relapse-free and overall survival were 33.3{\%} (95{\%} CI: 2.5-64.1{\%}) or 37.5{\%} (95{\%} CI: 5.9-69.1{\%}), respectively. The causes of death were relapse (n=2), infection (n=2), bleeding (n=1), thrombotic microangiopathy (n=1), and chronic GVHD (n=1). Conclusions: Therapy-related mortality accounted for five out of seven deceased patients in this case series. Attempts to reduce toxicities should be considered in HSCT for patients with DS-ALL. Pediatr Blood Cancer 2015;62:148-152.",
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AU - Goto, Hiroaki

AU - Kaneko, Takashi

AU - Shioda, Yoko

AU - Kajiwara, Michiko

AU - Sakashita, Kazuo

AU - Kitoh, Toshiyuki

AU - Hayakawa, Akira

AU - Miki, Mizuka

AU - Kato, Keisuke

AU - Ogawa, Atsushi

AU - Hashii, Yoshiko

AU - Inukai, Takeshi

AU - Kato, Chiaki

AU - Sakamaki, Hisashi

AU - Yabe, Hiromasa

AU - Suzuki, Ritsuro

AU - Kato, Koji

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AB - Background: Hematopoietic stem cell transplantation (HSCT) is one curable option for high-risk acute lymphoblastic leukemia (ALL); however, transplant-related toxicities might be severe in patients with Down syndrome and ALL (DS-ALL). Procedure: HSCTs performed in patients with DS-ALL were identified in the Japan Society for Hematopoietic Cell Transplantation registry. Results: In the registry data, 11 patients with DS-ALL were identified. The median age at HSCT was 9 years (range: 6-22 years). Six patients underwent HSCT at non-remission status. Allogeneic grafts were utilized in all patients, including eight patients who received HSCT from unrelated donors. Reduced intensity conditioning regimens were used in three patients. All patients achieved neutrophil engraftment by a median of day 18 (range: day 11-61). Ten patients experienced grade 3 or more infectious episodes. Six patients experienced complications of the respiratory system. The incidences of II-IV or III-IV acute GVHD were nine (81.8%) or seven patients (63.6%), respectively. Chronic GVHD was observed in five (55.6%) out of nine evaluable patients. Seven patients died at a median of 6 months (range: 0-24 months) after HSCT. Two-year relapse-free and overall survival were 33.3% (95% CI: 2.5-64.1%) or 37.5% (95% CI: 5.9-69.1%), respectively. The causes of death were relapse (n=2), infection (n=2), bleeding (n=1), thrombotic microangiopathy (n=1), and chronic GVHD (n=1). Conclusions: Therapy-related mortality accounted for five out of seven deceased patients in this case series. Attempts to reduce toxicities should be considered in HSCT for patients with DS-ALL. Pediatr Blood Cancer 2015;62:148-152.

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