Allogeneic stem cell transplantation with reduced intensity conditioning for patients with adrenoleukodystrophy
Objective: The prognosis of adrenoleukodystrophy (ALD)with neurological involvement is generally dismal; however, allogeneic stem cell transplantation (SCT) is recognized as effective to stabilize or improve the clinical symptoms of ALD. Herein, we report the clinical outcomes of patients with ALD w...
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| Format: | Article |
| Language: | English |
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Elsevier
2019-03-01
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| Series: | Molecular Genetics and Metabolism Reports |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S2214426918300570 |
| _version_ | 1818343829622751232 |
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| author | Koji Kato Ryo Maemura Manabu Wakamatsu Ayako Yamamori Motoharu Hamada Shinsuke Kataoka Atsushi Narita Shunsuke Miwata Yuko Sekiya Nozomu Kawashima Kyogo Suzuki Kotaro Narita Sayoko Doisaki Hideki Muramatsu Hirotoshi Sakaguchi Kimikazu Matsumoto Yuka Koike Osamu Onodera Makiko Kaga Nobuyuki Shimozawa Nao Yoshida |
| author_facet | Koji Kato Ryo Maemura Manabu Wakamatsu Ayako Yamamori Motoharu Hamada Shinsuke Kataoka Atsushi Narita Shunsuke Miwata Yuko Sekiya Nozomu Kawashima Kyogo Suzuki Kotaro Narita Sayoko Doisaki Hideki Muramatsu Hirotoshi Sakaguchi Kimikazu Matsumoto Yuka Koike Osamu Onodera Makiko Kaga Nobuyuki Shimozawa Nao Yoshida |
| author_sort | Koji Kato |
| collection | DOAJ |
| description | Objective: The prognosis of adrenoleukodystrophy (ALD)with neurological involvement is generally dismal; however, allogeneic stem cell transplantation (SCT) is recognized as effective to stabilize or improve the clinical symptoms of ALD. Herein, we report the clinical outcomes of patients with ALD who consecutively underwent allogeneic stem cell transplantation with reduced intensity conditioning at our institution. Patients: Sixteen patients with ALD, who were symptomatic (n = 14) or presymptomatic (n = 2), received SCT from 2010 to 2016. The stem cell source was cord blood (n = 14), or bone marrow from a human leukocyte antigen identical sibling (n = 2). The conditioning regimen prior to transplantation was reduced intensity and consisted of fludarabine (125 mg/m2), melphalan (140 mg/m2) and low dose total body irradiation (TBI) of 4Gy (n = 15) or 3Gy (n = 1). Results: Primary engraftment was obtained in 11 patients, and 4 of the 5 patients who lost the primary graft received a second cord blood transplantation and were engrafted. Five years overall and event-free survival were 90.9% and 61.1% respectively, with a median of 45 months (range 16–91). Loes score stabilized or improved by 18 months after transplantation except for patients with internal capsule involvement. Conclusion: Allogeneic SCT with reduced intensity conditioning for patients with ALD was safely performed without major transplant-related complications even in symptomatic patients and neurological symptoms were stabilized after SCT in patients without internal capsule involvement. Keywords: Adrenoleukodystrophy, Allogeneic stem cell transplantation, Loes score, Very long chain fatty acid |
| first_indexed | 2024-12-13T16:36:48Z |
| format | Article |
| id | doaj.art-2bec548df4fc4e8fbd0c4373239d7c0b |
| institution | Directory Open Access Journal |
| issn | 2214-4269 |
| language | English |
| last_indexed | 2024-12-13T16:36:48Z |
| publishDate | 2019-03-01 |
| publisher | Elsevier |
| record_format | Article |
| series | Molecular Genetics and Metabolism Reports |
| spelling | doaj.art-2bec548df4fc4e8fbd0c4373239d7c0b2022-12-21T23:38:22ZengElsevierMolecular Genetics and Metabolism Reports2214-42692019-03-011816Allogeneic stem cell transplantation with reduced intensity conditioning for patients with adrenoleukodystrophyKoji Kato0Ryo Maemura1Manabu Wakamatsu2Ayako Yamamori3Motoharu Hamada4Shinsuke Kataoka5Atsushi Narita6Shunsuke Miwata7Yuko Sekiya8Nozomu Kawashima9Kyogo Suzuki10Kotaro Narita11Sayoko Doisaki12Hideki Muramatsu13Hirotoshi Sakaguchi14Kimikazu Matsumoto15Yuka Koike16Osamu Onodera17Makiko Kaga18Nobuyuki Shimozawa19Nao Yoshida20Department of Hematology and Oncology, Children' Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, Japan; Corresponding author at: Department of Hematology and Oncology, Children' Medical Center, Japanese Red Cross Nagoya First Hospital, 3-35, Michishita-cho, Nakamura-ku, Nagoya 453-8511, Japan.Department of Hematology and Oncology, Children' Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, JapanDepartment of Hematology and Oncology, Children' Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, JapanDepartment of Hematology and Oncology, Children' Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, JapanDepartment of Hematology and Oncology, Children' Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, JapanDepartment of Hematology and Oncology, Children' Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, JapanDepartment of Hematology and Oncology, Children' Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, JapanDepartment of Hematology and Oncology, Children' Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, JapanDepartment of Hematology and Oncology, Children' Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, JapanDepartment of Hematology and Oncology, Children' Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, JapanDepartment of Hematology and Oncology, Children' Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, JapanDepartment of Hematology and Oncology, Children' Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, JapanDepartment of Hematology and Oncology, Children' Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, JapanDepartment of Hematology and Oncology, Children' Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, JapanDepartment of Hematology and Oncology, Children' Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, JapanDepartment of Hematology and Oncology, Children' Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, JapanDepartment of Neurology, Clinical Neuroscience Branch, Brain Research Institute, Niigata University, Niigata, JapanDepartment of Neurology, Clinical Neuroscience Branch, Brain Research Institute, Niigata University, Niigata, JapanDepartment of Developmental Disorders, National Center of Neurology and Psychiatry, National Institute of Mental Health, Tokyo, JapanDivision of Genomics Research, Life Science Research Center, Gifu University, Gifu, JapanDepartment of Hematology and Oncology, Children' Medical Center, Japanese Red Cross Nagoya First Hospital, Nagoya, JapanObjective: The prognosis of adrenoleukodystrophy (ALD)with neurological involvement is generally dismal; however, allogeneic stem cell transplantation (SCT) is recognized as effective to stabilize or improve the clinical symptoms of ALD. Herein, we report the clinical outcomes of patients with ALD who consecutively underwent allogeneic stem cell transplantation with reduced intensity conditioning at our institution. Patients: Sixteen patients with ALD, who were symptomatic (n = 14) or presymptomatic (n = 2), received SCT from 2010 to 2016. The stem cell source was cord blood (n = 14), or bone marrow from a human leukocyte antigen identical sibling (n = 2). The conditioning regimen prior to transplantation was reduced intensity and consisted of fludarabine (125 mg/m2), melphalan (140 mg/m2) and low dose total body irradiation (TBI) of 4Gy (n = 15) or 3Gy (n = 1). Results: Primary engraftment was obtained in 11 patients, and 4 of the 5 patients who lost the primary graft received a second cord blood transplantation and were engrafted. Five years overall and event-free survival were 90.9% and 61.1% respectively, with a median of 45 months (range 16–91). Loes score stabilized or improved by 18 months after transplantation except for patients with internal capsule involvement. Conclusion: Allogeneic SCT with reduced intensity conditioning for patients with ALD was safely performed without major transplant-related complications even in symptomatic patients and neurological symptoms were stabilized after SCT in patients without internal capsule involvement. Keywords: Adrenoleukodystrophy, Allogeneic stem cell transplantation, Loes score, Very long chain fatty acidhttp://www.sciencedirect.com/science/article/pii/S2214426918300570 |
| spellingShingle | Koji Kato Ryo Maemura Manabu Wakamatsu Ayako Yamamori Motoharu Hamada Shinsuke Kataoka Atsushi Narita Shunsuke Miwata Yuko Sekiya Nozomu Kawashima Kyogo Suzuki Kotaro Narita Sayoko Doisaki Hideki Muramatsu Hirotoshi Sakaguchi Kimikazu Matsumoto Yuka Koike Osamu Onodera Makiko Kaga Nobuyuki Shimozawa Nao Yoshida Allogeneic stem cell transplantation with reduced intensity conditioning for patients with adrenoleukodystrophy Molecular Genetics and Metabolism Reports |
| title | Allogeneic stem cell transplantation with reduced intensity conditioning for patients with adrenoleukodystrophy |
| title_full | Allogeneic stem cell transplantation with reduced intensity conditioning for patients with adrenoleukodystrophy |
| title_fullStr | Allogeneic stem cell transplantation with reduced intensity conditioning for patients with adrenoleukodystrophy |
| title_full_unstemmed | Allogeneic stem cell transplantation with reduced intensity conditioning for patients with adrenoleukodystrophy |
| title_short | Allogeneic stem cell transplantation with reduced intensity conditioning for patients with adrenoleukodystrophy |
| title_sort | allogeneic stem cell transplantation with reduced intensity conditioning for patients with adrenoleukodystrophy |
| url | http://www.sciencedirect.com/science/article/pii/S2214426918300570 |
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