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Pediatric Hematology and Oncology Volume 41, 2024 - Issue 4
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Letter to the Editor

Successful haploidentical bone marrow transplantation in Griscelli syndrome type 2 with non-busulfan-based regimen and post-transplantation cyclophosphamide: a case report and review of the literature

Saori Yamadaa Department of Pediatrics, Shinshu University School of Medicine, Matsumoto, JapanView further author information
,
Yuta Maruyamaa Department of Pediatrics, Shinshu University School of Medicine, Matsumoto, JapanView further author information
,
Shoji Saitoa Department of Pediatrics, Shinshu University School of Medicine, Matsumoto, JapanCorrespondence[email protected]
ORCID Iconhttps://orcid.org/0000-0003-3866-3582View further author information
ORCID Icon,
Kazutoshi Komorib Department of Hematology and Oncology, Nagano Children’s Hospital, Azumino, JapanView further author information
,
Hirokazu Morokawaa Department of Pediatrics, Shinshu University School of Medicine, Matsumoto, Japan;b Department of Hematology and Oncology, Nagano Children’s Hospital, Azumino, JapanView further author information
,
Eri Okuraa Department of Pediatrics, Shinshu University School of Medicine, Matsumoto, JapanView further author information
,
Koichi Hirabayashia Department of Pediatrics, Shinshu University School of Medicine, Matsumoto, JapanORCID Iconhttps://orcid.org/0000-0002-2193-1184View further author information
ORCID Icon,
Yu Furuib Department of Hematology and Oncology, Nagano Children’s Hospital, Azumino, JapanView further author information
,
Takashi Kuratab Department of Hematology and Oncology, Nagano Children’s Hospital, Azumino, JapanView further author information
,
Makoto Nishiokaa Department of Pediatrics, Shinshu University School of Medicine, Matsumoto, JapanView further author information
,
Tetsuhiro Fukuyamaa Department of Pediatrics, Shinshu University School of Medicine, Matsumoto, JapanView further author information
,
Kazuo Sakashitab Department of Hematology and Oncology, Nagano Children’s Hospital, Azumino, JapanView further author information
&
Yozo Nakazawaa Department of Pediatrics, Shinshu University School of Medicine, Matsumoto, JapanORCID Iconhttps://orcid.org/0000-0003-0793-815XView further author information
ORCID Icon show all
Pages 296-300 | Received 02 Jun 2023, Accepted 17 Oct 2023, Published online: 31 Oct 2023

References

  • Pachlopnik Schmid J, Moshous D, Boddaert N, et al. Hematopoietic stem cell transplantation in Griscelli syndrome type 2: a single-center report on 10 patients. Blood. Jul 2 2009;114(1):211–218. doi:10.1182/blood-2009-02-207845.
  • Al-Mofareh M, Ayas M, Al-Seraihy A, et al. Hematopoietic stem cell transplantation in children with Griscelli syndrome type 2: a single-center report on 35 patients. Bone Marrow Transplant. Oct 2020;55(10):2026–2034. doi:10.1038/s41409-020-0885-6.
  • Zhang Q, Zhao YZ, Ma HH, et al. Successful rescue of a lethal Griscelli syndrome type 2 presenting with neurological involvement and hemophagocytic lymphohistiocytosis: a case report. BMC Pediatr. May 31 2021;21(1):253. doi:10.1186/s12887-021-02720-1.
  • Nishi M, Nishimura R, Suzuki N, et al. Reduced-intensity conditioning in unrelated donor cord blood transplantation for familial hemophagocytic lymphohistiocytosis. Am J Hematol. Jun 2012;87(6):637–639. doi:10.1002/ajh.23190.
  • Marsh RA, Hebert K, Kim S, et al. Comparison of hematopoietic cell transplant conditioning regimens for hemophagocytic lymphohistiocytosis disorders. J Allergy Clin Immunol. Mar 2022;149(3):1097–1104.e2. doi:10.1016/j.jaci.2021.07.031.
  • Hamidieh AA, Pourpak Z, Yari K, et al. Hematopoietic stem cell transplantation with a reduced-intensity conditioning regimen in pediatric patients with Griscelli syndrome type 2. Pediatr Transplant. Aug 2013;17(5):487–491. doi:10.1111/petr.12092.
  • Patiroglu T, Akar HH, Unal E, Ozdemir MA, Karakukcu M. Hematopoietic Stem Cell Transplant for Primary Immunodeficiency Diseases: A Single-Center Experience. Exp Clin Transplant. Jun 2017;15(3):337–343. doi:10.6002/ect.2015.0233.
  • Uppuluri R, Sivasankaran M, Patel S, et al. Haploidentical Stem Cell Transplantation with Post-Transplant Cyclophosphamide for Primary Immune Deficiency Disorders in Children: Challenges and Outcome from a Tertiary Care Center in South India. J Clin Immunol. Feb 2019;39(2):182–187. doi:10.1007/s10875-019-00600-z.
  • Swaminathan VV, Uppuluri R, Meena SK, et al. Treosulfan-Based Conditioning in Matched Family, Unrelated and Haploidentical Hematopoietic Stem Cell Transplantation for Genetic Hemophagocytic Lymphohistiocytosis: Experience and Outcomes over 10 Years from India. Indian J Hematol Blood Transfus. Jan 2022;38(1):84–91. doi:10.1007/s12288-021-01422-z.
  • Felber M, Steward CG, Kentouche K, et al. Targeted busulfan-based reduced-intensity conditioning and HLA-matched HSCT cure hemophagocytic lymphohistiocytosis. Blood Adv. 2020;4(9):1998–2010. Blood Adv. Aug 25 2020;4(16):3926. doi:10.1182/bloodadvances.2020003085.
  • Vrooman LM, Millard HR, Brazauskas R, et al. Survival and Late Effects after Allogeneic Hematopoietic Cell Transplantation for Hematologic Malignancy at Less than Three Years of Age. Biol Blood Marrow Transplant. Aug 2017;23(8):1327–1334. doi:10.1016/j.bbmt.2017.04.017.

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