Clinical outcome of cord blood transplantation for nine children with juvenile myelomonocytic leukemia receiving fludarabine-busulfan-cyclophosphamide-based conditioning
Guifang Li
Division of Life Sciences and Medicine, Department of Geriatrics, The First Affiliated Hospital of USTC, University of Science and Technology of China, Hefei, China
Division of Life Sciences and Medicine, Department of Hematology, The First Affiliated Hospital of USTC, University of Science and Technology of China, Hefei, China
Search for more papers by this authorZimin Sun
Division of Life Sciences and Medicine, Department of Hematology, The First Affiliated Hospital of USTC, University of Science and Technology of China, Hefei, China
Division of Life Sciences and Medicine, Blood and Cell Therapy Institute, University of Science and Technology of China, Hefei, China
Search for more papers by this authorLiangquan Geng
Division of Life Sciences and Medicine, Department of Hematology, The First Affiliated Hospital of USTC, University of Science and Technology of China, Hefei, China
Search for more papers by this authorXiang Wan
Division of Life Sciences and Medicine, Department of Hematology, The First Affiliated Hospital of USTC, University of Science and Technology of China, Hefei, China
Search for more papers by this authorXiaoyu Zhu
Division of Life Sciences and Medicine, Department of Hematology, The First Affiliated Hospital of USTC, University of Science and Technology of China, Hefei, China
Division of Life Sciences and Medicine, Blood and Cell Therapy Institute, University of Science and Technology of China, Hefei, China
Search for more papers by this authorBaolin Tang
Division of Life Sciences and Medicine, Department of Hematology, The First Affiliated Hospital of USTC, University of Science and Technology of China, Hefei, China
Search for more papers by this authorJuan Tong
Division of Life Sciences and Medicine, Department of Hematology, The First Affiliated Hospital of USTC, University of Science and Technology of China, Hefei, China
Search for more papers by this authorWen Yao
Division of Life Sciences and Medicine, Department of Hematology, The First Affiliated Hospital of USTC, University of Science and Technology of China, Hefei, China
Search for more papers by this authorKaidi Song
Division of Life Sciences and Medicine, Department of Hematology, The First Affiliated Hospital of USTC, University of Science and Technology of China, Hefei, China
Search for more papers by this authorPing Qiang
Division of Life Sciences and Medicine, Department of Hematology, The First Affiliated Hospital of USTC, University of Science and Technology of China, Hefei, China
Search for more papers by this authorLei Zhang
Division of Life Sciences and Medicine, Department of Hematology, The First Affiliated Hospital of USTC, University of Science and Technology of China, Hefei, China
Search for more papers by this authorXuhan Zhang
Division of Life Sciences and Medicine, Department of Hematology, The First Affiliated Hospital of USTC, University of Science and Technology of China, Hefei, China
Search for more papers by this authorShiyang Zhang
Division of Life Sciences and Medicine, Department of Geriatrics, The First Affiliated Hospital of USTC, University of Science and Technology of China, Hefei, China
Search for more papers by this authorCorresponding Author
Huilan Liu
Division of Life Sciences and Medicine, Department of Hematology, The First Affiliated Hospital of USTC, University of Science and Technology of China, Hefei, China
Division of Life Sciences and Medicine, Blood and Cell Therapy Institute, University of Science and Technology of China, Hefei, China
Correspondence
Huilan Liu, Division of Life Sciences and Medicine, Department of Hematology, The First Affiliated Hospital of USTC, University of Science and Technology of China, 17 Lujiang Road, Hefei, Anhui, 230001, China.
Email: [email protected]
Search for more papers by this authorGuifang Li
Division of Life Sciences and Medicine, Department of Geriatrics, The First Affiliated Hospital of USTC, University of Science and Technology of China, Hefei, China
Division of Life Sciences and Medicine, Department of Hematology, The First Affiliated Hospital of USTC, University of Science and Technology of China, Hefei, China
Search for more papers by this authorZimin Sun
Division of Life Sciences and Medicine, Department of Hematology, The First Affiliated Hospital of USTC, University of Science and Technology of China, Hefei, China
Division of Life Sciences and Medicine, Blood and Cell Therapy Institute, University of Science and Technology of China, Hefei, China
Search for more papers by this authorLiangquan Geng
Division of Life Sciences and Medicine, Department of Hematology, The First Affiliated Hospital of USTC, University of Science and Technology of China, Hefei, China
Search for more papers by this authorXiang Wan
Division of Life Sciences and Medicine, Department of Hematology, The First Affiliated Hospital of USTC, University of Science and Technology of China, Hefei, China
Search for more papers by this authorXiaoyu Zhu
Division of Life Sciences and Medicine, Department of Hematology, The First Affiliated Hospital of USTC, University of Science and Technology of China, Hefei, China
Division of Life Sciences and Medicine, Blood and Cell Therapy Institute, University of Science and Technology of China, Hefei, China
Search for more papers by this authorBaolin Tang
Division of Life Sciences and Medicine, Department of Hematology, The First Affiliated Hospital of USTC, University of Science and Technology of China, Hefei, China
Search for more papers by this authorJuan Tong
Division of Life Sciences and Medicine, Department of Hematology, The First Affiliated Hospital of USTC, University of Science and Technology of China, Hefei, China
Search for more papers by this authorWen Yao
Division of Life Sciences and Medicine, Department of Hematology, The First Affiliated Hospital of USTC, University of Science and Technology of China, Hefei, China
Search for more papers by this authorKaidi Song
Division of Life Sciences and Medicine, Department of Hematology, The First Affiliated Hospital of USTC, University of Science and Technology of China, Hefei, China
Search for more papers by this authorPing Qiang
Division of Life Sciences and Medicine, Department of Hematology, The First Affiliated Hospital of USTC, University of Science and Technology of China, Hefei, China
Search for more papers by this authorLei Zhang
Division of Life Sciences and Medicine, Department of Hematology, The First Affiliated Hospital of USTC, University of Science and Technology of China, Hefei, China
Search for more papers by this authorXuhan Zhang
Division of Life Sciences and Medicine, Department of Hematology, The First Affiliated Hospital of USTC, University of Science and Technology of China, Hefei, China
Search for more papers by this authorShiyang Zhang
Division of Life Sciences and Medicine, Department of Geriatrics, The First Affiliated Hospital of USTC, University of Science and Technology of China, Hefei, China
Search for more papers by this authorCorresponding Author
Huilan Liu
Division of Life Sciences and Medicine, Department of Hematology, The First Affiliated Hospital of USTC, University of Science and Technology of China, Hefei, China
Division of Life Sciences and Medicine, Blood and Cell Therapy Institute, University of Science and Technology of China, Hefei, China
Correspondence
Huilan Liu, Division of Life Sciences and Medicine, Department of Hematology, The First Affiliated Hospital of USTC, University of Science and Technology of China, 17 Lujiang Road, Hefei, Anhui, 230001, China.
Email: [email protected]
Search for more papers by this authorAbstract
Background
Juvenile myelomonocytic leukemia (JMML) is a rare hematological malignancy in young children and can only be cured through the allogeneic stem cell transplantation.
Procedure
We have retrospectively analyzed the outcomes of nine children with JMML after unrelated cord blood transplantation (UCBT).
Results
Eight patients who have received a myeloablative conditioning regimen of fludarabine (FLU), busulfan (BU), and cyclophosphamide (CY) have gotten engraftment. None of the nine patients has relapsed following initial UCBT. Six patients are still alive and in complete remission after UCBT with a median observation time of 43 months (range: 10–80 months).
Conclusions
This study shows that UCBT with FLU-BU-CY conditioning regimen can represent a suitable option for children with JMML.
CONFLICT OF INTEREST
No potential conflicts of interest were disclosed.
Open Research
DATA AVAILABILITY STATEMENT
There is no data availability statement.
REFERENCES
- 1Arber DA, Orazi A, Hasserjian R, et al. The 2016 revision to the World Health Organization classification of myeloid neoplasms and acute leukemia. Blood. 2016; 127(20): 2391-2405.
- 2Emanuel PD, Bates LJ, Castleberry RP, et al. Selective hypersensitivity to granulocyte-macrophage conony-stimulating factor by juvenile chronic myeloid leukemia hematopoietic progenitors. Blood. 1991; 77(5): 925-929.
- 3Niemeyer CM, Arico M, Basso G, et al. Chronic myelomonocytic leukemia in childhood: a retrospective analysis of 110 cases European Working Group on Myelodysplastic Syndromes in Childhood (EWOG-MDS). Blood. 1997; 89(10): 3534-3543.
- 4Niemeyer CM, Flotho C. Juvenile myelomonocytic leukemia: who’s the driver at the wheel? Blood. 2019; 133(10): 1060-1070.
- 5Niemeyer CM. JMML genomics and decisions. Am Soc Hematol. 2018; 1: 307-312.
- 6Locatelli F, Crotta A, Ruggeri A, et al. Analysis of risk factors influencing outcomes after cord blood transplantation in children with juvenile myelomonocytic leukemia: a EUROCORD, EBMT, EWOG-MDS, CIBMTR study. Blood. 2013; 122(12): 2135-2141.
- 7Yoshida N, Sakaguchi H, Yabe M, et al. Clinical outcomes after allogeneic hematopoietic stem cell transplantation in children with juvrnile myelomonocytic leukemia: a report from the Japan Society for Hematopoietic Cell Transplantation. Biol Blood Marrow Transplant. 2020; 26(5): 902-910.
- 8Milano F, Gooley T, Wood BL, et al. Cord-blood transplantation in patients with minimal residual disease. N Engl J Med. 2016; 375(10): 944-953.
- 9Madureira AB, Eapen M, Locatelli F, et al. Analysis of risk factors influencing outcome in children with myelodysplastic syndrome after unrelated cord blood transplantation. Leukemia. 2011; 25(3): 449-454.
- 10Sharma P, Purev E, Haverkos B, et al. Adult cord blood transplant results in comparable overall survival and improved GRFS vs matched related transplant. Blood. 2020; 4(10): 2227-2235.
- 11Eapen M, Klein JP, Ruggeri A, et al. Impact of allele level HLA matching on outcomes after myeloablative single unit umbilical cord blood transplantation for hematologic malignancy. Blood. 2014; 123(1): 133-140.
- 12Locatelli F, Nollke P, Zecca M, et al. Hematopoietic stem cell transplantation (HSCT) in children with juvenile myelomonocytic leukemia(JMML): results of the EWOG-MDS/EBMT trial. Blood. 2005; 105(1): 410-419.
- 13Rondon G, Saliba RM, Khouri I, et al. Long-term follow-up of patients who experienced graft failure post allogeneic progenitor cell transplantation. Results of single institute on analysis. Bio Blood Marrow Transplant. 2008; 14(8): 859-866.
- 14Przepiorka D, Weisdorf D, Martin P, et al. 1994 consensus conference on acute GVHD grading. Bone Marrow Transplant. 1995; 15(6): 825-828.
- 15Filipovich AH, Weisdorf D, Pavletic S, et al. National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus- host disease: I. Diagnosis and staging working group report. Biol Blood Marrow Transplant. 2005; 11(12): 945-956.
- 16Patel KJ, Rice RD, Hawke R, et al. Pre-engraftment syndrome after double-unit cord blood transplantation: a distinct syndrome not associated with acute graft-versus-host disease. Biol Blood Marrow Transplant. 2010; 16(3): 435-440.
- 17Eapen M, Rubinstein P, Zhang MJ, et al. Outcomes of transplantation of unrelated donor umbilical cord blood and bone marrow in children with acute leukemia: a comparison study. Lancet. 2007; 369(9577): 1947-1954.
- 18Bizzetto R, Bonfim C, Rocha V, et al. Outcomes after related and unrelted umbilical cord blood transplantation for hereditary bone marrow failure syndromes other than Fanconi anemia. Haematoligica. 2011; 96(1): 134-141.
- 19Yabe M, Ohtsuka Y, Watanabe K, et al. Transplantation for juvenile myelomonocytic leukemia: a retrospective study of 30 children treated with a regimen of busulfan, fludarabine, and melphalan. Int J Hematol. 2015; 101(2): 184-190.
- 20Giralt S, Thall PF, Khouri I, et al. Melphalan and purine analog-containing preparative regimens: reduced-intensity conditioning for patients with hematologic malignancies undergoing allogeneic progenitor cell transplantation. Blood. 2001; 97(3): 631-637.
- 21Barker JN, Weisdorf DJ, DeFor TE, et al. Rapid and complete donor chimerism in adult recipients of unrelated donor umbilical cord blood transplantation after reduced-intensity conditioning. Blood. 2003; 102(5): 1915-1919.
- 22Chan KW, Bekassy AN, Ha CS, et al. Fludarabine-based preparative protocol for unrelated donor cord blood transplantation in children: successful engraftment with minimal toxicity. Bone Marrow Transplant. 1999; 23(8): 849-851.
- 23Locatelli F, Niemeyer C, Angelucci E, et al. Allogeneic bone marrow transplantation for chronic myelomonocytic leukemia in childhood: a report from the European Working Group on Myelodysplastic Syndrome in Childhood. J Clin Oncol. 1997; 15(2): 566-573.
- 24Manabe A, Okamura J, Yumura-Yagi K, et al. Allogeneic hematopoietic stem cell transplantation for 27 children with juvenile myelomonocytic leukemia diagnosed based on the criteria of the International JMML Working Group. Leukemia. 2002; 16(4): 645-649.
- 25Isobe M, Konuma T, Kato S, et al. Development of pre-engraftment syndrome, but not acute graft-versus-host disease, reduces relapse rate of acute myelogenous leukemia after single cord blood transplantation. Biol Blood Marrow Transplant. 2019; 25(6): 1187-1196.