Unmanipulated haploidentical hematopoietic stem cell transplantation for pediatric de novo acute megakaryoblastic leukemia without Down syndrome in China: A single-center study
BackgroundAMKL without DS is a rare but aggressive hematological malignant disease in children, and it is associated with inferior outcomes. Several researchers have regarded pediatric AMKL without DS as high-risk or at least intermediate-risk AML and proposed that upfront allogenic hematopoietic st...
| Main Authors: | , , , , , , , , , , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Frontiers Media S.A.
2023-02-01
|
| Series: | Frontiers in Oncology |
| Subjects: | |
| Online Access: | https://www.frontiersin.org/articles/10.3389/fonc.2023.1116205/full |
| _version_ | 1828014329257000960 |
|---|---|
| author | Junbin Huang Guanhua Hu Pan Suo Lu Bai Yifei Cheng Yu Wang XiaoHui Zhang KaiYan Liu YuQian Sun LanPing Xu Jun Kong ChenHua Yan Xiaojun Huang |
| author_facet | Junbin Huang Guanhua Hu Pan Suo Lu Bai Yifei Cheng Yu Wang XiaoHui Zhang KaiYan Liu YuQian Sun LanPing Xu Jun Kong ChenHua Yan Xiaojun Huang |
| author_sort | Junbin Huang |
| collection | DOAJ |
| description | BackgroundAMKL without DS is a rare but aggressive hematological malignant disease in children, and it is associated with inferior outcomes. Several researchers have regarded pediatric AMKL without DS as high-risk or at least intermediate-risk AML and proposed that upfront allogenic hematopoietic stem cell transplantation (HSCT) in first complete remission might improve long-term survival.Patients and methodWe conducted a retrospective study with twenty-five pediatric (< 14 years old) AMKL patients without DS who underwent haploidentical HSCT in the Peking University Institute of Hematology, Peking University People’s Hospital from July 2016 to July 2021. The diagnostic criteria of AMKL without DS were adapted from the FAB and WHO: ≥ 20% blasts in the bone marrow, and those blasts expressed at least one or more of the platelet glycoproteins: CD41, CD61, or CD42. AMKL with DS and therapy related AML was excluded. Children without a suitable closely HLA-matched related or unrelated donor (donors with more than nine out of 10 matching HLA-A, HLA-B, HLA-C, HLA-DR, and HLA-DQ loci), were eligible to receive haploidentical HSCT. Definition was adapted from international cooperation group. All statistical tests were conducted with SPSS v.24 and R v.3.6.3.ResultsThe 2-year OS was 54.5 ± 10.3%, and the EFS was 50.9 ± 10.2% in pediatric AMKL without DS undergoing haplo-HSCT. Statistically significantly better EFS was observed in patients with trisomy 19 than in patients without trisomy 19 (80 ± 12.6% and 33.3 ± 12.2%, respectively, P = 0.045), and OS was better in patients with trisomy 19 but with no statistical significance (P = 0.114). MRD negative pre-HSCT patients showed a better OS and EFS than those who were positive (P < 0.001 and P = 0.003, respectively). Eleven patients relapsed post HSCT. The median time to relapse post HSCT was 2.1 months (range: 1.0–14.4 months). The 2-year cumulative incidence of relapse (CIR) was 46.1 ± 11.6%. One patient developed bronchiolitis obliterans and respiratory failure and died at d + 98 post HSCT.ConclusionAMKL without DS is a rare but aggressive hematological malignant disease in children, and it is associated with inferior outcomes. Trisomy 19 and MRD negative pre-HSCT might contribute to a better EFS and OS. Our TRM was low, haplo-HSCT might be an option for high-risk AMKL without DS. |
| first_indexed | 2024-04-10T09:59:15Z |
| format | Article |
| id | doaj.art-c9ccd54fdc7f41d6807e88d272330523 |
| institution | Directory Open Access Journal |
| issn | 2234-943X |
| language | English |
| last_indexed | 2024-04-10T09:59:15Z |
| publishDate | 2023-02-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Oncology |
| spelling | doaj.art-c9ccd54fdc7f41d6807e88d2723305232023-02-16T10:53:26ZengFrontiers Media S.A.Frontiers in Oncology2234-943X2023-02-011310.3389/fonc.2023.11162051116205Unmanipulated haploidentical hematopoietic stem cell transplantation for pediatric de novo acute megakaryoblastic leukemia without Down syndrome in China: A single-center studyJunbin Huang0Guanhua Hu1Pan Suo2Lu Bai3Yifei Cheng4Yu Wang5XiaoHui Zhang6KaiYan Liu7YuQian Sun8LanPing Xu9Jun Kong10ChenHua Yan11Xiaojun Huang12Division of Hematology/Oncology, Department of Pediatrics, The Seventh Affiliated Hospital, Sun Yat-sen University, Shenzhen, ChinaBeijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Peking-Tsinghua Center for Life Science, Research Unit of Key Technique for Diagnosis and Treatment of Hematologic Malignancies, National Clinical Research Center for Hematologic Disease, Peking University People’s Hospital, Peking University Institute of Hematology, Beijing, ChinaBeijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Peking-Tsinghua Center for Life Science, Research Unit of Key Technique for Diagnosis and Treatment of Hematologic Malignancies, National Clinical Research Center for Hematologic Disease, Peking University People’s Hospital, Peking University Institute of Hematology, Beijing, ChinaBeijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Peking-Tsinghua Center for Life Science, Research Unit of Key Technique for Diagnosis and Treatment of Hematologic Malignancies, National Clinical Research Center for Hematologic Disease, Peking University People’s Hospital, Peking University Institute of Hematology, Beijing, ChinaBeijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Peking-Tsinghua Center for Life Science, Research Unit of Key Technique for Diagnosis and Treatment of Hematologic Malignancies, National Clinical Research Center for Hematologic Disease, Peking University People’s Hospital, Peking University Institute of Hematology, Beijing, ChinaBeijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Peking-Tsinghua Center for Life Science, Research Unit of Key Technique for Diagnosis and Treatment of Hematologic Malignancies, National Clinical Research Center for Hematologic Disease, Peking University People’s Hospital, Peking University Institute of Hematology, Beijing, ChinaBeijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Peking-Tsinghua Center for Life Science, Research Unit of Key Technique for Diagnosis and Treatment of Hematologic Malignancies, National Clinical Research Center for Hematologic Disease, Peking University People’s Hospital, Peking University Institute of Hematology, Beijing, ChinaBeijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Peking-Tsinghua Center for Life Science, Research Unit of Key Technique for Diagnosis and Treatment of Hematologic Malignancies, National Clinical Research Center for Hematologic Disease, Peking University People’s Hospital, Peking University Institute of Hematology, Beijing, ChinaBeijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Peking-Tsinghua Center for Life Science, Research Unit of Key Technique for Diagnosis and Treatment of Hematologic Malignancies, National Clinical Research Center for Hematologic Disease, Peking University People’s Hospital, Peking University Institute of Hematology, Beijing, ChinaBeijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Peking-Tsinghua Center for Life Science, Research Unit of Key Technique for Diagnosis and Treatment of Hematologic Malignancies, National Clinical Research Center for Hematologic Disease, Peking University People’s Hospital, Peking University Institute of Hematology, Beijing, ChinaBeijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Peking-Tsinghua Center for Life Science, Research Unit of Key Technique for Diagnosis and Treatment of Hematologic Malignancies, National Clinical Research Center for Hematologic Disease, Peking University People’s Hospital, Peking University Institute of Hematology, Beijing, ChinaBeijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Peking-Tsinghua Center for Life Science, Research Unit of Key Technique for Diagnosis and Treatment of Hematologic Malignancies, National Clinical Research Center for Hematologic Disease, Peking University People’s Hospital, Peking University Institute of Hematology, Beijing, ChinaBeijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Peking-Tsinghua Center for Life Science, Research Unit of Key Technique for Diagnosis and Treatment of Hematologic Malignancies, National Clinical Research Center for Hematologic Disease, Peking University People’s Hospital, Peking University Institute of Hematology, Beijing, ChinaBackgroundAMKL without DS is a rare but aggressive hematological malignant disease in children, and it is associated with inferior outcomes. Several researchers have regarded pediatric AMKL without DS as high-risk or at least intermediate-risk AML and proposed that upfront allogenic hematopoietic stem cell transplantation (HSCT) in first complete remission might improve long-term survival.Patients and methodWe conducted a retrospective study with twenty-five pediatric (< 14 years old) AMKL patients without DS who underwent haploidentical HSCT in the Peking University Institute of Hematology, Peking University People’s Hospital from July 2016 to July 2021. The diagnostic criteria of AMKL without DS were adapted from the FAB and WHO: ≥ 20% blasts in the bone marrow, and those blasts expressed at least one or more of the platelet glycoproteins: CD41, CD61, or CD42. AMKL with DS and therapy related AML was excluded. Children without a suitable closely HLA-matched related or unrelated donor (donors with more than nine out of 10 matching HLA-A, HLA-B, HLA-C, HLA-DR, and HLA-DQ loci), were eligible to receive haploidentical HSCT. Definition was adapted from international cooperation group. All statistical tests were conducted with SPSS v.24 and R v.3.6.3.ResultsThe 2-year OS was 54.5 ± 10.3%, and the EFS was 50.9 ± 10.2% in pediatric AMKL without DS undergoing haplo-HSCT. Statistically significantly better EFS was observed in patients with trisomy 19 than in patients without trisomy 19 (80 ± 12.6% and 33.3 ± 12.2%, respectively, P = 0.045), and OS was better in patients with trisomy 19 but with no statistical significance (P = 0.114). MRD negative pre-HSCT patients showed a better OS and EFS than those who were positive (P < 0.001 and P = 0.003, respectively). Eleven patients relapsed post HSCT. The median time to relapse post HSCT was 2.1 months (range: 1.0–14.4 months). The 2-year cumulative incidence of relapse (CIR) was 46.1 ± 11.6%. One patient developed bronchiolitis obliterans and respiratory failure and died at d + 98 post HSCT.ConclusionAMKL without DS is a rare but aggressive hematological malignant disease in children, and it is associated with inferior outcomes. Trisomy 19 and MRD negative pre-HSCT might contribute to a better EFS and OS. Our TRM was low, haplo-HSCT might be an option for high-risk AMKL without DS.https://www.frontiersin.org/articles/10.3389/fonc.2023.1116205/fullhaploidenticalhematopoietic stem cell transplantationpediatricacute megakaryoblastic leukemiade novo |
| spellingShingle | Junbin Huang Guanhua Hu Pan Suo Lu Bai Yifei Cheng Yu Wang XiaoHui Zhang KaiYan Liu YuQian Sun LanPing Xu Jun Kong ChenHua Yan Xiaojun Huang Unmanipulated haploidentical hematopoietic stem cell transplantation for pediatric de novo acute megakaryoblastic leukemia without Down syndrome in China: A single-center study Frontiers in Oncology haploidentical hematopoietic stem cell transplantation pediatric acute megakaryoblastic leukemia de novo |
| title | Unmanipulated haploidentical hematopoietic stem cell transplantation for pediatric de novo acute megakaryoblastic leukemia without Down syndrome in China: A single-center study |
| title_full | Unmanipulated haploidentical hematopoietic stem cell transplantation for pediatric de novo acute megakaryoblastic leukemia without Down syndrome in China: A single-center study |
| title_fullStr | Unmanipulated haploidentical hematopoietic stem cell transplantation for pediatric de novo acute megakaryoblastic leukemia without Down syndrome in China: A single-center study |
| title_full_unstemmed | Unmanipulated haploidentical hematopoietic stem cell transplantation for pediatric de novo acute megakaryoblastic leukemia without Down syndrome in China: A single-center study |
| title_short | Unmanipulated haploidentical hematopoietic stem cell transplantation for pediatric de novo acute megakaryoblastic leukemia without Down syndrome in China: A single-center study |
| title_sort | unmanipulated haploidentical hematopoietic stem cell transplantation for pediatric de novo acute megakaryoblastic leukemia without down syndrome in china a single center study |
| topic | haploidentical hematopoietic stem cell transplantation pediatric acute megakaryoblastic leukemia de novo |
| url | https://www.frontiersin.org/articles/10.3389/fonc.2023.1116205/full |
| work_keys_str_mv | AT junbinhuang unmanipulatedhaploidenticalhematopoieticstemcelltransplantationforpediatricdenovoacutemegakaryoblasticleukemiawithoutdownsyndromeinchinaasinglecenterstudy AT guanhuahu unmanipulatedhaploidenticalhematopoieticstemcelltransplantationforpediatricdenovoacutemegakaryoblasticleukemiawithoutdownsyndromeinchinaasinglecenterstudy AT pansuo unmanipulatedhaploidenticalhematopoieticstemcelltransplantationforpediatricdenovoacutemegakaryoblasticleukemiawithoutdownsyndromeinchinaasinglecenterstudy AT lubai unmanipulatedhaploidenticalhematopoieticstemcelltransplantationforpediatricdenovoacutemegakaryoblasticleukemiawithoutdownsyndromeinchinaasinglecenterstudy AT yifeicheng unmanipulatedhaploidenticalhematopoieticstemcelltransplantationforpediatricdenovoacutemegakaryoblasticleukemiawithoutdownsyndromeinchinaasinglecenterstudy AT yuwang unmanipulatedhaploidenticalhematopoieticstemcelltransplantationforpediatricdenovoacutemegakaryoblasticleukemiawithoutdownsyndromeinchinaasinglecenterstudy AT xiaohuizhang unmanipulatedhaploidenticalhematopoieticstemcelltransplantationforpediatricdenovoacutemegakaryoblasticleukemiawithoutdownsyndromeinchinaasinglecenterstudy AT kaiyanliu unmanipulatedhaploidenticalhematopoieticstemcelltransplantationforpediatricdenovoacutemegakaryoblasticleukemiawithoutdownsyndromeinchinaasinglecenterstudy AT yuqiansun unmanipulatedhaploidenticalhematopoieticstemcelltransplantationforpediatricdenovoacutemegakaryoblasticleukemiawithoutdownsyndromeinchinaasinglecenterstudy AT lanpingxu unmanipulatedhaploidenticalhematopoieticstemcelltransplantationforpediatricdenovoacutemegakaryoblasticleukemiawithoutdownsyndromeinchinaasinglecenterstudy AT junkong unmanipulatedhaploidenticalhematopoieticstemcelltransplantationforpediatricdenovoacutemegakaryoblasticleukemiawithoutdownsyndromeinchinaasinglecenterstudy AT chenhuayan unmanipulatedhaploidenticalhematopoieticstemcelltransplantationforpediatricdenovoacutemegakaryoblasticleukemiawithoutdownsyndromeinchinaasinglecenterstudy AT xiaojunhuang unmanipulatedhaploidenticalhematopoieticstemcelltransplantationforpediatricdenovoacutemegakaryoblasticleukemiawithoutdownsyndromeinchinaasinglecenterstudy |