Hematopoietic stem cell transplant therapy, therapy, clinical trials, complications, and quality of life for patients with Sickle cell anemia: Clinical potential and future perspectives

被引:0
|
作者
Verma, Henu Kumar [1 ,6 ]
Ratre, Yashwant Kumar [2 ]
Bhaskar, L. V. K. S. [3 ]
Sahu, Tarun [4 ]
Lingojwar, Devendra Purushottam [5 ]
机构
[1] Helmholtz Zent, Inst lungs Biol & Dis, Comprehens Pneumol Ctr, Dept Immunopathol, D-85764 Munich, Germany
[2] Guru Ghasidas Vishwavidyalay, Dept Biotechnol, Bilaspur, India
[3] Guru Ghasidas Vishwavidyalaya, Dept Zool, Bilalpur, India
[4] AIIMS, Dept Physiol, Raipur, India
[5] Reg Soc Educ & Res Community Hlth, Pune, India
[6] Helmholtz Zent, Comprehens Pneumol Ctr, Inst lungs Biol & Dis, Dept Immunopathol, D-85764 Munich, Germany
关键词
Hemoglobinopathy; Hematopoietic stem-cell transplantation; Quality of Life; Sickle cell Anemia; BONE-MARROW-TRANSPLANTATION; CORD BLOOD TRANSPLANTATION; DISEASE; CHILDREN; THALASSEMIA; HYDROXYUREA; EXPERIENCE; SURVIVAL; DONORS; STATE;
D O I
暂无
中图分类号
R72 [儿科学];
学科分类号
100202 ;
摘要
Background: Sickle cell anemia (SCA) is an inherited monogenic disorder. The clinical symptoms of SCA are protean, including vasoocclusion, hemolysis, early stroke leg ulcers, multi-organ failure, and increased risk of premature death. Hematopoietic stem cell transplantation is the only treatment identified to reduce SCA-related organ damage. Unfortunately, graft rejection is a significant impediment to these strategies. Materials and Methods: The current standard of treatment for the past two decades is limited to myeloablative- matched sibling donors, which is likely to be only for minor patients and is feasible for non-malignant giant disease. Cumulative studies showed that HSCT increases overall survival and quality of life in patients with SCA.<br />Results: Hematopoietic stem cell transplantation (HSCT) is significantly associated with a higher risk of graft versus host disease and moderate mortality risk. New strategy lacking standard donors includes cord blood, matched unrelated donors/Haploidentical donors.<br />Conclusion: This review summarized evidence from HSCT clinical trials from different transplantation methods, specific HSCT and HSCT-related health problems that need to be addressed in medical contexts with patients and family members, and other areas that enhance the quality of life in SCA.
引用
收藏
页码:272 / 299
页数:28
相关论文
共 50 条
  • [1] Access to hematopoietic stem cell transplant for patients with sickle cell anemia
    Meier, Emily Riehm
    Johnson, Theodore
    Pinkney, Kerice
    Velez, Maria C.
    Kamani, Naynesh
    Odame, Isaac
    PEDIATRIC BLOOD & CANCER, 2018, 65 (09)
  • [2] Current attitudes of parents and patients toward hematopoietic stem cell transplantation for sickle cell anemia
    Meier, Emily Riehm
    Dioguardi, Jacqueline V.
    Kamani, Naynesh
    PEDIATRIC BLOOD & CANCER, 2015, 62 (07) : 1277 - 1284
  • [3] Infectious complications in adult sickle cell anemia patients undergoing hematopoietic stem cell transplantation
    Abuelgasim, Khadega A.
    Damlaj, Moussab
    Bosaeed, Mohammad
    Altamimi, Sumayyah
    Abdullah, Hind
    Ramli, Reem M.
    Alzahrani, Rahma
    Alzughaibi, Nourah
    Alahmari, Bader
    Alhejazi, Ayman
    Alaskar, Ahmed
    Alzahrani, Mohsen
    BONE MARROW TRANSPLANTATION, 2021, 56 (12) : 3078 - 3080
  • [4] A systematic review comparing allogeneic hematopoietic stem cell transplant to gene therapy in sickle cell disease
    Rotin, Lianne E. E.
    Viswabandya, Auro
    Kumar, Rajat
    Patriquin, Christopher J. J.
    Kuo, Kevin H. M.
    HEMATOLOGY, 2023, 28 (01)
  • [5] Traffic Light: prognosis-based eligibility for clinical trials of hematopoietic SCT in adults with sickle cell anemia
    Rotz, S. J.
    O'Riordan, M. A.
    Kim, C.
    de Lima, M.
    Gladwin, M. T.
    Little, J. A.
    BONE MARROW TRANSPLANTATION, 2015, 50 (07) : 918 - 923
  • [6] Diamond-Blackfan anemia in Japan: Clinical outcomes of prednisolone therapy and hematopoietic stem cell transplantation
    Ohga, S
    Mugishima, H
    Ohara, A
    Kojima, S
    Fujisawa, K
    Yagi, K
    Higashigawa, M
    Tsukimoto, I
    INTERNATIONAL JOURNAL OF HEMATOLOGY, 2004, 79 (01) : 22 - 30
  • [7] Diamond-Blackfan Anemia in Japan: Clinical Outcomes of Prednisolone Therapy and Hematopoietic Stem Cell Transplantation
    Shouichi Ohga
    Hideo Mugishima
    Akira Ohara
    Seiji Kojima
    Kohji Fujisawa
    Keiko Yagi
    Masamune Higashigawa
    Ichiro Tsukimoto
    International Journal of Hematology, 2004, 79 : 22 - 30
  • [8] Debating the Future of Sickle Cell Disease Curative Therapy: Haploidentical Hematopoietic Stem Cell Transplantation vs. Gene Therapy
    Kassim, Adetola A.
    Leonard, Alexis
    JOURNAL OF CLINICAL MEDICINE, 2022, 11 (16)
  • [9] A systematic review of quality of life in sickle cell disease and thalassemia after stem cell transplant or gene therapy
    Badawy, Sherif M.
    Beg, Usman
    Liem, Robert, I
    Chaudhury, Sonali
    Thompson, Alexis A.
    BLOOD ADVANCES, 2021, 5 (02) : 570 - 583
  • [10] Quality of Life of Sickle Cell Disease Patients After Hematopoietic Stem Cell Transplantation: a Longitudinal Study
    dos Santos Lotério L.
    de Oliveira-Cardoso É.A.
    Simões B.P.
    de Oliveira M.C.
    Garcia J.T.
    Guimarães A.L.C.
    Pereira K.C.
    Costa T.C.M.
    Cunha R.L.G.
    dos Santos M.A.
    Trends in Psychology, 2022, 30 (3) : 513 - 527