Abstract
References
- 1. Barone A, Lucarelli A, Onofrillo D,Verzegnassi F, Bonanomi S, Cesaro S, et al. Diagnosis and management of acquired aplastic anemia in childhood. Guidelines from the Marrow Failure Study Group of the Pediatric Haemato-Oncology Italian Association (AIEOP). Blood Cells Mol Dis 2015;55:40-7.
- 2. Sun Q, Wu B, Zhu Z, Sun C, Xu J, Long H, et al. Allogeneic hematopoietic stem cell transplant for severe aplastic anemia: Current state and future directions. Curr Stem Cell Res Ther 2017; Feb 27 (Epub ahead of print).
- 3. Kook H, Chung NG, Kang HJ, Kang HJ, Im HJ. Acquired aplastic anemia in Korean children: Treatment guidelines from the Bone Marrow Failure Committee of the Korean Society of Pediatric Hematology Oncology. Int J Hematol 2016;103:380-6.
- 4. Xu LP, Zhang XH, Wang FR, Xu LP, Zhang XH, Wang FR, et al. Haploidentical transplantation for pediatric patients with acquired severe aplastic anemia. Bone Marrow Transplant 2017;52:381-7.
- 5. Hamidieh AA, Mozafari M, Noshad S, Hadjibabaie M, Ashouri A, Jahangard-Rafsanjani Z, et al. Matched related donor hematopoietic stem cell transplantation results in a long-term follow-up of a pediatric acquired severe aplastic anemia subset: A stem cell source perspective. Pediatr Transplant 2015;19:399- 407.
- 6. Yoshida N, Kobayashi R, Yabe H, Kosaka Y, Yagasaki H, Watanabe K, et al. First-line treatment for severe aplastic anemia in children: Bone marrow transplantation from a matched family donor versus immunosuppressive therapy. Haematologica 2014;99:1784-91.
- 7. Hartung HD, Olson TS, Bessler M. Acquired aplastic anemia in children. Pediatr Clin North Am 2013;60:1311-36.
- 8. Williams DA, Bennett C, Bertuch A, Bessler M, Coates T, Corey S, et al. Diagnosis and treatment of pediatric acquired aplastic anemia (AAA): An initial survey of the North American Pediatric Aplastic Anemia Consortium (NAPAAC). Pediatr Blood Cancer 2014;61:869-74.
- 9. Georges GE, Storb R. Hematopoietic stem cell transplantation for acquired aplastic anemia. Curr Opin Hematol 2016;23:495-500.
- 10. Xiao PF, Hu SY, He HL, Lu J, Li J, Chai YH. Efficacy analysis of allogeneic hematopoietic stem cell transplantation for children with severe aplastic Anemia. Zhongguo Shi Yan Xue Ye Xue Za Zhi 2015;23:1103-7.
- 11. Xu LP, Wang SQ, Wu DP, Wang JM, Gao SJ, Jiang M, et al. Haploidentical transplantation for acquired severe aplastic anaemia in a multicentre prospective study. Br J Haematol 2016;175:265-74.
- 12. Yamazaki H. Hematopoietic stem cell transplantation for acquired aplastic anemia. Rinsho Ketsueki 2015;56:2153-9.
- 13. Zhang Y, Guo Z, Liu XD, He XP, Yang K, Chen P, et al. Comparison of haploidentical hematopoietic stem cell transplantation and immunosuppressive therapy for the treatment of acquired severe aplastic anemia in pediatric patients. Am J Ther 2017;24:e196-e201.
- 14. Liu L, Wang X, Jin S, Hao L, Zhang Y, Zhang X, et al. Haploidentical hematopoietic stem cell transplantation for nonresponders to immunosuppressive therapy against acquired severe aplastic anemia. Bone Marrow Transplant 2016;51:424-7.
Abstract
Amaç: Pediatrik ağır edinsel aplastik anemide eğer hastanın HLA-özdeş bir aile içi donörü varsa hematopoetik kök hücre nakli ilk basamak tedavi olarak önerilmektedir. Çalışmada edinsel aplastik anemi tanısı ile izlenen hastalarımızın allojenik kök hücre nakli sonuçlarını değerlendirmeyi amaçladık.
Gereç ve Yöntemler: Nisan 2010-Temmuz 2017 tarihleri arasında merkezimizde allojenik hematopoetik kök hücre nakli ile tedavi edilen 10 pediatrik edinsel aplastik anemi hastasının verileri hasta dosyalarından ve bilgisayar kayıtlarından geriye dönük olarak değerlendirildi.
Bulgular: Ağır ve çok ağır edinsel aplastik anemi tanılı 10 hastaya toplam 12 allojenik hematopoetik kök hücre nakli yapıldı. Nakil sırasında ortalama yaş 10.9±3.76 (min=4.6-max=16.1) yıl ve hastaların %80’i kızdı. Ortalama tanı-nakil arası süre 10.1±7.65 (min=3-max=31) aydı ve vericilerin hepsi kardeşdi. İnfüze edilen ortalama çekirdekli hücre sayısı 4.69±2.17x108 (min=1.13x108-max=8.43x108) ve CD34+hücre sayısı 3.25±1.39x106 (min=1.65x106- max=6.55x106)’di. Ortalama nötrofil engrafman zamanı 16.2±0.8 (min=15-max=17) gün ve trombosit engrafman zamanı 26±6.5 (min=16-max= 37) gündü. Hiçbir hastada akut greft versus host hastalığı gözlenmedi, sadece bir hastada sınırlı kronik akciğer greft versus host hastalığı oluştu. Bir hastada primer greft yetmezliği, bir diğerinde ise sekonder greft yetmezliği gelişti. Takip süresince, yaklaşık 78 ay hiçbir hastada miyelodisplastik sendrom (MDS), lösemi, paroksismal nokturnal hemoglobinüri (PNH) ve sekonder malignite gelişmedi. Bir hasta henüz engrafman olmadan nakil sonrası +16. günde intrakranial kanama nedeni ile kaybedildi. Hayatta kalan hastaların %66.7’sinde kimerizm karmaydı (mix) ve tüm hastalar halen tam hematolojik remisyon olarak takip edilmektedir.
Sonuç: Yaklaşık 7 yıllık takip süresinde hastalarımızda %90 genel sağkalım elde ettik. Literatürde bildirilen çalışmalarda immünosupresif tedavi sonrası görülen yüksek relaps ve klonal hematopoez oranları dikkate alındığında özellikle HLA tam uyumlu aile içi vericisi varsa allojenik hematopoetik kök hücre naklinin çocuklarda aplastik anemi tedavisinde birinci basamak tedavi olarak kullanılabileceğini düşündük.
Keywords
References
- 1. Barone A, Lucarelli A, Onofrillo D,Verzegnassi F, Bonanomi S, Cesaro S, et al. Diagnosis and management of acquired aplastic anemia in childhood. Guidelines from the Marrow Failure Study Group of the Pediatric Haemato-Oncology Italian Association (AIEOP). Blood Cells Mol Dis 2015;55:40-7.
- 2. Sun Q, Wu B, Zhu Z, Sun C, Xu J, Long H, et al. Allogeneic hematopoietic stem cell transplant for severe aplastic anemia: Current state and future directions. Curr Stem Cell Res Ther 2017; Feb 27 (Epub ahead of print).
- 3. Kook H, Chung NG, Kang HJ, Kang HJ, Im HJ. Acquired aplastic anemia in Korean children: Treatment guidelines from the Bone Marrow Failure Committee of the Korean Society of Pediatric Hematology Oncology. Int J Hematol 2016;103:380-6.
- 4. Xu LP, Zhang XH, Wang FR, Xu LP, Zhang XH, Wang FR, et al. Haploidentical transplantation for pediatric patients with acquired severe aplastic anemia. Bone Marrow Transplant 2017;52:381-7.
- 5. Hamidieh AA, Mozafari M, Noshad S, Hadjibabaie M, Ashouri A, Jahangard-Rafsanjani Z, et al. Matched related donor hematopoietic stem cell transplantation results in a long-term follow-up of a pediatric acquired severe aplastic anemia subset: A stem cell source perspective. Pediatr Transplant 2015;19:399- 407.
- 6. Yoshida N, Kobayashi R, Yabe H, Kosaka Y, Yagasaki H, Watanabe K, et al. First-line treatment for severe aplastic anemia in children: Bone marrow transplantation from a matched family donor versus immunosuppressive therapy. Haematologica 2014;99:1784-91.
- 7. Hartung HD, Olson TS, Bessler M. Acquired aplastic anemia in children. Pediatr Clin North Am 2013;60:1311-36.
- 8. Williams DA, Bennett C, Bertuch A, Bessler M, Coates T, Corey S, et al. Diagnosis and treatment of pediatric acquired aplastic anemia (AAA): An initial survey of the North American Pediatric Aplastic Anemia Consortium (NAPAAC). Pediatr Blood Cancer 2014;61:869-74.
- 9. Georges GE, Storb R. Hematopoietic stem cell transplantation for acquired aplastic anemia. Curr Opin Hematol 2016;23:495-500.
- 10. Xiao PF, Hu SY, He HL, Lu J, Li J, Chai YH. Efficacy analysis of allogeneic hematopoietic stem cell transplantation for children with severe aplastic Anemia. Zhongguo Shi Yan Xue Ye Xue Za Zhi 2015;23:1103-7.
- 11. Xu LP, Wang SQ, Wu DP, Wang JM, Gao SJ, Jiang M, et al. Haploidentical transplantation for acquired severe aplastic anaemia in a multicentre prospective study. Br J Haematol 2016;175:265-74.
- 12. Yamazaki H. Hematopoietic stem cell transplantation for acquired aplastic anemia. Rinsho Ketsueki 2015;56:2153-9.
- 13. Zhang Y, Guo Z, Liu XD, He XP, Yang K, Chen P, et al. Comparison of haploidentical hematopoietic stem cell transplantation and immunosuppressive therapy for the treatment of acquired severe aplastic anemia in pediatric patients. Am J Ther 2017;24:e196-e201.
- 14. Liu L, Wang X, Jin S, Hao L, Zhang Y, Zhang X, et al. Haploidentical hematopoietic stem cell transplantation for nonresponders to immunosuppressive therapy against acquired severe aplastic anemia. Bone Marrow Transplant 2016;51:424-7.
Details
| Primary Language | Turkish |
|---|---|
| Subjects | Internal Diseases |
| Journal Section | ORIGINAL ARTICLES |
| Authors | |
| Publication Date | March 26, 2019 |
| Submission Date | September 12, 2017 |
| Published in Issue | Year 2019 Volume: 13 Issue: 2 |
The publication language of Turkish Journal of Pediatric Disease is English.
Manuscripts submitted to the Turkish Journal of Pediatric Disease will go through a double-blind peer-review process. Each submission will be reviewed by at least two external, independent peer reviewers who are experts in the field, in order to ensure an unbiased evaluation process. The editorial board will invite an external and independent editor to manage the evaluation processes of manuscripts submitted by editors or by the editorial board members of the journal. The Editor in Chief is the final authority in the decision-making process for all submissions. Articles accepted for publication in the Turkish Journal of Pediatrics are put in the order of publication, with at least 10 original articles in each issue, taking into account the acceptance dates. If the articles sent to the reviewers for evaluation are assessed as a senior for publication by the reviewers, the section editor and the editor considering all aspects (originality, high scientific quality and citation potential), it receives publication priority in addition to the articles assigned for the next issue.
The aim of the Turkish Journal of Pediatrics is to publish high-quality original research articles that will contribute to the international literature in the field of general pediatric health and diseases and its sub-branches. It also publishes editorial opinions, letters to the editor, reviews, case reports, book reviews, comments on previously published articles, meeting and conference proceedings, announcements, and biography. In addition to the field of child health and diseases, the journal also includes articles prepared in fields such as surgery, dentistry, public health, nutrition and dietetics, social services, human genetics, basic sciences, psychology, psychiatry, educational sciences, sociology and nursing, provided that they are related to this field. can be published.