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Late effects after hematopoietic stem cell transplantation for thalassemia major
References
1. Angelucci E, Barosi G, Camaschella C, et al. Italian Society of Hematology Practice Guidelines for the Management of Iron Overload in Thalassemia Major and Related Disorders. Haematologica. 2008;93(5):741- 752.
2. Rachmilewitz EA, Giardina PJ. How I treat thalassemia. Blood. 2011;118(13):3479–3488
3. Cavazzana M, Antoniani C, Miccio A. Gene therapy for -hemoglobinopathies. Mol Ther. 2017;25(3):1142-1154.
4. Thomas ED, Buckner CD, Sanders JE, Papayannopoulou T, et al. Marrow Transplantation for Thalassaemia. Lancet. 1982;2(8292):227-229.
5. Lucarelli G, Polchi P, Galimberti M, et al. Marrow Transplantation for Thalassaemia Following Busulphan and Cyclophosphamide. Lancet. 1985;1(8442): 1355-1357.
6. Lucarelli G, Galimberti M, Polchi P, et al. Marrow Transplantation in Patients with Thalassemia Responsive to Iron Chelation Therapy. N Engl J Med. 1993;329(12):840- 844.
7. Angelucci E. Hematopoietic stem cell trans- plantation in thalassemia. Hematology Am Soc Hematol Educ Program. 2010;2010:456- 462
8. Sabloff M, Chandy M, Wang Z, et al. HLA- matched sibling bone marrow transplanta- tion for beta-thalassemia major. Blood. 2011;117(5):1745-1750.
9. Baronciani D, Angelucci E, Potschger U, et al. Hematopoietic stem cell transplantation in thalassemia: a report from the European Society for Blood and Bone Marrow Transplantation Hemoglobinopathy Registry, 2000-2010. Bone Marrow Transplant. 2016;51(4):536-541.
10. Galambrun C, Pondarré C, Bertrand Y, et al. French Multicenter 22-Year Experience in Stem Cell Transplantation for Beta- Thalassemia Major: Lessons and Future Directions. Biol Blood Marrow Transplant. 2013;19(1):62-68.
11. Di Bartolomeo P, Santarone S, Di Bartolomeo E, et al. Long-Term Results of Survival in Patients with Thalassemia Major Treated with Bone Marrow Transplantation. Am J Hematol. 2008;83(7):528-530.
12. La Nasa G, Caocci G, Efficace F, et al. Long- Term Health-Related Quality of Life Evaluated More than 20 Years after Hematopoietic Stem Cell Transplantation for Thalassemia. Blood. 2013;122(13):2262-2270.
13. De Simone M, Olioso P, Di Bartolomeo P, et al. Growth and Endocrine Function Following Bone Marrow Transplantation for Thalassemia. Bone Marrow Transplant. 1995;15(2):227-233.
14. Shenoy S, Angelucci E, Arnold SD, et al. Current Results and Future Research Priorities in Late Effects after Hematopoietic Stem Cell Transplantation (HCT) for Children with Sickle Cell Disease and Thalassemia: a Consensus Statement From the Second Pediatric Blood and Marrow Transplant Consortium International Conference on Late Effects after Pediatric HCT. Biol Blood Marrow Transplant. 2017;23(4):552-561.
15. Thuret I, Pondarré C, Loundou A, et al. Complications and treatment of patients with -thalassemia in France: results of the National Registry. Haematologica. 2010; 95(5): 724-729.
16. Sempé M, Capron JP. [“Chronos”: analysis of bone maturation by an automated numerical
method]. Pediatrie. 1979;34(8):833-845.
17. Brown H, Prescott R. Applied Mixed Models in Medicine, 2nd Ed. John Wiley& Sons Ltd:
Chichester, UK; 2006.
18. De Sanctis, V. Growth and Puberty and Its
Management in Thalassaemia. Horm Res.
2002;58 (Suppl 1):72-79.
19. Li CK, Chik KW, Wong GW, Cheng PS, Lee
V, Shing MM. Growth and Endocrine Function Following Bone Marrow Transplantation for Thalassemia Major. Pediatr Hematol Oncol. 2004;21(5):411-419.
20. Vlachopapadopoulou E, Kitra V, Peristeri J, et al. Gonadal Function of Young Patients with Beta-Thalassemia Following Bone Marrow Transplantation. J Pediatr Endocrinol Metab. 2005;18(5):477-483.
21. Khalil A, Zaidman, Elhasid R, Peretz-Nahum M, Futerman B, Ben-Arush M. Factors influ- encing outcome and incidence of late compli- cations in children who underwent allogeneic Hematopoietic Stem Cell Transplantation for Hemoglobinopathy. Pediatr Hematol Oncol. 2012;29(8):694-703.
22. Aldemir-Kocaba B, Tezcan-Karasu G, Bircan I, Bircan O, Akta -Samur A, Ye ilipek MA. Evaluating the Patients with Thalassemia Major for Long-Term Endocrinological Complications after Bone Marrow Transplantation. Pediatr Hematol Oncol. 2014;31(7):616-623.
23. Poomthavorn P, Chawalitdamrong P, Hongeng S, et al. Gonadal Function of Beta- Thalassemics Following Stem Cell Transplantation Conditioned with Myeloablative and Reduced Intensity Regimens. J Pediatr Endorinol Metab. 2013;26(9-10):925-932.
24. Chaudhury S, Ayas M, Rosen C, et al. A Multicenter Retrospective Analysis Stressing the Importance of Long-Term Follow-Up after Hematopoietic Cell Transplantation for
-Thalassemia. Biol Blood Marrow Transplant. 2017;10(10):1695-1700.
25. See WQ, Tung JY, Cheuk DK, et al. Endocrine complications in patients with transfusion- dependent thalassaemia after haematopoietic stem cell transplantation. Bone Marrow Transplantat. 2018;53(3):356-360.
26. Baker TG. A quantitative and cytological study of germ cells in human ovaries. Proc R Soc Lond B Biol Sci. 1963;158:417-433.
27. Rivkees SA, Crawford JD. The Relationship of Gonadal Activity and Chemotherapy- Induced Gonadal Damage. JAMA. 1988;259(14):2123-2125.
28- Afify Z, Shaw PJ, Clavano-Harding A, Cowell CT. Growth and Endocrine Function in Children with Acute Myeloid Leukaemia after Bone Marrow Transplantation Using Busulfan/Cyclophosphamide. Bone Marrow Transplant. 2000;25(10):1087-1092.
29. Allewelt H, El-Khorazaty J, Mendizabal A, et al. Late Effects after Umbilical Cord Blood Transplantation in Very Young Children after Busulfan-Based, Myeloablative Conditioning. Biol Blood Marrow Transplant. 2016;22(9):1627-1635.
30. Cho WK, Lee JW, Chung NG, Jung MH, et al. Primary Ovarian Dysfunction after Hematopoietic Stem Cell Transplantation during Childhood: Busulfan-Based Conditioning Is a Major Concern. J Pediatr Endocrinol Metab. 2011;24(11-12):1031- 1035.
31. Santarone S, Natale A, Olioso P, et al. Pregnancy Outcome Following Hematopoietic Cell Transplantation for Thalassemia Major. Bone Marrow Transplantation 2017;52(3):388-393.
32. De Simone M, Verrotti A, Iughetti L, et al.
Final Height of Thalassemic Patients Who Underwent Bone Marrow Transplantation during Childhood. Bone Marrow Transplant. 2001;28(2):201-205.
33. Bernard F, Bordigoni P, Simeoni MC, et al. Height Growth during Adolescence and Final Height after Haematopoietic SCT for Childhood Acute Leukaemia: The Impact of a Conditioning Regimen with BU or TBI. Bone Marrow Transplant. 2009;43(8):637- 642.
34. Bernard F, Auquier P, Herrmann I, et al. Health Status of Childhood Leukemia Survivors Who Received Hematopoietic Cell Transplantation after BU or TBI: An LEA Study. Bone Marrow Transplant. 2014;49(5):709-716.
35. Michel G, Socié G, Gebhard F, et al. Late Effects of Allogeneic Bone Marrow Transplantation for Children with Acute Myeloblastic Leukemia in First Complete Remission: The Impact of Conditioning Regimen without Total-Body Irradiation--a Report from the Société Française de Greffe de Moelle. J Clin Oncol. 1997;15(6):2238- 2246.
36. Sanders JE, Hoffmeister PA, Woolfrey AE, et al. Thyroid function following hematopoietic cell transplantation in children: 30 years' experience. Blood. 2009;113(2):306-308.
37. Mariotti E, Angelucci E, Agostini A, Baronciani D, Sqarbi E, Lucarelli G. Evaluation of cardiac status in iron-loaded thalassaemia patients following bone mar- row transplantation: improvement in cardiac function during reduction in body iron bur- den. Br J Haematol. 1998;103(4):916-921.
38. Caocci G, Orofino MG, Vacca A, et al. Long- term survival of beta thalassemia major patients treated with hematopoietic stem cell transplantation compared with survival with conventional treatment. Am J Hematol. 2017;92(12):1303-1310.
39. Santarone S, Pepe A, Meloni A, et al. Secondary solid cancer following hematopoi- etic cell transplantation in patients with tha- lassemia major. Bone Marrow Transplant. 2018;53(1):39-43.
40. Angelucci E, Muretto P, Lucarelli G, et al. Phlebotomy to reduce iron overload in patients cured of thalassemia by bone mar- row transplantation. Italian Cooperative Group for Phlebotomy Treatment of Transplanted Thalassemia Patients. Blood. 1997;90(3):994-998.
41. Angelucci E, Pilo F. Management of iron over- load before, during, and after hematopoietic stem cell transplantation for thalassemia major. Ann NY Acad Sci. 2016;1368(1):115- 121.
42. Inati A, Kahale M, Sbeiti N, et al. One-year results from a prospective randomized trial comparing phlebotomy with deferasirox for the treatment of iron overload in pediatric patients with thalassemia major following curative stem cell transplantation. Pediatr Blood Cancer. 2017;64(1):188-196.
43. Bernardo ME, Piras E, Vacca A, et al. Allogeneic Hematopoietic Stem Cell Transplantationin Thalassemia Major: Results of a Reduced-Toxicity Conditioning Regimen Based on the Use of Treosulfan. Blood. 2012;120(2):473-476.
44. Anurathapan U, Pakakasama S, Mekjaruskul P, et al. Outcomes of Thalassemia Patients Undergoing Hematopoietic Stem Cell Transplantation by Using a Standard Myeloablative versus a Novel Reduced- Toxicity Conditioning Regimen according to a New Risk Stratification. Biol Blood Marrow Transplant. 2014;20(12):2066-2071.
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