Outcome of aplastic anemia in adolescence: A survey of the Severe Aplastic Anemia Working Party of the European Group for Blood and Marrow Transplantation

Haematologica

Volumn 99, Issue 10, 2014, Pages 1574-1581

  Dufour, Carlo ^a   Pillon, Marta ^b   Passweg, Jakob ^c   Socié, Gerard ^d   Bacigalupo, Andrea ^e   Franceschetto, Genny ^b   Carraro, Elisa ^b   Oneto, Rosi ^e   Risitano, Antonio Maria ^f   de Latour, Regis Peffault ^d   Tichelli, André ^c   Rovo, Alicia ^c   Peters, Christina ^g   Hoechsmann, Britta ^h   Samarasinghe, Sujith ^i,l   Kulasekararaj, Austin G ^j   Schrezenmeier, Hubert ^h   Aljurf, Mahmoud ^k   Marsh, Judith ^j  

^a G GASLINI INSTITUTE   (Italy)

^b UNIVERSITY OF PADOVA   (Italy)

^c UNIVERSITY HOSPITAL BASEL   (Switzerland)

^d SAINT LOUIS HOSPITAL   (France)

^e UNIVERSITY OF GENOA   (Italy)

^f UNIVERSITY OF NAPLES FEDERICO II   (Italy)

^g ST ANNA CHILDREN S HOSPITAL   (Austria)

^h UNIVERSITY OF ULM   (Germany)

ⁱ Infectious Diseases Allergy   (United Kingdom)

^j KING S COLLEGE HOSPITAL   (United Kingdom)

^k KING FAISAL SPECIALIST HOSPITAL AND RESEARCH CENTER   (Saudi Arabia)

^l GREAT ORMOND STREET HOSPITAL   (United Kingdom)

more..

Author keywords

[No Author keywords available]

Indexed keywords

ALEMTUZUMAB; BUSULFAN; CYCLOPHOSPHAMIDE; CYCLOSPORIN A; FLUDARABINE; MELPHALAN; METHOTREXATE; MYCOPHENOLIC ACID 2 MORPHOLINOETHYL ESTER; THYMOCYTE ANTIBODY;

ACUTE GRAFT VERSUS HOST DISEASE; ACUTE LEUKEMIA; ADOLESCENT; ADULT; APLASTIC ANEMIA; ARTICLE; BONE MARROW TRANSPLANTATION; CHILD; CHRONIC GRAFT VERSUS HOST DISEASE; EVENT FREE SURVIVAL; FEMALE; FOLLOW UP; GRAFT REJECTION; HEALTH SURVEY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; IMMUNOSUPPRESSIVE TREATMENT; INCIDENCE; LYMPHOMA; MAJOR CLINICAL STUDY; MALE; MULTIVARIATE ANALYSIS; MYELODYSPLASTIC SYNDROME; OVERALL SURVIVAL; PROGNOSIS; QUALITY OF LIFE; UNIVARIATE ANALYSIS; ADVERSE EFFECTS; ANEMIA, APLASTIC; GRAFT VS HOST DISEASE; HEALTH CARE SURVEY; HEMATOLOGIC NEOPLASMS; MORTALITY; OUTCOME ASSESSMENT; TREATMENT OUTCOME; YOUNG ADULT;

ADOLESCENT; ANEMIA, APLASTIC; BONE MARROW TRANSPLANTATION; CHILD; FEMALE; GRAFT REJECTION; GRAFT VS HOST DISEASE; HEALTH CARE SURVEYS; HEMATOLOGIC NEOPLASMS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; INCIDENCE; MALE; PATIENT OUTCOME ASSESSMENT; PROGNOSIS; TRANSPLANTATION CONDITIONING; TREATMENT OUTCOME; YOUNG ADULT;

EID: 84907499847     PISSN: 03906078     EISSN: 15928721     Source Type: Journal    
DOI: 10.3324/haematol.2014.106096     Document Type: Article

References (35)

1. Young NS, Calado RT, Scheinberg P. Current concepts in the pathophysiology and treatment of aplastic anemia. Blood. 2006;108(8):2509-19.
2. Podesta M, Piaggio G, Frassoni F, Pitto A, Zikos P, Sessarego M, et al. The assessment of the hematopoietic reservoir after immunosuppressive therapy or bone marrow transplantation in severe aplastic anemia. Blood. 1998;91(6):1959-65.
3. Marsh JC, Ball SE, Cavenagh J, Darbyshire P, Dokal I, Gordon-Smith EC, et al. Guidelines for the diagnosis and management of aplastic anaemia. Br J Haematol. 2009;147(1):43-70.
4. Locasciulli A, Oneto R, Bacigalupo A, Socie G, Korthof E, Bekassy A, et al. Outcome of patients with acquired aplastic anemia given first line bone marrow transplantation or immunosuppressive treatment in the last decade: a report from the European Group for Blood and Marrow Transplantation (EBMT). Haematologica. 2007;92(1):11-8.
5. Scheinberg P. Aplastic anemia: therapeutic updates in immunosuppression and transplantation. Hematology Am Soc Hematol Educ Program. 2012;292-300.
6. Gupta V, Eapen M, Brazauskas R, Carreras J, Aljurf M, Gale RP, et al. Impact of age on outcomes after bone marrow transplantation for acquired aplastic anemia using HLA-matched sibling donors. Haematologica. 2010;95(12):2119-25.
7. Eapen M, Le Rademacher J, Antin JH, Champlin RE, Carreras J, Fay J, et al. Effect of stem cell source on outcomes after unrelated donor transplantation in severe aplastic anemia. Blood. 2011;118(9):2618-21.
8. Bacigalupo A, Socié G, Schrezenmeier H, Tichelli A, Locasciulli A, Fuehrer M, et al. Bone marrow versus peripheral blood sibling transplants in acquired aplastic anemia: survival advantage for marrow in all age groups. Haematologica. 2012;97(8):1142-8.
9. Young NS. Epidemiology of Aplastic Anemia. In: Neal S Young, ed. Bone Marrow Failure Syndromes, 1sted, WB Saunders Company; 1996:1-46.
10. Hummel K, McFann KK, Realsen J, Messer LH, Klingensmith GJ, Chase HP. The increasing onset of type 1 diabetes in children. J Pediatr. 2012;161(4):652-7.
11. Derraik JG, Reed PW, Jefferies C, Cutfield SW, Hofman PL, Cutfield WS. Increasing incidence and age at diagnosis among children with type 1 diabetes mellitus over a 20-year period in Auckland (New Zealand) PLoS One. 2012;7(2):e32640.
12. Oen K, Malleson PN, Cabral DA, Rosenberg AM, Petty RE, Cheang M. Disease course and outcome of juvenile rheumatoid arthritis in a multicenter cohort. J Rheumatol. 2002;29(9):1989-99.
13. García-García E, Vázquez-López MÁ, García-Fuentes E, Rodríguez-Sánchez FI, Muñoz FJ, Bonillo-Perales A, et al. Iodine intake and prevalence of thyroid autoimmunity and autoimmune thyroiditis in children and adolescents aged between 1 and 16 years. Eur J Endocrinol. 2012;167(3): 387-92.
14. Piatkowska E, Szalecki M. Autoimmune thyroiditis in children and adolescents with type 1 diabetes. Pediatr Endocrinol Diabetes Metab. 2011;17(4):173-7.
15. Prindaville B, Rivkees SA. Incidence of vitiligo in children with Graves' disease and Hashimoto's thyroiditis. Int J Pediatr Endocrinol. 2011;2011(1):18.
16. Passweg JR, Aljurf M. Treatment and hematopoietic SCT in aplastic anemia. Bone Marrow Transplant. 2013;48(2):161.
17. Tonorezos ES, Sharp L. Health promotion for adolescent leukemia survivors. Pediatr Blood Cancer. 2013;60(6):893-4.
18. WHO Internet; health topics; adolescent health. Available from: http:// www.who.int/topics/adolescent_health/ en/.
19. th ed, Draft 2011.
20. Peto R, Peto J. Asymptotically efficent rank invariant test procedures. J R Stat Soc A. 1972;135:185-98.
21. Kaplan EL, Meier P. Nonparametric estimation from incomplete observation. J Am Stat Assoc. 1958;53:457-81.
22. Kalbfleisch JD, Prentice RL.The Statistical Analysis of Failure Time Data. Wiley: New York, 1980, p 169.
23. Gray RJ. A class of K-sample tests for comparing the cumulative incidence of a competing risk. Ann Stat. 1988;16:1141-54
24. Yoshida N, Yagasaki H, Hama A, Takahashi Y, Kosaka Y, Kobayashi R, et al. Predicting response to immunosuppressive therapy in childhood aplastic anemia. Haematologica. 2011;96(5):771-4.
25. Scheinberg P, Nunez O, Weinstein B, Scheinberg P, Biancotto A, Wu CO, et al. Horse versus rabbit antithymocyte globulin in acquired aplastic anemia. N Engl J Med. 2011;365:430-8.
26. Marsh JC, Bacigalupo A, Schrezenmeier H, Tichelli A, Risitano AM, Passweg JR, et al. Prospective study of rabbit antithymocyte globulin and cyclosporine for aplastic anemia from the EBMT Severe Aplastic Anemia Working Party. Blood. 2012; 119(23):5391-6.
27. Samarasinghe S, Steward C, Hiwarkar P, Saif MA, Hough R, Webb D, et al. Excellent outcome of matched unrelated donor transplantation in paediatric aplastic anaemia following failure with immunosuppressive therapy: a United Kingdom multicentre retrospective experience. Br J Haematol. 2012;157(3):339-46.
28. Jeong DC, Chung NG, Cho B, Zou Y, Ruan M, Takahashi Y, et al. Long-term outcome after immunosuppressive therapy with horse or rabbit antithymocyte globulin and cyclosporine for severe aplastic anemia in children. Haematologica. 2014;99(4):664-71.
29. Schrezenmeier H, Passweg JR, Marsh JC, Bacigalupo A, Bredeson CN, Bullorsky E, et al. Worse outcome and more chronic GVHD with peripheral blood progenitor cells than bone marrow in HLA-matched sibling donor transplants for young patients with severe acquired aplastic anemia. Blood. 2007;110(4):1397-400.
30. Socié G, Henry-Amar M, Bacigalupo A, Hows J, Tichelli A, Ljungman P, et al. Malignant tumors occurring after treatment of aplastic anemia. N Engl J Med. 1993; 329(16):1152-7.
31. Konopacki J, Procher R, Robin M, Bieri S, Cayuela JM, Larghero J, et al. Long-term follow-up after allogeneic stem cell transplantation in patients with severe aplastic anemia after cyclophosphamide plus antithymocyte globulin conditioning. Haematologica. 2012;97(5):710-6.
32. Di Bona E, Rodeghiero F, Bruno B, Gabbas A, Foa P, Locasciulli A, et al. Rabbit antithymocyte globulin (r-ATG) plus cyclosporine and granulocyte colony stimulating factor is an effective treatment for aplastic anaemia patients unresponsive to a first course of intensive immunosuppressive therapy. Gruppo Italiano Trapianto di Midollo Osseo (GITMO). Br J Haematol. 1999;107(2):330-4.
33. Scheinberg P, Young NS. How I treat acquired aplastic anemia. Blood. 2012; 120(6):1185-96.
34. Scheinberg P, Nunez O, Young NS. Retreatment with rabbit anti-thymocyte globulin and ciclosporin for patients with relapsed or refractory severe aplastic anaemia. Br J Haematol. 2006;133(6):622-7.
35. Kosaka Y, Yagasaki H, Sano K, Kobayashi R, Ayukawa H, Kaneko T, et al. Prospective multicenter trial comparing repeated immunosuppressive therapy with stem-cell transplantation from an alternative donor as second-line treatment for children with severe and very severe aplastic anemia. Blood. 2008;111(3):1054-9.