Long-Term Outcomes of Hematopoietic Stem Cell Transplantation for Severe Treatment-Resistant Autoimmune Cytopenia in Children

Biology of Blood and Marrow Transplantation

Volumn 19, Issue 4, 2013, Pages 666-669

  Rabusin, Marco ^a   Snowden, J A ^b   Veys, Paul ^c   Quartier, Pierre ^d   Dalle, Jean Hughes ^e   Dhooge, Catharina ^f   Di Bartolomeo, Paolo ^g   Gonzalez Vicent, Maria ^h   Gibson, Brenda ⁱ   Iriondo, Arturo ^j   Juergens, Herbert ^k   Lisukov, Igor ^l   Messina, Chiara ^m   Mialou, Valerie ⁿ   Steward, Colin Graham ^o   Urban, Christian ^p   Renard, Marleen ^q   Giurici, Nagua ^a   Peters, Christina ^r   Badoglio, Manuela ^s   Ronfani, Luca ^a   Dini, Giorgio ^t   Farge, Dominique ^u   Saccardi, Riccardo ^v   more..

^a INSTITUTE FOR MATERNAL AND CHILD HEALTH IRCCS BURLO GAROFOLO   (Italy)

^b UNIVERSITY OF SHEFFIELD   (United Kingdom)

^c GREAT ORMOND STREET HOSPITAL   (United Kingdom)

^d UNIVERSITÉ PARIS DESCARTES   (France)

^e HÔPITAL ROBERT DEBRÉ   (France)

^f GHENT UNIVERSITY HOSPITAL   (Belgium)

^g Ospedale Civile di Pescara   (Italy)

^h HOSPITAL INFANTIL UNIVERSITARIO NIÑO JESÚS   (Spain)

ⁱ ROYAL HOSPITAL FOR SICK CHILDREN   (United Kingdom)

^j HOSPITAL UNIVERSITARIO MARQUÉS DE VALDECILLA   (Spain)

^k UNIVERSITY HOSPITAL MÜNSTER   (Germany)

^l INSTITUTE OF CLINICAL IMMUNOLOGY   (Russian Federation)

^m UNIVERSITY OF PADOVA   (Italy)

ⁿ HOSPICES CIVILS DE LYON   (France)

^o Royal Hospital for Children   (United Kingdom)

^p MEDICAL UNIVERSITY OF GRAZ   (Austria)

^q UNIVERSITY HOSPITALS LEUVEN   (Belgium)

^r ST ANNA CHILDREN S HOSPITAL   (Austria)

^s SORBONNE UNIVERSITÉ   (France)

^t G GASLINI INSTITUTE   (Italy)

^u SAINT LOUIS HOSPITAL   (France)

^v CAREGGI UNIVERSITY HOSPITAL   (Italy)

more..

Author keywords

Allogeneic transplant; Childhood; Evans syndrome; HSCT; Refractory cytopenia

Indexed keywords

BUSULFAN; CYCLOPHOSPHAMIDE; FLUDARABINE; MELPHALAN; THYMOCYTE ANTIBODY;

ADOLESCENT; ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; ARTICLE; AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION; CHILD; CLINICAL ARTICLE; CONTROLLED STUDY; CYTOPENIA; DISEASE SEVERITY; EVENT FREE SURVIVAL; FEMALE; FOLLOW UP; GRAFT RECIPIENT; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; INFANT; LONG TERM CARE; MALE; MORTALITY; NEWBORN; OUTCOME ASSESSMENT; PRESCHOOL CHILD; REFRACTORY AUTOIMMUNE CYTOPENIA; RELAPSE; RETROSPECTIVE STUDY; SCHOOL CHILD;

ADOLESCENT; AUTOIMMUNE DISEASES; CHILD; CHILD, PRESCHOOL; FEMALE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; INFANT; MALE; NEUTROPENIA; RECURRENCE; RETROSPECTIVE STUDIES; SURVIVAL ANALYSIS; THROMBOCYTOPENIA; TRANSPLANTATION, AUTOLOGOUS; TRANSPLANTATION, HOMOLOGOUS; TREATMENT OUTCOME;

EID: 84875514124     PISSN: 10838791     EISSN: 15236536     Source Type: Journal    
DOI: 10.1016/j.bbmt.2012.12.008     Document Type: Article

References (24)

1. Norton A., Roberts I. Management of Evans syndrome. Br J Haematol 2006, 132:125-137.
2. Wang W. Evans syndrome in childhood: pathophysiology, clinical course, and treatment. Am J Pediatr Hematol Oncol 1988, 15:381-390.
3. Savasan S., Warrier I., Ravindranatah Y. The spectrum of Evans syndrome. Arch Dis Child 1997, 77:245-248.
4. Blouin P., Auvrignon A., Pagnier A., et al. Evans syndrome: a retrospective study from the ship. Arch Pediatr 2005, 12:1600-1607.
5. Mathew P., Chen G., Wang W. Evans syndrome: results of a national survey. J Pediatr Hematol Oncol 1997, 19:433-437.
6. Ware R.E., Rose W.F. Autoimmune hemolytic anemia. Nathan and Oski's hematology of infancy and childhood 1998, 499-522. Saunders, Philadelphia.
7. Alajdidi N., Leverger G., Leblanc T., et al. New insight into childhood hemolytic anemia: a French national observational study of 265 children. Hematologica 2011, 96:655-663.
8. Provan D., Stasi R., Newland A.C., et al. International consensus report on the investigation and management of primary immune thrombocytopenia. Blood 2010, 115:168-186.
9. Jayabose S., Levendoglu-Tugal O., Ozkaynak M.F., et al. Long-term outcome of chronic idiopathic thrombocytopenic purpura in children. J Pediatr Hematol Oncol 2004, 26:724-726.
10. Watts R.G. Idiopathic thrombocytopenic purpura: a 10-year natural history study at the children's hospital of Alabama. Clin Pediatr (Phila) 2004, 43:691-702.
11. Blanchette V.S., Price V. Childhood chronic immune thrombocytopenia purpura: unresolved issues. J Pediatr Hematol Oncol 2003, 25:S28-S33.
12. Bansal D., Bhamare T.A., Trehan A., et al. Outcome of Idiopathic thrombocytopenic purpura in children. Pediatr Blood Cancer 2010, 54:403-407.
13. Snowden J.A., Saccardi R., Allez M., et al. Haematopoietic stem cell transplantation in severe autoimmune diseases: updated guidelines of the European Group for Blood and Marrow Transplantation. Bone Marrow Transplant 2012, 47:770-790.
14. Burt R.K., Marmont A., Oyama Y., et al. Randomized controlled trials of autologous hematopoietic stem cell transplantation for autoimmune diseases: the evolution from myeloablative to lymphoablative transplant regimens. Arthritis Rheum 2006, 54:3750-3760.
15. Huhn R.D., Fogarty P.F., Nakamura R., et al. High-dose cyclophosphamide with autologous lymphocyte-depleted peripheral blood stem cell (PBSC) support for treatment of refractory chronic autoimmune thrombocytopenia. Blood 2003, 101:71-77.
16. Passweg J., Rabusin M., Musso M., et al. Haematopoietic stem cell transplantation for refractory autoimmune cytopenia. Br J Hematol 2004, 125:749-755.
17. Seeliger S., Baumann M., Mohr M., et al. Autologous peripheral blood stem cell transplantation and anti-B cell-directed therapy for refractory autoimmune hemolytic anemia. Eur J Pediatr 2001, 160:493-496.
18. Raetz E., Beatty P.G., Adams R.H. Treatment of severe Evans syndrome with an allogeneic cord blood transplant. Bone Marrow Transplant 1997, 20:427-429.
19. Urban C., Lackner H., Sovinz P., et al. Successful unrelated cord blood transplantation in a 7-year-old boy with Evans syndrome refractory to immunosuppression and double autologous stem cell transplantation. Eur J Hematol 2006, 76:526-530.
20. Marmont A., Gualandi F., Occhini D., et al. Catastrophic relapse of Evans syndrome five years after allogeneic BMT notwithstanding full donor chimerism. Autoimmunity 2006, 39:505-511.
21. Marmont A.M. Immune ablation and stem cell transplantation for severe Evans syndrome and refractory thrombocytopenic purpura. Bone Marrow Transplant 1999, 23:1215-1216.
22. Pession A., Zama D., Masetti R., et al. Hematopoietic stem cell transplantation for curing children with severe autoimmune diseases: is this a valid option?. Pediatr Transplant 2012, 16:413-425.
23. Farge D., Labopin M., Tyndall A., et al. Autologous hematopoietic stem cell transplantation for autoimmune diseases: an observational study on 12 years' experience from the European Group for Blood and Marrow Transplantation Working Party on Autoimmune Diseases. Haematologica 2010, 95:284-292.
24. Daikeler T., Hugle T., Farge D., et al. Allogeneic hematopoietic SCT for patients with autoimmune diseases. Bone Marrow Transplant 2009, 44:27-33.