Stem cell transplantation in severe congenital neutropenia: An analysis from the European Society for Blood and Marrow Transplantation

Blood

Volumn 126, Issue 16, 2015, Pages 1885-1892

  Fioredda, Francesca ^a   Iacobelli, Simona ^b   Van Biezen, Anja ^c   Gaspar, Bobby ^d   Ancliff, Phil ^e   Donadieu, Jean ^f   Aljurf, Mahmoud ^g   Peters, Christina ^h   Calvillo, Michaela ^a   Matthes Martin, Susanne ^h   Morreale, Giuseppe ^a   Van't Veer Tazelaar, Nelleke ^c   De Wreede, Liesbeth ^c   Al Seraihy, Amal ^g   Yesilipek, Akif ⁱ   Fischer, Alain ^j   Bierings, Marc ^k   Ozturk, Gulyuz ^l   Smith, Owen ^m   Veys, Paul ^e   Ljungman, Per ⁿ   De Latour, Régis Peffault ^o   De Toledo Codina, José Sánchez ^p   Or, Reuven ^q   Ganser, Arnold ^r   Afanasyev, Boris ^s   Wynn, Robert ^t   Kalwak, Krzysztof ^u   Marsh, Judith ^v   Dufour, Carlo ^a   more..

^a G GASLINI INSTITUTE   (Italy)

^b UNIVERSITY OF ROME TOR VERGATA   (Italy)

^c LEIDEN UNIVERSITY MEDICAL CENTER   (Netherlands)

^d UNIVERSITY COLLEGE LONDON   (United Kingdom)

^e GREAT ORMOND STREET HOSPITAL   (United Kingdom)

^f ARMAND TROUSSEAU HOSPITAL   (France)

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

^h St Anna Children's Hospital   (Australia)

ⁱ AKDENIZ UNIVERSITY   (Turkey)

^j HÔPITAL NECKER ENFANTS MALADES   (France)

^k UNIVERSITY MEDICAL CENTER UTRECHT   (Netherlands)

^l ISTANBUL UNIVERSITY   (Turkey)

^m OUR LADY S CHILDREN S HOSPITAL   (Ireland)

ⁿ KAROLINSKA UNIVERSITY HOSPITAL   (Sweden)

^o SAINT LOUIS HOSPITAL   (France)

^p HOSPITAL UNIVERSITARI VALL D HEBRON   (Spain)

^q HADASSAH HEBREW UNIVERSITY MEDICAL CENTER   (Israel)

^r HANNOVER MEDICAL SCHOOL   (Germany)

^s PAVLOV FIRST SAINT PETERSBURG STATE MEDICAL UNIVERSITY   (Russian Federation)

^t ROYAL MANCHESTER CHILDREN S HOSPITAL   (United Kingdom)

^u WROCLAW MEDICAL UNIVERSITY   (Poland)

^v KING S COLLEGE HOSPITAL   (United Kingdom)

more..

Author keywords

[No Author keywords available]

Indexed keywords

CYCLOSPORIN A; METHOTREXATE; CYCLOSPORIN;

ACUTE GRAFT VERSUS HOST DISEASE; ADOLESCENT; ADULT; ARTICLE; CHILD; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; EUROPEAN; FEMALE; GRAFT FAILURE; HLA MATCHING; HUMAN; IMMUNOPROPHYLAXIS; INFANT; LIVING DONOR; MAJOR CLINICAL STUDY; MALE; MEDICAL SOCIETY; MORTALITY; MULTICENTER STUDY; OVERALL SURVIVAL; POSTOPERATIVE COMPLICATION; PRIORITY JOURNAL; SEVERE CONGENITAL NEUTROPENIA; STEM CELL TRANSPLANTATION; UNRELATED DONORS; ACUTE DISEASE; ALLOGRAFT; CLINICAL TRIAL; EUROPE; GRAFT REJECTION; GRAFT VS HOST DISEASE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HISTOCOMPATIBILITY TEST; INCIDENCE; MIDDLE EAST; NEUTROPENIA; PRESCHOOL CHILD; RETROSPECTIVE STUDY;

ACUTE DISEASE; ADOLESCENT; ADULT; ALLOGRAFTS; CHILD; CHILD, PRESCHOOL; CYCLOSPORINE; EUROPE; FEMALE; GRAFT REJECTION; GRAFT VS HOST DISEASE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HISTOCOMPATIBILITY TESTING; HUMANS; INCIDENCE; MALE; METHOTREXATE; MIDDLE EAST; NEUTROPENIA; RETROSPECTIVE STUDIES; SOCIETIES, MEDICAL; UNRELATED DONORS;

EID: 84944233630     PISSN: 00064971     EISSN: 15280020     Source Type: Journal    
DOI: 10.1182/blood-2015-02-628859     Document Type: Article

References (39)

1. Welte K, Zeidler C, Dale DC. Severe congenital neutropenia. Semin Hematol. 2006; 43(3): 189-195.
2. Xia J, Bolyard AA, Rodger E, et al. Prevalence of mutations in ELANE, GFI1, HAX1, SBDS, WAS and G6PC3 in patients with severe congenital neutropenia. Br J Haematol. 2009; 147(4): 535-542.
3. Dale DC, Person RE, Bolyard AA, et al. Mutations in the gene encoding neutrophil elastase in congenital and cyclic neutropenia. Blood. 2000; 96(7): 2317-2322.
4. Klein C, Grudzien M, Appaswamy G, et al. HAX1 deficiency causes autosomal recessive severe congenital neutropenia (Kostmann disease). Nat Genet. 2007; 39(1): 86-92.
5. Person RE, Li FQ, Duan Z, et al. Mutations in proto-oncogene GFI1 cause human neutropenia and target ELA2. Nat Genet. 2003; 34(3): 308-312.
6. Boztug K, Appaswamy G, Ashikov A, et al. A syndrome with congenital neutropenia and mutations in G6PC3. N Engl J Med. 2009; 360(1): 32-43.
7. Devriendt K, Kim AS, Mathijs G, et al. Constitutively activating mutation in WASP causes X-linked severe congenital neutropenia. Nat Genet. 2001; 27(3): 313-317.
8. Dale DC, Bonilla MA, Davis MW, et al. A randomized controlled phase III trial of recombinant human granulocyte colony-stimulating factor (filgrastim) for treatment of severe chronic neutropenia. Blood. 1993; 81(10): 2496-2502.
9. Donadieu J, Leblanc T, Bader Meunier B, et al; French Severe Chronic Neutropenia Study Group. Analysis of risk factors for myelodysplasias, leukemias and death from infection among patients with congenital neutropenia. Experience of the French Severe Chronic Neutropenia Study Group. Haematologica. 2005; 90(1): 45-53.
10. Rosenberg PS, Zeidler C, Bolyard AA, et al. Stable long-term risk of leukaemia in patients with severe congenital neutropenia maintained on G-CSF therapy. Br J Haematol. 2010; 150(2): 196-199.
11. Choi SW, Levine J. Indications for hematopoietic cell transplantation for children with severe congenital neutropenia. Pediatr Transplant. 2010; 14(8): 937-939.
12. Zeidler C, Welte K, Barak Y, et al. Stem cell transplantation in patients with severe congenital neutropenia without evidence of leukemic transformation. Blood. 2000; 95(4): 1195-1198.
13. Ferry C, Ouachée M, Leblanc T, et al. Hematopoietic stem cell transplantation in severe congenital neutropenia: Experience of the French SCN register. Bone Marrow Transplant. 2005; 35(1): 45-50.
14. Oshima K, Hanada R, Kobayashi R, et al; Hematopoietic Stem Cell Transplantation Committee of the Japanese Society of Pediatric Hematology. Hematopoietic stem cell transplantation in patients with severe congenital neutropenia: An analysis of 18 Japanese cases. Pediatr Transplant. 2010; 14(5): 657-663.
15. Choi SW, Boxer LA, Pulsipher MA, et al. Stem cell transplantation in patients with severe congenital neutropenia with evidence of leukemic transformation. Bone Marrow Transplant. 2005; 35(5): 473-477.
16. Bizzetto R, Bonfim C, Rocha V, et al; Eurocord and SAA-WP from EBMT. Outcomes after related and unrelated umbilical cord blood transplantation for hereditary bone marrow failure syndromes other than Fanconi anemia. Haematologica. 2011; 96(1): 134-141.
17. Ringdén O, Okas M, Uhlin M, Uzunel M, Remberger M, Mattsson J. Unrelated cord blood and mismatched unrelated volunteer donor transplants, two alternatives in patients who lack an HLA-identical donor. Bone Marrow Transplant. 2008; 42(10): 643-648.
18. Carlsson G, Winiarski J, Ljungman P, et al. Hematopoietic stem cell transplantation in severe congenital neutropenia. Pediatr Blood Cancer. 2011; 56(3): 444-451.
19. Mino E, Kobayashi R, Yoshida M, Suzuki Y, Yamada M, Kobayashi K. Umbilical cord blood stem cell transplantation from unrelated HLAmatched donor in an infant with severe congenital neutropenia. Bone Marrow Transplant. 2004; 33(9): 969-971.
20. Nakazawa Y, Sakashita K, Kinoshita M, et al. Successful unrelated cord blood transplantation using a reduced-intensity conditioning regimen in a 6-month-old infant with congenital neutropenia complicated by severe pneumonia. Int J Hematol. 2004; 80(3): 287-290.
21. Cojean N, Blondet C, Marcellin L, et al. Successful stem cell transplantation in an infant with severe congenital neutropenia complicated by pretransplant inflammatory pseudotumor of the liver. Bone Marrow Transplant. 2006; 38(9): 641-643.
22. Thachil J, Caswell M, Bolton-Maggs PH, Pizer B, Keenan R. Non-myeloablative transplantation for severe congenital neutropenia. Pediatr Blood Cancer. 2008; 50(4): 920-921.
23. Markel MK, Haut PR, Renbarger JA, Robertson KA, Goebel WS. Unrelated cord blood transplantation for severe congenital neutropenia: Report of two cases with very different transplant courses. Pediatr Transplant. 2008; 12(8): 896-901.
24. Yesilipek MA, Tezcan G, Germeshausen M, Kupesiz A, Uygun V, Hazar V. Unrelated cord blood transplantation in children with severe congenital neutropenia. Pediatr Transplant. 2009; 13(6): 777-781.
25. Toyoda H, Azuma E, Hori H, et al. Successful unrelated BMT in a patient with Kostmann syndrome complicated by pre-transplant pulmonary 'bacterial' abscesses. Bone Marrow Transplant. 2001; 28(4): 413-415.
26. Fukano R, Nagatoshi Y, Shinkoda Y, et al. Unrelated bone marrow transplantation using a reduced-intensity conditioning regimen for the treatment of Kostmann syndrome. Bone Marrow Transplant. 2006; 38(9): 635-636.
27. Dallorso S, Manzitti C, Dodero P, Faraci M, Rosanda C, Castagnola E. Uneventful outcome of unrelated hematopoietic stem cell transplantation in a patient with leukemic transformation of Kostmann syndrome and long-lasting invasive pulmonary mycosis. Eur J Haematol. 2003; 70(5): 322-325.
28. Fioredda F, Calvillo M, Bonanomi S, et al; Neutropenia Committee of the Marrow Failure Syndrome Group of the AIEOP (Associazione Italiana Emato-Oncologia Pediatrica). Congenital and acquired neutropenias consensus guidelines on therapy and follow-up in childhood from the Neutropenia Committee of the Marrow Failure Syndrome Group of the AIEOP (Associazione Italiana Emato-Oncologia Pediatrica). Am J Hematol. 2012; 87(2): 238-243.
29. Przepiorka D, Weisdorf D, Martin P, et al. 1994 Consensus Conference on Acute GVHD Grading. Bone Marrow Transplant. 1995; 15(6): 825-828.
30. Shulman HM, Sullivan KM, Weiden PL, et al. Chronic graft-versus-host syndrome in man. A long-term clinicopathologic study of 20 Seattle patients. Am J Med. 1980; 69(2): 204-217.
31. Giralt S, Ballen K, Rizzo D, et al. Reducedintensity conditioning regimen workshop: Defining the dose spectrum. Report of a workshop convened by the Center for International Blood and Marrow Transplant Research. Biol Blood Marrow Transplant. 2009; 15(3): 367-369.
32. Carlsson G, Fasth A, Berglöf E, et al. Incidence of severe congenital neutropenia in Sweden and risk of evolution to myelodysplastic syndrome/leukaemia. Br J Haematol. 2012; 158(3): 363-369.
33. Connelly JA, Choi SW, Levine JE. Hematopoietic stem cell transplantation for severe congenital neutropenia. Curr Opin Hematol. 2012; 19(1): 44-51.
34. Lucarelli G, Andreani M, Angelucci E. The cure of thalassemia by bone marrow transplantation. Blood Rev. 2002; 16(2): 81-85.
35. Vlachos A, Muir E. How I treat Diamond-Blackfan anemia. Blood. 2010; 116(19): 3715-3723.
36. Peffault de Latour R, Porcher R, Dalle JH, et al; FA Committee of the Severe Aplastic Anemia Working Party; Pediatric Working Party of the European Group for Blood and Marrow Transplantation. Allogeneic hematopoietic stem cell transplantation in Fanconi anemia: The European Group for Blood and Marrow Transplantation experience. Blood. 2013; 122(26): 4279-4286.
37. Bacigalupo A, Socié G, Schrezenmeier H, et al; Aplastic Anemia Working Party of the European Group for Blood and Marrow Transplantation (WPSAA-EBMT). Bone marrow versus peripheral blood as the stem cell source for sibling transplants in acquired aplastic anemia: Survival advantage for bone marrow in all age groups. Haematologica. 2012; 97(8): 1142-1148.
38. Dufour C, Pillon M, Passweg J, et al. 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. 2014; 99(10): 1574-1581.
39. Dale DC, Cottle TE, Fier CJ, et al. Severe chronic neutropenia: Treatment and follow-up of patients in the Severe Chronic Neutropenia International Registry. Am J Hematol. 2003; 72(2): 82-93.