Frequency and natural history of inherited bone marrow failure syndromes: The Israeli inherited bone marrow failure registry

Haematologica

Volumn 95, Issue 8, 2010, Pages 1300-1307

  Tamary, Hannah ^a   Nishri, Daniella ^b   Yacobovich, Joanne ^a   Zilber, Rama ^a   Dgany, Orly ^a   Krasnov, Tanya ^a   Aviner, Shraga ^c   Stepensky, Polina ^d   Ravel Vilk, Shoshana ^d   Bitan, Menachem ^d   Kaplinsky, Chaim ^e   Barak, Ayelet Ben ^f   Elhasid, Ronit ^f   Kapelusnik, Joseph ^g   Koren, Ariel ^h   Levin, Carina ^h   Attias, Dina ⁱ   Laor, Ruth ⁱ   Yaniv, Isaac ^a   Rosenberg, Philip S ^j   Alter, Blanche P ^j   more..

^a TEL AVIV UNIVERSITY   (Israel)

^b SCHNEIDER CHILDREN S MEDICAL CENTER OF ISRAEL   (Israel)

^c BARZILAI MEDICAL CENTER   (Israel)

^d HADASSAH HEBREW UNIVERSITY MEDICAL CENTER   (Israel)

^e SHEBA MEDICAL CENTER   (Israel)

^f RAMBAM MEDICAL CENTER   (Israel)

^g BEN GURION UNIVERSITY OF THE NEGEV   (Israel)

^h HAEMEK MEDICAL CENTER   (Israel)

ⁱ Kaplan Medical Center   (Israel)

^j NATIONAL CANCER INSTITUTE   (United States)

more..

Author keywords

Fanconi anemia; Inherited bone marrow failure syndromes; Stem cell transplantation

Indexed keywords

BRCA2 PROTEIN; DYSKERIN; FANCONI ANEMIA GROUP A PROTEIN; FANCONI ANEMIA GROUP C PROTEIN; FANCONI ANEMIA GROUP G PROTEIN; TELOMERASE REVERSE TRANSCRIPTASE; THROMBOPOIETIN RECEPTOR;

ACUTE LYMPHOBLASTIC LEUKEMIA; ACUTE MYELOBLASTIC LEUKEMIA; ADOLESCENT; ADULT; ARTICLE; BLACKFAN DIAMOND ANEMIA; BONE MARROW DEPRESSION; CANCER SUSCEPTIBILITY; CHILD; CONGENITAL AMEGAKARYOTIC THROMBOCYTOPENIA; CONSANGUINITY; CONTROLLED STUDY; DISEASE REGISTRY; DISEASE SEVERITY; DYSKERATOSIS CONGENITA; FANCONI ANEMIA; FEMALE; GENE MUTATION; GENETIC ANALYSIS; HEALTH CARE FACILITY; HEALTH PROGRAM; HUMAN; INCIDENCE; INFANT; ISRAEL; MAJOR CLINICAL STUDY; MALE; MORBIDITY; MORTALITY; MYELODYSPLASTIC SYNDROME; NEUTROPENIA; PRESCHOOL CHILD; PROGNOSIS; RADIUS APLASIA; SCHOOL CHILD; SEVERE CONGENITAL NEUTROPENIA; SHWACHMAN SYNDROME; STEM CELL TRANSPLANTATION; THROMBOCYTOPENIA;

ADOLESCENT; ADULT; BONE MARROW DISEASES; CHILD; CHILD, PRESCHOOL; FEMALE; HEMATOLOGIC NEOPLASMS; HUMANS; INCIDENCE; INFANT; INFANT, NEWBORN; ISRAEL; KAPLAN-MEIER ESTIMATE; MALE; QUESTIONNAIRES; REGISTRIES; SYNDROME; YOUNG ADULT;

EID: 77954348335     PISSN: 03906078     EISSN: 15928721     Source Type: Journal    
DOI: 10.3324/haematol.2009.018119     Document Type: Article

References (23)

1. Bagby GC. Constitutional marrow failure: Introduction. Semin Hematol. 2006;43(3): 145-146.
2. Bagby GC, Alter BP. Fanconi anemia. Semin Hematol. 2006;43(3):147-156.
3. Skokowa J, Germeshausen M, Zeidler C, Welte K. Severe congenital neutropenia: inheritance and pathophysiology. Curr Opin Hematol. 2007;14(1):22-28.
4. Lipton JM. Diamond blackfan anemia: New paradigms for a not so pure inherited red cell aplasia. Semin Hematol. 2006; 43(3):167-177.
5. Vulliamy T, Dokal I. Dyskeratosis congenita. Semin Hematol. 2006;43(3):157-166.
6. Shimamura A. Shwachman-Diamond syndrome. Semin Hematol. 2006;43(3):178-188.
7. Geddis AE. Inherited thrombocytopenia: Congenital amegakaryocytic thrombocytopenia and thrombocytopenia with absent radii. Semin Hematol. 2006;43(3): 196-203.
8. Kutler DI, Singh B, Satagopan J, Batish SD, Berwick M, Giampietro PF, et al. A 20-year perspective on the International Fanconi Anemia Registry (IFAR). Blood. 2003;101 (4):1249-1256.
9. Lipton JM, Atsidaftos E, Zyskind I, Vlachos A. Improving clinical care and elucidating the pathophysiology of Diamond Blackfan anemia: an update from the Diamond Blackfan Anemia Registry. Pediatr Blood Cancer. 2006;46(5):558-564.
10. Dale DC, Bolyard AA, Schwinzer BG, Pracht G, Bonilla MA, Boxer L, et al. The Severe Chronic Neutropenia International Registry: 10-Year Follow-up Report. Support Cancer Ther. 2006;3(4):220-231.
11. Rosenberg PS, Greene MH, Alter BP. Cancer incidence in persons with Fanconi anemia. Blood. 2003;101(3):822-826.
12. Rosenberg PS, Huang Y, Alter BP. Individualized risks of first adverse events in patients with Fanconi anemia. Blood. 2004;104(2):350-355.
13. Rosenberg PS, Alter BP, Ebell W. Cancer risks in Fanconi anemia: findings from the German Fanconi Anemia Registry. Haematologica. 2008;93(4):511-517.
14. de Winter JP, Joenje H. The genetic and molecular basis of Fanconi anemia. Mutat Res. 2009;668(1-2):11-19.
15. Dokal I. Dyskeratosis congenita in all its forms. Br J Haematol. 2000;110(4):768-779.
16. Savage SA, Alter BP. Dyskeratosis congenita congenita. Hematol Oncol Clin North Am. 2009; 23(2):215-231.
17. Vlachos A, Ball S, Dahl N, Alter BP, Sheth S, Ramenghi U, et al. Diagnosing and treating Diamond Blackfan anaemia: results of an international clinical consensus conference. Br J Haematol. 2008;142(6):859-876.
18. Lipton JM, Ellis SR. Diamond-Blackfan anemia: diagnosis, treatment, and molecular pathogenesis. Hematol Oncol Clin North Am. 2009;23(2):261-282.
19. Welte K, Zeidler C. Severe congenital neutropenia. Hematol Oncol Clin North Am. 2009;23(2):307-320.
20. Statistics ICBo. Israeli Central Bureau of Statistics. Available from: http://www.cbs.gov.il/energy/shnatonhnew_site.htm.
21. Ries LAG, Melbert D, Krapcho M, Stinchcomb DG, Howlader N, Horner MJ, et al. SEER Cancer Statistics Review, 1975-2005. 2008; Available from: http://seer.cancer.gov/csr/1975_2005/.
22. Joenje H, Patel KJ. The emerging genetic and molecular basis of Fanconi anaemia. Nat Rev Genet. 2001;2(6):446-457.
23. Alter BP, Giri N, Savage SA, Rosenberg PS. Cancer in dyskeratosis congenita. Blood. 2009;113(26):6549-6557.