Evidence-based focused review of the status of hematopoietic stem cell transplantation as treatment of sickle cell disease and thalassemia

Blood

Volumn 123, Issue 20, 2014, Pages 3089-3094

  King, Allison ^a   Shenoy, Shalini ^a  

^a ST LOUIS CHILDREN S HOSPITAL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ALEMTUZUMAB; AZATHIOPRINE; DEFERASIROX; DEFEROXAMINE; FERRITIN; FLUDARABINE; HEMOGLOBIN S; IRON; MELPHALAN; OPIATE; THIOTEPA;

ABSENTEEISM; ACADEMIC ACHIEVEMENT; ADOLESCENT; ANALGESIA; BETA THALASSEMIA; CASE REPORT; CEREBROVASCULAR ACCIDENT; CHELATION THERAPY; CLINICAL TRIAL (TOPIC); CYTAPHERESIS; ERYTHROCYTAPHERESIS; ERYTHROCYTE TRANSFUSION; FEMALE; GRAFT VERSUS HOST REACTION; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEPATOSPLENOMEGALY; HOSPITAL ADMISSION; HUMAN; IRON CHELATION; LIVER SIZE; LONG TERM CARE; MYELOABLATIVE CONDITIONING; NUCLEAR MAGNETIC RESONANCE IMAGING; PAIN; PALPATION; PATIENT COMPLIANCE; PRIORITY JOURNAL; REDUCED INTENSITY CONDITIONING; REVIEW; SHORT STATURE; SICKLE CELL ANEMIA; SPLEEN; SPLEEN SIZE; SYSTEMATIC REVIEW (TOPIC); THALASSEMIA MAJOR; WELLBEING;

ADOLESCENT; ANEMIA, SICKLE CELL; ANIMALS; BLOOD TRANSFUSION; FEMALE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; MALE; THALASSEMIA; TREATMENT OUTCOME;

EID: 84901471467     PISSN: 00064971     EISSN: 15280020     Source Type: Journal    
DOI: 10.1182/blood-2013-01-435776     Document Type: Review

References (62)

1. Atkins D, Best D, Briss PA, et al; GRADE Working Group. Grading quality of evidence and strength of recommendations. BMJ. 2004;328(7454):1490.
2. Ohene-Frempong K, Weiner SJ, Sleeper LA, et al. Cerebrovascular accidents in sickle cell disease: rates and risk factors. Blood. 1998;91(1):288-294. (Pubitemid 28018756)
3. Kwiatkowski JL, Zimmerman RA, Pollock AN, et al. Silent infarcts in young children with sickle cell disease. Br J Haematol. 2009;146(3):300-305.
4. Vichinsky EP, Styles LA, Colangelo LH, Wright EC, Castro O, Nickerson B; Cooperative Study of Sickle Cell Disease. Acute chest syndrome in sickle cell disease: clinical presentation and course. Blood. 1997;89(5):1787-1792. (Pubitemid 27097475)
5. Lanzkron S, Carroll CP Jr, Haywood C Jr. Mortality rates and age at death from sickle cell disease: U.S., 1979-2005. Public Health Rep. 2013;128(2):110-116.
6. Steinberg MH, McCarthy WF, Castro O, et al; Investigators of the Multicenter Study of Hydroxyurea in Sickle Cell Anemia and MSH Patients' Follow-Up. The risks and benefits of long-term use of hydroxyurea in sickle cell anemia: a 17.5 year follow-up. Am J Hematol. 2010;85(6):403-408.
7. Voskaridou E, Christoulas D, Bilalis A, et al. The effect of prolonged administration of hydroxyurea on morbidity and mortality in adult patients with sickle cell syndromes: results of a 17-year, single-center trial (LaSHS). Blood. 2010;115(12):2354-2363.
8. Wang WC, Ware RE, Miller ST, et al; BABY HUG investigators. Hydroxycarbamide in very young children with sickle-cell anaemia: a multicentre, randomised, controlled trial (BABY HUG). Lancet. 2011;377(9778):1663-1672.
9. Walters MC, Hardy K, Edwards S, et al; Multicenter Study of Bone Marrow Transplantation for Sickle Cell Disease. Pulmonary, gonadal, and central nervous system status after bone marrow transplantation for sickle cell disease. Biol Blood Marrow Transplant. 2010;16(2):263-272.
10. Bernaudin F, Socie G, Kuentz M, et al; SFGM-TC. Long-term results of related myeloablative stemcell transplantation to cure sickle cell disease. Blood. 2007;110(7):2749-2756. (Pubitemid 47523201)
11. Vermylen C, Cornu G, Ferster A, et al. Haematopoietic stem cell transplantation for sickle cell anaemia: the first 50 patients transplanted in Belgium. Bone Marrow Transplant. 1998;22(1):1-6. (Pubitemid 28330528)
12. Walters MC, Patience M, Leisenring W, et al; Multicenter Investigation of Bone Marrow Transplantation for Sickle Cell Disease. Stable mixed hematopoietic chimerism after bone marrow transplantation for sickle cell anemia. Biol Blood Marrow Transplant. 2001;7(12):665-673. (Pubitemid 34055819)
13. Lucarelli G, Gaziev J, Isgrò A, et al. Allogeneic cellular gene therapy in hemoglobinopathies - evaluation of hematopoietic SCT in sickle cell anemia. Bone Marrow Transplant. 2012;47(2):227-230.
14. Horan JT, Liesveld JL, Fenton P, Blumberg N, Walters MC. Hematopoietic stem cell transplantation for multiply transfused patients with sickle cell disease and thalassemia after lowdose total body irradiation, fludarabine, and rabbit anti-thymocyte globulin. Bone Marrow Transplant. 2005;35(2):171-177. (Pubitemid 40185933)
15. Iannone R, Casella JF, Fuchs EJ, et al. Results of minimally toxic nonmyeloablative transplantation in patients with sickle cell anemia and betathalassemia. Biol Blood Marrow Transplant. 2003; 9(8):519-528. (Pubitemid 38351796)
16. Hsieh MM, Kang EM, Fitzhugh CD, et al. Allogeneic hematopoietic stem-cell transplantation for sickle cell disease. N Engl J Med. 2009;361(24):2309-2317.
17. Eggleston B, Patience M, Edwards S, et al; Multicenter Study of HCT for SCA. Effect of myeloablative bone marrow transplantation on growth in children with sickle cell anaemia: results of the multicenter study of haematopoietic cell transplantation for sickle cell anaemia. Br J Haematol. 2007;136(4):673-676. (Pubitemid 46148207)
18. Hoppe CC, Walters MC. Bone marrow transplantation in sickle cell anemia. Curr Opin Oncol. 2001;13(2):85-90. (Pubitemid 32242854)
19. Walters MC, Sullivan KM, Bernaudin F, et al. Neurologic complications after allogeneic marrow transplantation for sickle cell anemia. Blood. 1995;85(4):879-884.
20. Dallas MH, Triplett B, Shook DR, et al. Long-term outcome and evaluation of organ function in pediatric patients undergoing haploidentical and matched related hematopoietic cell transplantation for sickle cell disease. Biol Blood Marrow Transplant. 2013;19(5):820-830.
21. Hsieh MM, Wu CJ, Tisdale JF. In mixed hematopoietic chimerism, the donor red cells win. Haematologica. 2011;96(1):13-15.
22. Krishnamurti L, Kharbanda S, Biernacki MA, et al. Stable long-term donor engraftment following reduced-intensity hematopoietic cell transplantation for sickle cell disease. Biol Blood Marrow Transplant. 2008;14(11):1270-1278.
23. Hulbert ML, McKinstry RC, Lacey JL, et al. Silent cerebral infarcts occur despite regular blood transfusion therapy after first strokes in children with sickle cell disease. Blood. 2011;117(3):772-779.
24. Scothorn DJ, Price C, Schwartz D, et al. Risk of recurrent stroke in children with sickle cell disease receiving blood transfusion therapy for at least five years after initial stroke. J Pediatr. 2002; 140(3):348-354. (Pubitemid 34289713)
25. King AA, Strouse JJ, Rodeghier MJ, et al. Parent education and biologic factors influence on cognition in sickle cell anemia [published online ahead of print October 9, 2013]. Am J Hematol.
26. van Besien K, Bartholomew A, Stock W, et al. Fludarabine-based conditioning for allogeneic transplantation in adults with sickle cell disease. Bone Marrow Transplant. 2000;26(4):445-449. (Pubitemid 30649562)
27. Shenoy S, Grossman WJ, DiPersio J, et al. A novel reduced-intensity stem cell transplant regimen for nonmalignant disorders. Bone Marrow Transplant. 2005;35(4):345-352. (Pubitemid 40309479)
28. Kamani NR, Walters MC, Carter S, et al. Unrelated donor cord blood transplantation for children with severe sickle cell disease: results of one cohort from the phase II study from the Blood and Marrow Transplant Clinical Trials Network (BMT CTN). Biol Blood Marrow Transplant. 2012; 18(8):1265-1272.
29. Ruggeri A, Eapen M, Scaravadou A, et al; Eurocord Registry; Center for International Blood and Marrow Transplant Research; New York Blood Center. Umbilical cord blood transplantation for children with thalassemia and sickle cell disease. Biol Blood Marrow Transplant. 2011;17(9):1375-1382.
30. Bolaños-Meade J, Fuchs EJ, Luznik L, et al. HLAhaploidentical bone marrow transplantation with posttransplant cyclophosphamide expands the donor pool for patients with sickle cell disease. Blood. 2012;120(22):4285-4291.
31. Delvecchio M, Cavallo L. Growth and endocrine function in thalassemia major in childhood and adolescence. J Endocrinol Invest. 2010;33(1):61-68.
32. Lucarelli G, Galimberti M, Polchi P, et al. Marrow transplantation in patientswith advanced thalassemia. N Engl JMed. 1987;316(17):1050-1055. (Pubitemid 17047293)
33. Sabloff M, Chandy M, Wang Z, et al. HLAmatched sibling bone marrow transplantation for b-thalassemia major. Blood. 2011;117(5):1745-1750.
34. Mathews V, George B, Viswabandya A, et al. Improved clinical outcomes of high risk b thalassemia major patients undergoing a HLA matched related allogeneic stem cell transplant with a treosulfan based conditioning regimen and peripheral blood stem cell grafts. PLoS ONE. 2013;8(4):e61637.
35. Resnick IB, Aker M, Tsirigotis P, et al. Allogeneic stem cell transplantation from matched related and unrelated donors in thalassemia major patients using a reduced toxicity fludarabinebased regimen. Bone Marrow Transplant. 2007; 40(10):957-964. (Pubitemid 350048194)
36. Sodani P, Gaziev D, Polchi P, et al. New approach for bone marrow transplantation in class 3 thalassemic patients aged less than 17 years. Blood. 2004;104(4):1201-1203. (Pubitemid 39038044)
37. Galambrun C, Pondarré C, Bertrand Y, et al; French Rare Disease Center for Thalassemia; French Society of Bone Marrow Transplantation. French multicenter 22-year experience in stem cell transplantation for betathalassemia major: lessons and future directions. Biol Blood Marrow Transplant. 2013;19(1):62-68.
38. Gaziev J, Isgrò A, Marziali M, et al. Higher CD3(1) and CD34(1) cell doses in the graft increase the incidence of acute GVHD in children receiving BMT for thalassemia. Bone Marrow Transplant. 2012; 47(1):107-114.
39. Ball LM, Lankester AC, Giordano PC, et al. Paediatric allogeneic bone marrow transplantation for homozygous beta-thalassaemia, the Dutch experience. Bone Marrow Transplant. 2003; 31(12):1081-1087. (Pubitemid 36857365)
40. Yesilipek MA, Ertem M, Cetin M, et al. HLA-matched family hematopoetic stem cell transplantation in children with beta thalassemia major: the experience of the Turkish Pediatric Bone Marrow Transplantation Group. Pediatr Transplant. 2012;16(8):846-851.
41. Chiesa R, Cappelli B, Crocchiolo R, et al. Unpredictability of intravenous busulfan pharmacokinetics in children undergoing hematopoietic stem cell transplantation for advanced beta thalassemia: limited toxicity with a dose-adjustment policy. Biol Blood Marrow Transplant. 2010;16(5):622-628.
42. Choudhary D, Sharma SK, Gupta N, et al. Treosulfan-thiotepa-fludarabine- based conditioning regimen for allogeneic transplantation in patients with thalassemia major: a single-center experience from north India. Biol Blood Marrow Transplant. 2013;19(3):492-495.
43. Hussein AA, Al-Zaben A, Ghatasheh L, et al. Risk adopted allogeneic hematopoietic stem cell transplantation using a reduced intensity regimen for children with thalassemia major. Pediatr Blood Cancer. 2013;60(8):1345-1349.
44. Lawson SE, Roberts IA, Amrolia P, Dokal I, Szydlo R, Darbyshire PJ. Bone marrow transplantation for beta-thalassaemia major: the UK experience in two paediatric centres. Br J Haematol. 2003;120(2):289-295. (Pubitemid 36194180)
45. Locatelli F, Kabbara N, Ruggeri A, et al; Eurocord and European Blood and Marrow Transplantation (EBMT) group. Outcome of patients with hemoglobinopathies given either cord blood or bone marrow transplantation from an HLAidentical sibling. Blood. 2013;122(6):1072-1078.
46. La Nasa G, Argiolu F, Giardini C, et al. Unrelated bone marrow transplantation for beta-thalassemia patients: the experience of the Italian Bone Marrow Transplant Group. Ann N Y Acad Sci. 2005;1054:186-195. (Pubitemid 43031024)
47. Lucarelli G, Clift RA, Galimberti M, et al. Marrow transplantation for patients with thalassemia: results in class 3 patients. Blood. 1996;87(5):2082-2088. (Pubitemid 26068926)
48. Bernardo ME, Piras E, Vacca A, et al. Allogeneic hematopoietic stem cell transplantation in thalassemia major: results of a reduced-toxicity conditioning regimen based on the use of treosulfan. Blood. 2012;120(2):473-476.
49. Anurathapan U, Pakakasama S, Rujkiyanont P, et al. Pharmacalogic Immunoablation followed by reduced-toxicity conditioning and stem cell transplantation in high-risk thalassemia: a safe approach to disease control. Biol Blood Marrow Transplant. 2013;19(8):1259-1262.
50. Li C, Wu X, Feng X, et al. A novel conditioning regimen improves outcomes in b-thalassemia major patients using unrelated donor peripheral blood stem cell transplantation. Blood. 2012; 120(19):3875-3881.
51. Jaing TH, Hung IJ, Yang CP, et al. Unrelated cord blood transplantation for thalassaemia: a singleinstitution experience of 35 patients. Bone Marrow Transplant. 2012;47(1):33-39.
52. Sodani P, Isgrò A, Gaziev J, et al. T cell-depleted hla-haploidentical stem cell transplantation in thalassemia young patients. Pediatr Rep. 2011; 3(suppl 2):e13.
53. Haywood CJ Jr, Beach MC, Lanzkron S, et al. A systematic review of barriers and interventions to improve appropriate use of therapies for sickle cell disease. J Natl Med Assoc. 2009;101(10):1022-1033.
54. Hansbury EN, SchultzWH,Ware RE, Aygun B. Bone marrow transplant options and preferences in a sickle cell anemia cohort on chronic transfusions. Pediatr Blood Cancer. 2012;58(4):611-615.
55. Roth M, Krystal J, Manwani D, Driscoll C, Ricafort R. Stem cell transplant for children with sickle cell anemia: parent and patient interest. Biol Blood Marrow Transplant. 2012;18(11):1709-1715.
56. O'Brien SH, Hankins JS. Decision analysis of treatment strategies in children with severe sickle cell disease. J Pediatr Hematol Oncol. 2009; 31(11):873-878.
57. Nietert P, Abboud M, Silverstein M, Jackson S. Bone marrow transplantation versus periodic prophylactic blood transfusion in sickle cell patients at high risk of ischemic stroke: a decision analysis. Blood. 2000;15;95(10):3057-3064. (Pubitemid 30321154)
58. Amendah DD, Mvundura M, Kavanagh PL, Sprinz PG, Grosse SD. Sickle cell disease-related pediatric medical expenditures in the U.S. Am J Prev Med. 2010;38(4 Suppl):S550-S556.
59. Wayne AS, Schoenike SE, Pegelow CH. Financial analysis of chronic transfusion for stroke prevention in sickle cell disease. Blood. 2000; 96(7):2369-2372.
60. Caocci G, Efficace F, Ciotti F, et al. Prospective assessment of health-related quality of life in pediatric patients with beta-thalassemia following hematopoietic stem cell transplantation. Biol Blood Marrow Transplant. 2011;17(6):861-866.
61. 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.
62. Sruamsiri R, Chaiyakunapruk N, Pakakasama S, et al. Cost utility analysis of reduced intensity hematopoietic stem cell transplantation in adolescence and young adult with severe thalassemia compared to hypertransfusion and iron chelation program. BMC Health Serv Res. 2013;13:45.