Anemia falciforme

Bain BJ, Daniel Y, Henthorn J, et al. Significant haemoglobinopathies: a guideline for screening and diagnosis: a British Society for Haematology guideline. Br J Haematol. 2023 Jun;201(6):1047-65.Texto completo  Resumo

DeBaun MR, Jordan LC, King AA, et al. American Society of Hematology 2020 guidelines for sickle cell disease: prevention, diagnosis, and treatment of cerebrovascular disease in children and adults. Blood Adv. 2020 Apr 28;4(8):1554-88.Texto completo  Resumo

National Heart, Lung, and Blood Institute. Evidence-based management of sickle cell disease: expert panel report, 2014. Sep 2014 [internet publication].Texto completo

Chou ST, Alsawas M, Fasano RM, et al. American Society of Hematology 2020 guidelines for sickle cell disease: transfusion support. Blood Adv. 2020 Jan 28;4(2):327-55.Texto completo  Resumo

Trompeter S, Massey E, Robinson S, et al. Position paper on International Collaboration for Transfusion Medicine (ICTM) Guideline 'Red blood cell specifications for patients with hemoglobinopathies: a systematic review and guideline'. Br J Haematol. 2020 May;189(3):424-27.Texto completo  Resumo

Wethers DL. Sickle cell disease in childhood: part I. Laboratory diagnosis, pathophysiology and health maintenance. Am Fam Physician. 2000 Sep 1;62(5):1013-20, 1027-8.Texto completo  Resumo

1. Pass KA, Lane PA, Fernhoff PM, et al. US newborn screening system guidelines II: follow-up of children, diagnosis, management, and evaluation. Statement of the Council of Regional Networks for Genetic Services (CORN). J Pediatr. 2000 Oct;137(suppl 4):S1-46. Resumo

2. Dormandy E, James J, Inusa B, et al. How many people have sickle cell disease in the UK? J Public Health (Oxf). 2018 Sep 1;40(3):e291-5.Texto completo  Resumo

3. National Health Service (UK). NHS sickle cell and thalassaemia (SCT) screening programme. Oct 2023 [internet publication].Texto completo

4. Centers for Disease Control and Prevention (CDC). Data and statistics on sickle cell disease. May 2024 [internet publication].Texto completo

5. Piel FB, Hay SI, Gupta S, et al. Global burden of sickle cell anaemia in children under five, 2010-2050: modelling based on demographics, excess mortality, and interventions. PLoS Med. 2013;10(7):e1001484.Texto completo  Resumo

6. Piel FB, Patil AP, Howes RE, et al. Global epidemiology of sickle haemoglobin in neonates: a contemporary geostatistical model-based map and population estimates. Lancet. 2013 Jan 12;381(9861):142-51.Texto completo  Resumo

7. GBD 2021 Sickle Cell Disease Collaborators. Global, regional, and national prevalence and mortality burden of sickle cell disease, 2000-2021: a systematic analysis from the Global Burden of Disease Study 2021. Lancet Haematol. 2023 Aug;10(8):e585-99.Texto completo  Resumo

8. Sundd P, Gladwin MT, Novelli EM. Pathophysiology of sickle cell disease. Annu Rev Pathol. 2019 Jan 24;14:263-92.Texto completo  Resumo

9. Piel FB, Steinberg MH, Rees DC. Sickle cell disease. N Engl J Med. 2017 Apr 20;376(16):1561-73.Texto completo  Resumo

10. Hebbel RP, Yamada O, Moldow CF, et al. Abnormal adherence of sickle erythrocytes to cultured vascular endothelium: possible mechanism for microvascular occlusion in sickle cell disease. J Clin Invest. 1980 Jan;65(1):154-60.Texto completo  Resumo

11. Hebbel RP, Osarogiagbon R, Kaul D. The endothelial biology of sickle cell disease: inflammation and a chronic vasculopathy. Microcirculation. 2004 Mar;11(2):129-51. Resumo

12. Yale SH, Nagib N, Guthrie T. Approach to the vaso-occlusive crisis in adults with sickle cell disease. Am Fam Physician. 2000 Mar 1;61(5):1349-56, 1363-4. Resumo

13. Kato GJ, Onyekwere OC, Gladwin MT. Pulmonary hypertension in sickle cell disease: relevance to children. Pediatr Hematol Oncol. 2007 Apr-May;24(3):159-70.Texto completo  Resumo

14. Naik RP, Smith-Whitley K, Hassell KL, et al. Clinical outcomes associated with sickle cell trait: a systematic review. Ann Intern Med. 2018 Nov 6;169(9):619-27.Texto completo  Resumo

15. Bain BJ, Daniel Y, Henthorn J, et al. Significant haemoglobinopathies: a guideline for screening and diagnosis: a British Society for Haematology guideline. Br J Haematol. 2023 Jun;201(6):1047-65.Texto completo  Resumo

16. The American College of Obstetricians and Gynecologists. ACOG practice bulletin No. 78: hemoglobinopathies in pregnancy. Aug 2022 [internet publication].Texto completo

17. The American College of Obstetricians and Gynecologists. Committee opinion no. 690: carrier screening in the age of genomic medicine. Mar 2017 [internet publication].Texto completo  Resumo

18. American College of Obstetricians and Gynecologists. Committee opinion no. 691: carrier screening for genetic conditions. Mar 2017 [internet publication].Texto completo  Resumo

19. Dormandy E, Gulliford M, Bryan S, et al. Effectiveness of earlier antenatal screening for sickle cell disease and thalassaemia in primary care: cluster randomised trial. 2010 Oct 5;341:c5132.Texto completo  Resumo

20. US Preventive Services Task Force. Sickle cell disease (hemoglobinopathies) in newborns: screening - referred topic. Sep 2007 [internet publication].Texto completo

21. Simundic AM, Bölenius K, Cadamuro J, et al. Joint EFLM-COLABIOCLI recommendation for venous blood sampling. Clin Chem Lab Med 2018;56(12):2015-38. Resumo

22. Gill FM, Sleeper LA, Weiner SJ, et al; Cooperative Study of Sickle Cell Disease. Clinical events in the first decade in a cohort of infants with sickle cell disease. Blood. 1995 Jul 15;86(2):776-83.Texto completo  Resumo

23. Miller ST, Sleeper LA, Pegelow CH, et al. Prediction of adverse outcomes in children with sickle cell disease. N Engl J Med. 2000 Jan 13;342(2):83-9.Texto completo  Resumo

24. Gualandro SF, Fonseca GH, Yokomizo IK, et al. Cohort study of adult patients with haemoglobin SC disease: clinical characteristics and predictors of mortality. Br J Haematol. 2015 Nov;171(4):631-7.Texto completo  Resumo

25. Mukisi-Mukaza M, Elbaz A, Samuel-Leborgne Y, et al. Prevalence, clinical features, and risk factors of osteonecrosis of the femoral head among adults with sickle cell disease. Orthopedics. 2000 Apr;23(4):357-63. Resumo

26. Centers for disease control and prevention (CDC). Hemoglobinopathies: current practices for screening, confirmation and follow-up. Dec 2015 [internet publication].Texto completo

27. Vosmaer A, Pereira RR, Koenderman JS, et al. Coagulation abnormalities in Legg-Calvé-Perthes disease. J Bone Joint Surg Am. 2010 Jan;92(1):121-8. Resumo

28. Public Health England. Sickle cell and thalassaemia (SCT) screening: programme overview. Aug 2023 [internet publication].Texto completo

29. Public Health England. Newborn blood spot screening: programme overview. Nov 2018 [internet publication].Texto completo

30. Eckman J. Neonatal screening. In: Embry SH, Hebbel RP, Mohandas N, et al, eds. Sickle cell disease: basic principles and clinical practice. New York, NY: Raven Press Ltd.; 1994.

31. DeBaun MR, Jordan LC, King AA, et al. American Society of Hematology 2020 guidelines for sickle cell disease: prevention, diagnosis, and treatment of cerebrovascular disease in children and adults. Blood Adv. 2020 Apr 28;4(8):1554-88.Texto completo  Resumo

32. Cherry MG, Greenhalgh J, Osipenko L, et al. The clinical effectiveness and cost-effectiveness of primary stroke prevention in children with sickle cell disease: a systematic review and economic evaluation. Health Technol Assess. 2012;16(43):1-129.Texto completo  Resumo

33. Alexandrov AV, Sloan MA, Tegeler CH, et al; American Society of Neuroimaging Practice Guidelines Committee. Practice standards for transcranial Doppler (TCD) ultrasound. Part II. Clinical indications and expected outcomes. J Neuroimaging. 2012 Jul;22(3):215-24. Resumo

34. Pecker LH, Sharma D, Nero A, et al. Knowledge gaps in reproductive and sexual health in girls and women with sickle cell disease. Br J Haematol. 2021 Sep;194(6):970-79.Texto completo  Resumo

35. National Heart, Lung, and Blood Institute. Evidence-based management of sickle cell disease: expert panel report, 2014. Sep 2014 [internet publication].Texto completo

36. Asnani MR, Quimby KR, Bennett NR, et al. Interventions for patients and caregivers to improve knowledge of sickle cell disease and recognition of its related complications. Cochrane Database Syst Rev. 2016 Oct 6;(10):CD011175.Texto completo  Resumo

37. Rankine-Mullings AE, Owusu-Ofori S. Prophylactic antibiotics for preventing pneumococcal infection in children with sickle cell disease. Cochrane Database Syst Rev. 2021 Mar 8;(3):CD003427.Texto completo  Resumo

38. Dekker LH, Fijnvandraat K, Brabin BJ, et al. Micronutrients and sickle cell disease, effects on growth, infection and vaso-occlusive crisis: a systematic review. Pediatr Blood Cancer. 2012 Aug;59(2):211-5. Resumo

39. Platt OS, Brambilla DJ, Rosse WF, et al. Mortality in sickle cell disease - life expectancy and risk factors for early death. N Engl J Med. 1994 Jun 9;330(23):1639-44.Texto completo  Resumo

40. Redwood AM, Williams EM, Desai P, et al. Climate and painful crisis of sickle-cell disease in Jamaica. BMJ. 1976 Jan 10;1(6001):66-8.Texto completo  Resumo

41. Mohan J, Marshall JM, Reid HL, et al. Peripheral vascular response to mild indirect cooling in patients with homozygous sickle cell (SS) disease and the frequency of painful crisis. Clin Sci (Lond). 1998 Feb;94(2):111-20. Resumo

42. Beutler E. Disorders of hemoglobin structure: sickle cell anemia and related abnormalities. In: Lichtman MA, Beutler E, Kaushansky K, et al, eds. Williams hematology. 7th ed. New York, NY: McGraw-Hill; 2006:667-700.

43. Charache S, Terrin ML, Moore RD, et al. Effect of hydroxyurea on the frequency of painful crises in sickle cell anemia. N Engl J Med. 1995 May 18;332(20):1317-22.Texto completo  Resumo

44. Lanzkron S, Strouse JJ, Wilson R, et al. Systematic review: hydroxyurea for the treatment of adults with sickle cell disease. Ann Intern Med. 2008 Jun 17;148(12):939-55.Texto completo  Resumo

45. Segal JB, Strouse JJ, Beach MC, et al. Hydroxyurea for the treatment of sickle cell disease. Evid Rep Technol Assess (Full Rep). 2008 Mar;(165):1-95. Resumo

46. Hankins JS, Ware RE, Rogers ZR, et al. Long-term hydroxyurea therapy for infants with sickle cell anemia: the HUSOFT extension study. Blood. 2005 Oct 1;106(7):2269-75.Texto completo  Resumo

47. Scott JP, Hillery CA, Brown ER, et al. Hydroxyurea therapy in children severely affected with sickle cell disease. J Pediatr. 1996 Jun;128(6):820-8. Resumo

48. Wang WC, Helms RW, Lynn HS, et al. Effect of hydroxyurea on growth in children with sickle cell anemia: results of the HUG-KIDS Study. J Pediatr. 2002 Feb;140(2):225-9. Resumo

49. 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 May 14;377(9778):1663-72.Texto completo  Resumo

50. Zimmerman SA, Schultz WH, Davis JS, et al. Sustained long-term hematologic efficacy of hydroxyurea at maximum tolerated dose in children with sickle cell disease. Blood. 2004 Mar 15;103(6):2039-45.Texto completo  Resumo

51. Rankine-Mullings AE, Nevitt SJ. Hydroxyurea (hydroxycarbamide) for sickle cell disease. Cochrane Database Syst Rev. 2022 Sep 1;9(9):CD002202.Texto completo  Resumo

52. Ware RE, Helms RW; SWiTCH Investigators. Stroke with transfusions changing to hydroxyurea (SWiTCH). Blood. 2012 Apr 26;119(17):3925-32.Texto completo  Resumo

53. Kinney TR, Helms RW, O'Branski EE, et al. Safety of hydroxyurea in children with sickle cell anemia: results of the HUG-KIDS study, a phase I/II trial. Pediatric Hydroxyurea Group. Blood. 1999 Sep 1;94(5):1550-4.Texto completo  Resumo

54. Steinberg MH, Barton F, Castro O, et al. Effect of hydroxyurea on mortality and morbidity in adult sickle cell anemia: risks and benefits up to 9 years of treatment. JAMA. 2003 Apr 2;289(13):1645-51.Texto completo  Resumo

55. 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 Mar 25;115(12):2354-63.Texto completo  Resumo

56. Steinberg MH, McCarthy WF, Castro O, et al. The risks and benefits of long-term use of hydroxyurea in sickle cell anemia: a 17.5 year follow-up. Am J Hematol. 2010 Jun;85(6):403-8.Texto completo  Resumo

57. Cieri-Hutcherson NE, Hutcherson TC, Conway-Habes EE, et al. Systematic review of i-glutamine for prevention of vaso-occlusive pain crisis in patients with sickle cell disease. Pharmacotherapy. 2019 Nov;39(11):1095-1104. Resumo

58. Niihara Y, Miller ST, Kanter J, et al. A phase 3 trial of L-glutamine in sickle cell disease. N Engl J Med. 2018 Jul 19;379(3):226-35. Resumo

59. Quinn CT. L-glutamine for sickle cell anemia: more questions than answers. Blood. 2018 Aug 16;132(7):689-93. Resumo

60. ClinicalTrials.gov. Study of two doses of crizanlizumab versus placebo in adolescent and adult sickle cell disease patients (STAND). ClinicalTrials.gov Identifier: NCT03814746. May 2024 [internet publication].Texto completo

61. Ataga KI, Kutlar A, Kanter J, et al. Crizanlizumab for the prevention of pain crises in sickle cell disease. N Engl J Med. 2017 Feb 2;376(5):429-39.Texto completo  Resumo

62. Kutlar A, Kanter J, Liles DK, et al. Effect of crizanlizumab on pain crises in subgroups of patients with sickle cell disease: a SUSTAIN study analysis. Am J Hematol. 2019 Jan;94(1):55-61.Texto completo  Resumo

63. Han J, Saraf SL, Gordeuk VR. Systematic review of crizanlizumab: a new parenteral option to reduce vaso-occlusive pain crises in patients with sickle cell disease. Pharmacotherapy. 2020 Jun;40(6):535-43. Resumo

64. Vichinsky EP, Haberkern CM, Neumayr L, et al; The Preoperative Transfusion in Sickle Cell Disease Study Group. A comparison of conservative and aggressive transfusion regimens in the perioperative management of sickle cell disease. N Engl J Med. 1995 Jul 27;333(4):206-13.Texto completo  Resumo

65. Estcourt LJ, Kimber C, Trivella M, et al. Preoperative blood transfusions for sickle cell disease. Cochrane Database Syst Rev. 2020 Jul 2;(7):CD003149.Texto completo  Resumo

66. Howard J, Malfroy M, Llewelyn C, et al. The Transfusion Alternatives Preoperatively in Sickle Cell Disease (TAPS) study: a randomised, controlled, multicentre clinical trial. Lancet. 2013 Mar 16;381(9870):930-8. Resumo

67. Lee MT, Piomelli S, Granger S, et al. Stroke Prevention Trial in Sickle Cell Anemia (STOP): extended follow-up and final results. Blood. 2006 Aug 1;108(3):847-52.Texto completo  Resumo

68. Adams RJ, Brambilla D. Discontinuing prophylactic transfusions used to prevent stroke in sickle cell disease. N Engl J Med. 2005 Dec 29;353(26):2769-78.Texto completo  Resumo

69. Compernolle V, Chou ST, Tanael S, et al. Red blood cell specifications for patients with hemoglobinopathies: a systematic review and guideline. Transfusion. 2018 Jun;58(6):1555-66. Resumo

70. Chou ST, Alsawas M, Fasano RM, et al. American Society of Hematology 2020 guidelines for sickle cell disease: transfusion support. Blood Adv. 2020 Jan 28;4(2):327-55.Texto completo  Resumo

71. Trompeter S, Massey E, Robinson S, et al. Position paper on International Collaboration for Transfusion Medicine (ICTM) Guideline 'Red blood cell specifications for patients with hemoglobinopathies: a systematic review and guideline'. Br J Haematol. 2020 May;189(3):424-27.Texto completo  Resumo

72. Fasano RM, Meyer EK, Branscomb J, et al. Impact of red blood cell antigen matching on alloimmunization and transfusion complications in patients with sickle cell disease: a systematic review. Transfus Med Rev. 2019 Jan;33(1):12-23. Resumo

73. Oteng-Ntim E, Pavord S, Howard R, et al. Management of sickle cell disease in pregnancy. A British Society for Haematology Guideline. Br J Haematol. 2021 Sep;194(6):980-95.Texto completo  Resumo

74. Boafor TK, Olayemi E, Galadanci N, et al. Pregnancy outcomes in women with sickle-cell disease in low and high income countries: a systematic review and meta-analysis. BJOG. 2016 Apr;123(5):691-8. Resumo

75. Malinowski AK, Shehata N, D'Souza R, et al. Prophylactic transfusion for pregnant women with sickle cell disease: a systematic review and meta-analysis. Blood. 2015 Nov 19;126(21):2424-35.Texto completo  Resumo

76. Food and Drug Administration (FDA). FDA is alerting patients and health care professionals about the voluntary withdrawal of Oxbryta from the market due to safety concerns. September 2024 [internet publication].Texto completo

77. European Medicines Agency (EMA). EMA recommends suspension of sickle cell disease medicine Oxbryta. September 2024 [internet publication].Texto completo

78. Brandow AM, Carroll CP, Creary S, et al. American Society of Hematology 2020 guidelines for sickle cell disease: management of acute and chronic pain. Blood Adv. 2020 Jun 23;4(12):2656-2701.Texto completo  Resumo

79. Jacobson SJ, Kopecky EA, Joshi P, et al. Randomised trial of oral morphine for painful episodes of sickle-cell disease in children. Lancet. 1997 Nov 8;350(9088):1358-61. Resumo

80. Cooper TE, Hambleton IR, Ballas SK, et al. Pharmacological interventions for painful sickle cell vaso-occlusive crises in adults. Cochrane Database Syst Rev. 2019 Nov 14;2019(11):CD012187.Texto completo  Resumo

81. Aboursheid T, Albaroudi O, Alahdab F. Inhaled nitric oxide for treating pain crises in people with sickle cell disease. Cochrane Database Syst Rev. 2019 Oct 11;(10):CD011808.Texto completo  Resumo

82. Dampier CD, Wager CG, Harrison R, et al; Investigators of the Sickle Cell Disease Clinical Research Network (SCDCRN). Impact of PCA strategies on pain intensity and functional assessment measures in adults with sickle cell disease during hospitalized vaso-occlusive episodes. Am J Hematol. 2012 Oct;87(10):E71-4.Texto completo  Resumo

83. Vichinsky EP, Neumayr LD, Earles AN, et al. Causes and outcomes of the acute chest syndrome in sickle cell disease. National Acute Chest Syndrome Study Group. N Engl J Med. 2000 Jun 22;342(25):1855-65.Texto completo  Resumo

84. Dolatkhah R, Dastgiri S. Blood transfusions for treating acute chest syndrome in people with sickle cell disease. Cochrane Database Syst Rev. 2020 Jan 16;(1):CD007843.Texto completo  Resumo

85. Bundy DG, Richardson TE, Hall M, et al. Association of guideline-adherent antibiotic treatment with readmission of children with sickle cell disease hospitalized with acute chest syndrome. JAMA Pediatr. 2017 Nov 1;171(11):1090-9.Texto completo  Resumo

86. Kanter J, Liem RI, Bernaudin F, et al. American Society of Hematology 2021 guidelines for sickle cell disease: stem cell transplantation. Blood Adv. 2021 Sep 28;5(18):3668-89.Texto completo  Resumo

87. Oringanje C, Nemecek E, Oniyangi O. Hematopoietic stem cell transplantation for people with sickle cell disease. Cochrane Database Syst Rev. 2020 Jul 3;(7):CD007001.Texto completo  Resumo

88. Shenoy S, Angelucci E, Arnold SD, et al. Current results and future research priorities in late effects after hematopoietic stem cell transplantation for children with sickle cell disease and thalassemia: a consensus statement from the Second Pediatric Blood and Marrow Transplant Consortium International Conference on Late Effects after Pediatric Hematopoietic Stem Cell Transplantation. Biol Blood Marrow Transplant. 2017 Apr;23(4):552-61.Texto completo  Resumo

89. Smith WR, Penberthy LT, Bovbjerg VE, et al. Daily assessment of pain in adults with sickle cell disease. Ann Intern Med. 2008 Jan 15;148(2):94-101. Resumo

90. Anie KA, Green J. Psychological therapies for sickle cell disease and pain. Cochrane Database Syst Rev. 2015 May 8;(5):CD001916.Texto completo  Resumo

91. Webster J, Osborne S, Rickard CM, et al. Clinically-indicated replacement versus routine replacement of peripheral venous catheters. Cochrane Database Syst Rev. 2019 Jan 23;1(1):CD007798.Texto completo  Resumo

92. Martí-Carvajal AJ, Solà I, Agreda-Pérez LH. Treatment for avascular necrosis of bone in people with sickle cell disease. Cochrane Database Syst Rev. 2019 Dec 5;(12):CD004344.Texto completo  Resumo

93. Pfizer. ​Pfizer voluntarily withdraws all lots of sickle cell disease treatment OXBRYTA® (voxelotor) from worldwide markets. Sep 2024 [internet publication].Texto completo

94. US Food and Drug Administration. FDA drug safety communication: FDA restricts use of prescription codeine pain and cough medicines and tramadol pain medicines in children; recommends against use in breastfeeding women. Apr 2017 [internet publication].Texto completo

95. Medicines and Healthcare Products Regulatory Agency. Codeine: restricted use as analgesic in children and adolescents after European safety review. Jun 2013 [internet publication].Texto completo

96. European Medicines Agency. Restrictions on use of codeine for pain relief in children - CMDh endorses PRAC recommendation. Jun 2013 [internet publication].Texto completo

97. Frangoul H, Altshuler D, Cappellini MD, et al. CRISPR-Cas9 gene editing for sickle cell disease and beta-thalassemia. N Engl J Med. 2021 Jan 21;384(3):252-60.Texto completo  Resumo

98. ​ClinicalTrials.gov. A safety and efficacy study evaluating CTX001 in subjects with severe sickle cell disease. ClinicalTrials.gov ID: NCT03745287. Nov 2023 [internet publication].Texto completo

99. Locatelli F, Frangoul H, Corbacioglu S, et al. Efficacy and safety of a single dose of CTX001 for transfusion-dependent beta-thalassemia and severe sicle cell disease. Paper presented at: European Hematology Association Congress 2022. Vienna, Austria.Texto completo

100. ​ClinicalTrials.gov. A long-term follow-up study in subjects who received CTX001. ClinicalTrials.gov ID: NCT04208529. Nov 2022 [internet publiction].Texto completo

101. ​ClinicalTrials.gov. Evaluation of safety and efficacy of CTX001 in pediatric participants with severe sickle cell disease (SCD). ClinicalTrials.gov ID: NCT05329649. Aug 2023 [internet publication].Texto completo

102. Kanter J, Walters MC, Krishnamurti L, et al. Biologic and clinical efficacy of LentiGlobin for sickle cell disease. N Engl J Med. 2022 Feb 17;386(7):617-28.Texto completo  Resumo

103. ​ClinicalTrials.gov. A study evaluating gene therapy with BB305 Lentiviral vector in sickle cell disease. ClinicalTrials.gov ID: NCT04293185. Sep 2023 [internet publication].Texto completo

104. McArthur JG, Svenstrup N, Chen C, et al. A novel, highly potent and selective phosphodiesterase-9 inhibitor for the treatment of sickle cell disease. Haematologica. 2020 Mar; 105(3):623–31.Texto completo  Resumo

105. U.S. National library of medicine. Clinical trials. A study of IMR-687 in subjects with sickle cell disease. Jul 2020 [internet publication].Texto completo

106. ClinicalTrials.gov. A study evaluating the efficacy and safety of Mitapivat (AG-348) in participants with sickle cell disease. ClinicalTrials.gov Identifier: NCT05031780. Mar 2022 [internet publication].Texto completo

107. Rab MAE, Van Oirschot BA, Kosinski PA, et al. AG-348 (Mitapivat), an allosteric activator of red blood cell pyruvate kinase, increases enzymatic activity, protein stability, and ATP levels over a broad range of PKLR genotypes. Haematologica. 2021 Jan 1;106(1):238-49.Texto completo  Resumo

108. Jiao B, Johnson KM, Ramsey SD, et al. Long-term survival with sickle cell disease: a nationwide cohort study of Medicare and Medicaid beneficiaries. Blood Adv. 2023 Jul 11;7(13):3276-83.Texto completo  Resumo

109. Elmariah H, Garrett ME, De Castro LM, et al. Factors associated with survival in a contemporary adult sickle cell disease cohort. Am J Hematol. 2014 May;89(5):530-5.Texto completo  Resumo

110. Grosse SD, Odame I, Atrash HK, et al. Sickle cell disease in Africa: a neglected cause of early childhood mortality. Am J Prev Med. 2011 Dec;41(6 suppl 4):S398-405.Texto completo  Resumo

111. Ohene-Frempong K, Weiner SJ, Sleeper LA, et al. Cerebrovascular accidents in sickle cell disease: rates and risk factors. Blood. 1998 Jan 1;91(1):288-94.Texto completo  Resumo

112. Stockman JA, Nigro MA, Mishkin MM, et al. Occlusion of large cerebral vessels in sickle-cell anemia. N Engl J Med. 1972 Oct 26;287(17):846-9. Resumo

113. Ware RE, Davis BR, Schultz WH, et al. Hydroxycarbamide versus chronic transfusion for maintenance of transcranial doppler flow velocities in children with sickle cell anaemia-TCD With Transfusions Changing to Hydroxyurea (TWiTCH): a multicentre, open-label, phase 3, non-inferiority trial. Lancet. 2016 Feb 13;387(10019):661-70.Texto completo  Resumo

114. Miller ST, Jensen D, Rao SP. Less intensive long-term transfusion therapy for sickle cell anemia and cerebrovascular accident. J Pediatr. 1992 Jan;120(1):54-7. Resumo

115. Estcourt LJ, Kohli R, Hopewell S, et al. Blood transfusion for preventing primary and secondary stroke in people with sickle cell disease. Cochrane Database Syst Rev. 2020 Jul 27;(7):CD003146.Texto completo  Resumo

116. 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 Jan 20;117(3):772-9.Texto completo  Resumo

117. Estcourt LJ, Kimber C, Hopewell S, et al. Interventions for preventing silent cerebral infarcts in people with sickle cell disease. Cochrane Database Syst Rev. 2020 Apr 6;(4):CD012389.Texto completo  Resumo

118. Fortin PM, Hopewell S, Estcourt LJ. Red blood cell transfusion to treat or prevent complications in sickle cell disease: an overview of Cochrane reviews. Cochrane Database Syst Rev. 2018 Aug 1;(8):CD012082.Texto completo  Resumo

119. Vichinsky EP, Neumayr LD, Gold JI, et al; Neuropsychological Dysfunction and Neuroimaging Adult Sickle Cell Anemia Study Group. Neuropsychological dysfunction and neuroimaging abnormalities in neurologically intact adults with sickle cell anemia. JAMA. 2010 May 12;303(18):1823-31. Resumo

120. Krauss JS, Freant LJ, Lee JR. Gastrointestinal pathology in sickle cell disease. Ann Clin Lab Sci. 1998 Jan-Feb;28(1):19-23. Resumo

121. Omata M, Johnson CS, Tong M, et al. Pathological spectrum of liver diseases in sickle cell disease. Dig Dis Sci. 1986 Mar;31(3):247-56. Resumo

122. Schubert TT. Hepatobiliary system in sickle cell disease. Gastroenterology. 1986 Jun;90(6):2013-21. Resumo

123. Seguier-Lipszyc E, de Lagausie P, Benkerrou M, et al. Elective laparoscopic cholecystectomy. Surg Endosc. 2001 Mar;15(3):301-4. Resumo

124. Bonatsos G, Birbas K, Toutouzas K, et al. Laparoscopic cholecystectomy in adults with sickle cell disease. Surg Endosc. 2001 Aug;15(8):816-9. Resumo

125. Owusu-Ofori S, Remmington T. Splenectomy versus conservative management for acute sequestration crises in people with sickle cell disease. Cochrane Database Syst Rev. 2017 Nov 7;(11):CD003425.Texto completo  Resumo

126. Oligbu G, Fallaha M, Pay L, et al. Risk of invasive pneumococcal disease in children with sickle cell disease in the era of conjugate vaccines: a systematic review of the literature. Br J Haematol. 2019 May;185(4):743-751.Texto completo  Resumo

127. Claster S, Vichinsky EP. Managing sickle cell disease. BMJ. 2003 Nov 15;327(7424):1151-5.Texto completo  Resumo

128. Leitão Guerra RL, Leitão Guerra CL, Bastos MG, et al. Sickle cell retinopathy: What we now understand using optical coherence tomography angiography. A systematic review. Blood Rev. 2019 May;35:32-42. Resumo

129. Rodgers GP, Roy MS, Noguchi CT, et al. Is there a role for selective vasodilation in the management of sickle cell disease? Blood. 1988 Mar;71(3):597-602.Texto completo  Resumo

130. Myint KT, Sahoo S, Thein AW, et al. Laser therapy for retinopathy in sickle cell disease. Cochrane Database Syst Rev. 2015 Oct 9;(10):CD010790.Texto completo  Resumo

131. Morgan AG. Sickle cell leg ulcers. Int J Dermatol. 1985 Dec;24(10):643-4. Resumo

132. Castro O, Gladwin MT. Pulmonary hypertension in sickle cell disease: mechanisms, diagnosis, and management. Hematol Oncol Clin North Am. 2005 Oct;19(5):881-96. Resumo

133. Klings ES, Machado RF, Barst RJ, et al.; American Thoracic Society Ad Hoc Committee on Pulmonary Hypertension of Sickle Cell Disease. An official American Thoracic Society clinical practice guideline: diagnosis, risk stratification, and management of pulmonary hypertension of sickle cell disease. Am J Respir Crit Care Med. 2014 Mar 15;189(6):727-40. Resumo

134. Machado RF, Martyr S, Kato GJ, et al. Sildenafil therapy in patients with sickle cell disease and pulmonary hypertension. Br J Haematol. 2005 Aug;130(3):445-53.Texto completo  Resumo

135. Barst RJ, Rubin LJ, Long WA, et al. A comparison of continuous intravenous epoprostenol (prostacyclin) with conventional therapy for primary pulmonary hypertension. N Engl J Med. 1996 Feb 1;334(5):296-301.Texto completo  Resumo

136. McLaughlin VV, Genthner DE, Panella MM, et al. Compassionate use of continuous prostacyclin in the management of secondary pulmonary hypertension: a case series. Ann Intern Med. 1999 May 4;130(9):740-3. Resumo

137. Olaosebikan R, Ernest K, Bojang K, et al. A randomized trial to compare the safety, tolerability, and effectiveness of 3 antimalarial regimens for the prevention of malaria in Nigerian patients with sickle cell disease. J Infect Dis. 2015 Aug 15;212(4):617-25.Texto completo  Resumo

138. Liem RI, Lanzkron S, D Coates T, et al. American Society of Hematology 2019 guidelines for sickle cell disease: cardiopulmonary and kidney disease. Blood Adv. 2019 Dec 10;3(23):3867-97.Texto completo  Resumo

139. Mantadakis E, Cavender JD, Rogers ZR, et al. Prevalence of priapism in children and adolescents with sickle cell anemia. J Pediatr Hematol Oncol. 1999 Nov-Dec;21(6):518-22. Resumo

140. Arduini GAO, Trovó de Marqui AB. Prevalence and characteristics of priapism in sickle cell disease. Hemoglobin. 2018 Mar;42(2):73-77. Resumo

141. Chinegwundoh FI, Smith S, Anie KA. Treatments for priapism in boys and men with sickle cell disease. Cochrane Database Syst Rev. 2020 Apr 6;(4):CD004198.Texto completo  Resumo

142. Phebus CK, Gloninger MF, Maciak BJ. Growth patterns by age and sex in children with sickle cell disease. J Pediatr. 1984 Jul;105(1):28-33. Resumo

143. Fung EB, Harmatz PR, Milet M, et al. Disparity in the management of iron overload between patients with sickle cell disease and thalassemia who received transfusions. Transfusion. 2008 Sep;48(9):1971-80. Resumo

144. Shah FT, Porter JB, Sadasivam N, et al. Guidelines for the monitoring and management of iron overload in patients with haemoglobinopathies and rare anaemias. Br J Haematol. 2022 Jan;196(2):336-50.Texto completo  Resumo

145. Kwiatkowski JL, Hamdy M, El-Beshlawy A, et al. Deferiprone vs deferoxamine for transfusional iron overload in SCD and other anemias: a randomized, open-label noninferiority study. Blood Adv. 2022 Feb 22;6(4):1243-54.Texto completo  Resumo

146. Meerpohl JJ, Schell LK, Rücker G, et al. Deferasirox for managing transfusional iron overload in people with sickle cell disease. Cochrane Database Syst Rev. 2014 May 27;(5):CD007477.Texto completo  Resumo

147. Sridharan K, Sivaramakrishnan G. Efficacy and safety of iron chelators in thalassemia and sickle cell disease: a multiple treatment comparison network meta-analysis and trial sequential analysis. Expert Rev Clin Pharmacol. 2018 Jun;11(6):641-50. Resumo

148. Nelson DA, Deuster PA, Carter R 3rd, et al. Sickle cell trait, rhabdomyolysis, and mortality among U.S. Army Soldiers. N Engl J Med. 2016 Aug 4;375(5):435-42.Texto completo  Resumo

149. World Health Organization. Medical eligibility for contraceptive use. 5th ed. Geneva: World Health Organization; 2015.Texto completo

150. Wethers DL. Sickle cell disease in childhood: part I. Laboratory diagnosis, pathophysiology and health maintenance. Am Fam Physician. 2000 Sep 1;62(5):1013-20, 1027-8.Texto completo  Resumo

151. Lottenberg R, Hassell KL. An evidence-based approach to the treatment of adults with sickle cell disease. Hematology Am Soc Hematol Educ Program. 2005:58-65.Texto completo  Resumo

152. Haddad LB, Curtis KM, Legardy-Williams JK, et al. Contraception for individuals with sickle cell disease: a systematic review of the literature. Contraception. 2012 Jun;85(6):527-37. Resumo

153. UK Health Security Agency. The Green Book: immunisation against infectious disease. Nov 2020 [internet publication].Texto completo

154. ​Centers for Disease Control and Prevention. Influenza (flu): summary: 'prevention and control of seasonal influenza with vaccines: recommendations of the Advisory Committee on Immunization Practices (ACIP) - United States, 2023-24'. Aug 2023 [internet publication].Texto completo