Global burden of transfusion in sickle cell disease

Transfusion and Apheresis Science - Tập 62 - Trang 103764 - 2023
Baba PD Inusa1, Wale Atoyebi2, Biree Andemariam3, James N. Hourani4, Laurel Omert4
1Guy’s and Saint Thomas’ NHS Foundation Trust, London, UK
2Oxford University Hospitals, Oxford, UK
3New England Sickle Cell Institute, University of Connecticut Health, Farmington, CT, USA
4Hemanext, Inc., Lexington, MA, USA

Tài liệu tham khảo

Kaur, 2013, An overview on sickle cell disease profile, Asian J Pharm Clin Res, 6, 25 Herrick, 2001, Peculiar elongated and sickle-shaped red blood corpuscles in a case of severe anemia. 1910, Yale J Biol Med, 74, 179 Williams, 2018, Sickle cell anemia and its phenotypes, Annu Rev Genom Hum Genet, 19, 113, 10.1146/annurev-genom-083117-021320 World Health Assembly, 2006 Han, 2021, Indications for transfusion in the management of sickle cell disease, Hematol Am Soc Hematol Educ Program, 2021, 696, 10.1182/hematology.2021000307 Seck, 2022, Transfusion practice, post-transfusion complications and risk factors in sickle cell disease in Senegal, West Africa, Mediterr J Hematol Infect Dis, 14, 10.4084/MJHID.2022.004 Gyamfi, 2021, Evidence-based interventions implemented in low-and middle-income countries for sickle cell disease management: a systematic review of randomized controlled trials, PLoS One, 16, 10.1371/journal.pone.0246700 Wastnedge, 2018, The global burden of sickle cell disease in children under five years of age: a systematic review and meta-analysis, J Glob Health, 8, 10.7189/jogh.08.021103 Steinberg, 2008, Sickle cell anemia, the first molecular disease: overview of molecular etiology, pathophysiology, and therapeutic approaches, ScientificWorldJournal, 8, 1295, 10.1100/tsw.2008.157 Ashley-Koch, 2000, Sickle hemoglobin (HbS) allele and sickle cell disease: a HuGE review, Am J Epidemiol, 151, 839, 10.1093/oxfordjournals.aje.a010288 Kato, 2018, Sickle cell disease, Nat Rev Dis Prim, 4, 18010, 10.1038/nrdp.2018.10 Therrell, 2020, Empowering newborn screening programs in African countries through establishment of an international collaborative effort, J Community Genet, 11, 253, 10.1007/s12687-020-00463-7 Piel, 2013, Global burden of sickle cell anaemia in children under five, 2010-2050: modelling based on demographics, excess mortality, and interventions, PLoS Med, 10, 10.1371/journal.pmed.1001484 Grosse, 2011, Sickle cell disease in Africa: a neglected cause of early childhood mortality, Am J Prev Med, 41, S398, 10.1016/j.amepre.2011.09.013 Telfer, 2007, Clinical outcomes in children with sickle cell disease living in England: a neonatal cohort in East London, Haematologica, 92, 905, 10.3324/haematol.10937 Quinn, 2010, Improved survival of children and adolescents with sickle cell disease, Blood, 115, 3447, 10.1182/blood-2009-07-233700 Piel, 2013, Global epidemiology of sickle haemoglobin in neonates: a contemporary geostatistical model-based map and population estimates, Lancet, 381, 142, 10.1016/S0140-6736(12)61229-X United Nations DoEaSA, Population Division (2022). World Population Prospects 2022, Online Edition. Hassell, 2010, Population estimates of sickle cell disease in the U.S, Am J Prev Med, 38, S512, 10.1016/j.amepre.2009.12.022 Iolascon, 2019, EHA research roadmap on hemoglobinopathies and thalassemia: an update, Hemasphere, 3, 10.1097/HS9.0000000000000208 Aguilar Martinez, 2014, Haemoglobinopathies in Europe: health & migration policy perspectives, Orphanet J Rare Dis, 9, 97, 10.1186/1750-1172-9-97 Powell, 1992, Acute splenic sequestration crisis in sickle cell disease: early detection and treatment, J Pedia Surg, 27, 215, 10.1016/0022-3468(92)90315-X Hoppe, 2013, Prenatal and newborn screening for hemoglobinopathies, Int J Lab Hematol, 35, 297, 10.1111/ijlh.12076 Minkovitz, 2016, Newborn screening programs and sickle cell disease: a public health services and systems approach, Am J Prev Med, 51, S39, 10.1016/j.amepre.2016.02.019 Kuznik, 2016, Newborn screening and prophylactic interventions for sickle cell disease in 47 countries in sub-Saharan Africa: a cost-effectiveness analysis, BMC Health Serv Res, 16, 304, 10.1186/s12913-016-1572-6 Robitaille, 2006, Newborn screening for sickle cell disease: a 1988-2003 Quebec experience, Paediatr Child Health, 11, 223, 10.1093/pch/11.4.223 The National Institute for Health and Care Excellence. Sickle cell disease: What are the complications of sickle cell disease? 2021. p. March 02 2023. Veluswamy, 2019, Vaso-Occlusion in sickle cell disease: is autonomic dysregulation of the microvasculature the trigger?, J Clin Med, 8 Moerdler, 2018, New insights into the pathophysiology and development of novel therapies for sickle cell disease, Hematol Am Soc Hematol Educ Program, 2018, 493, 10.1182/asheducation-2018.1.493 Sundd, 2019, Pathophysiology of sickle cell disease, Annu Rev Pathol, 14, 263, 10.1146/annurev-pathmechdis-012418-012838 Chou, 2013, Transfusion therapy for sickle cell disease: a balancing act, Hematology, 2013, 439, 10.1182/asheducation-2013.1.439 Mehari, 2016, Chronic pulmonary complications of sickle cell disease, Chest, 149, 1313, 10.1016/j.chest.2015.11.016 Vichinsky, 2000, Causes and outcomes of the acute chest syndrome in sickle cell disease. National Acute Chest Syndrome Study Group, N Engl J Med, 342, 1855, 10.1056/NEJM200006223422502 Kanter, 2013, Management of sickle cell disease from childhood through adulthood, Blood Rev, 27, 279, 10.1016/j.blre.2013.09.001 Ballas, 2010, Definitions of the phenotypic manifestations of sickle cell disease, Am J Hematol, 85, 6, 10.1002/ajh.21550 Gladwin, 2004, Pulmonary hypertension as a risk factor for death in patients with sickle cell disease, N Engl J Med, 350, 886, 10.1056/NEJMoa035477 DeBaun, 2011, Secondary prevention of overt strokes in sickle cell disease: therapeutic strategies and efficacy, Hematol Am Soc Hematol Educ Program, 2011, 427, 10.1182/asheducation-2011.1.427 Hulbert, 2006, Exchange blood transfusion compared with simple transfusion for first overt stroke is associated with a lower risk of subsequent stroke: a retrospective cohort study of 137 children with sickle cell anemia, J Pedia, 149, 710, 10.1016/j.jpeds.2006.06.037 Powars, 1978, The natural history of stroke in sickle cell disease, Am J Med, 65, 461, 10.1016/0002-9343(78)90772-6 Migotsky, 2022, Recent advances in sickle-cell disease therapies: a review of voxelotor, crizanlizumab, and L-glutamine, Pharm (Basel), 10 Brandow, 2010, Hydroxyurea use in sickle cell disease: the battle with low prescription rates, poor patient compliance and fears of toxicities, Expert Rev Hematol, 3, 255, 10.1586/ehm.10.22 National Heart LaBI. Evidence-Based Management of Sickle Cell Disease: Expert Panel Report. 2014. Chou, 2020, American Society of Hematology 2020 guidelines for sickle cell disease: transfusion support, Blood Adv, 4, 327, 10.1182/bloodadvances.2019001143 World Health Organization. Blood safety and availability. Available at: https://www.who.int/news-room/fact-sheets/detail/blood-safety-and-availability (Accessed 23 June 23 2022). Wade, 2022, Procedural adverse events in pediatric patients with sickle cell disease undergoing chronic automated red cell exchange, Transfusion, 62, 584, 10.1111/trf.16807 Sarode, 2011, Advantages of isovolemic hemodilution-red cell exchange therapy to prevent recurrent stroke in sickle cell anemia patients, J Clin Apher, 26, 200, 10.1002/jca.20294 Rees, 2018, How I manage red cell transfusions in patients with sickle cell disease, Br J Haematol, 180, 607, 10.1111/bjh.15115 DeBaun, 2014, Controlled trial of transfusions for silent cerebral infarcts in sickle cell anemia, N Engl J Med, 371, 699, 10.1056/NEJMoa1401731 Biller, 2018, Red blood cell exchange in patients with sickle cell disease-indications and management: a review and consensus report by the therapeutic apheresis subsection of the AABB, Transfusion, 58, 1965, 10.1111/trf.14806 Lee, 2006, Stroke Prevention Trial in Sickle Cell Anemia (STOP): extended follow-up and final results, Blood, 108, 847, 10.1182/blood-2005-10-009506 Nouraie, 2015, Blood transfusion and 30-day readmission rate in adult patients hospitalized with sickle cell disease crisis, Transfusion, 55, 2331, 10.1111/trf.13155 Kalff, 2010, The impact of a regular erythrocytapheresis programme on the acute and chronic complications of sickle cell disease in adults, Br J Haematol, 149, 768, 10.1111/j.1365-2141.2010.08150.x Vichinsky, 1995, A comparison of conservative and aggressive transfusion regimens in the perioperative management of sickle cell disease. The preoperative transfusion in sickle cell disease study group, N Engl J Med, 333, 206, 10.1056/NEJM199507273330402 Alvarez, 2013, Pain and other non-neurological adverse events in children with sickle cell anemia and previous stroke who received hydroxyurea and phlebotomy or chronic transfusions and chelation: results from the SWiTCH clinical trial, Am J Hematol, 88, 932, 10.1002/ajh.23547 Ware, 2012, Stroke with transfusions changing to hydroxyurea (SWiTCH), Blood, 119, 3925, 10.1182/blood-2011-11-392340 Coates, 2019, Iron overload in transfusion-dependent patients, Hematology, 2019, 337, 10.1182/hematology.2019000036 Strobel, 2008, Hemolytic Transfusion Reactions, Transfus Med Hemother, 35, 346, 10.1159/000154811 Vekeman, 2016, Adherence to iron chelation therapy and associated healthcare resource utilization and costs in Medicaid patients with sickle cell disease and thalassemia, J Med Econ, 19, 292, 10.3111/13696998.2015.1117979 Coates, 2017, How we manage iron overload in sickle cell patients, Br J Haematol, 177, 703, 10.1111/bjh.14575 Chaudhary, 2013, Deferasirox: appraisal of safety and efficacy in long-term therapy, J Blood Med, 4, 101 Armstrong, 2013, Long-term persistency and costs associated with the use of iron chelation therapies in the treatment of Sickle cell disease within Medicaid programs, J Med Econ, 16, 10, 10.3111/13696998.2012.723081 Tsouana, 2015, Deferasirox for iron chelation in multitransfused children with sickle cell disease; long-term experience in the East London clinical haemoglobinopathy network, Eur J Haematol, 94, 336, 10.1111/ejh.12435 Jenny, 2017, Access to safe blood in low-income and middle-income countries: lessons from India, BMJ Glob Health, 2, 10.1136/bmjgh-2016-000167 Custer, 2018, Addressing gaps in international blood availability and transfusion safety in low- and middle-income countries: a NHLBI workshop, Transfusion, 58, 1307, 10.1111/trf.14598 WHO Guidelines Approved by the Guidelines Review Committee. Screening Donated Blood for Transfusion-Transmissible Infections: Recommendations. Geneva: World Health Organization; 2009. Adewoyin, 2017, Hydroxyurea therapy in adult Nigerian sickle cell disease: a monocentric survey on pattern of use, clinical effects and patient's compliance, Afr Health Sci, 17, 255, 10.4314/ahs.v17i1.31 Misra, 2017, A Phase Ib open label, randomized, safety study of SANGUINATE in patients with sickle cell anemia, Rev Bras Hematol Hemoter, 39, 20, 10.1016/j.bjhh.2016.08.004 Inusa, 2019, Geographic differences in phenotype and treatment of children with sickle cell anemia from the multinational DOVE study, J Clin Med, 8 Ofakunrin, 2021, Level of utilization and provider-related barriers to the use of hydroxyurea in the treatment of sickle cell disease patients in Jos, North-Central Nigeria, Afr Health Sci, 21, 765, 10.4314/ahs.v21i2.36 Ballas, 2009, The cost of health care for patients with sickle cell disease, Am J Hematol, 84, 320, 10.1002/ajh.21443 Wayne, 2000, Financial analysis of chronic transfusion for stroke prevention in sickle cell disease, Blood, 96, 2369, 10.1182/blood.V96.7.2369 Baldwin, 2022, Medical and non-medical costs of sickle cell disease and treatments from a US perspective: a systematic review and landscape analysis, Pharm Open, 6, 469 Hilliard, 2018, Red blood cell transfusion therapy for sickle cell patients with frequent painful events, Pedia Blood Cancer, 65, 10.1002/pbc.27423 Amarachukwu, 2022, Economic burden and catastrophic cost among people living with sickle cell disease, attending a tertiary health institution in south-east zone, Nigeria, PLoS One, 17, 10.1371/journal.pone.0272491 Alwaheed, 2022, Delayed hemolytic transfusion reaction in sickle cell disease: a case series, Am J Case Rep, 23 Balbuena-Merle, 2019, Red blood cell alloimmunization and delayed hemolytic transfusion reactions in patients with sickle cell disease, Transfus Clin Biol, 26, 112, 10.1016/j.tracli.2019.02.003 Tormey, 2019, Transfusion-related red blood cell alloantibodies: induction and consequences, Blood, 133, 1821, 10.1182/blood-2018-08-833962 Hendrickson, 2020, Red blood cell alloimmunization and sickle cell disease: a narrative review on antibody induction, Ann Blood, 5 Yazdanbakhsh, 2012, Red blood cell alloimmunization in sickle cell disease: pathophysiology, risk factors, and transfusion management, Blood, 120, 528, 10.1182/blood-2011-11-327361 Vichinsky, 1990, Alloimmunization in sickle cell anemia and transfusion of racially unmatched blood, N Engl J Med, 322, 1617, 10.1056/NEJM199006073222301 Rosse, 1990, Transfusion and alloimmunization in sickle cell disease. The cooperative study of sickle cell disease, Blood, 76, 1431, 10.1182/blood.V76.7.1431.1431 Murao, 2005, Risk factors for alloimmunization by patients with sickle cell disease, Braz J Med Biol Res, 38, 675, 10.1590/S0100-879X2005000500004 Wahl, 2012, Lower alloimmunization rates in pediatric sickle cell patients on chronic erythrocytapheresis compared to chronic simple transfusions, Transfusion, 52, 2671, 10.1111/j.1537-2995.2012.03659.x Coleman, 2019, Alloimmunization in patients with sickle cell disease and underrecognition of accompanying delayed hemolytic transfusion reactions, Transfusion, 59, 2282, 10.1111/trf.15328 Wayne, 1993, Transfusion management of sickle cell disease, Blood, 81, 1109, 10.1182/blood.V81.5.1109.bloodjournal8151109 Serjeant, 2016, Management of sickle cell disease: challenges and risks of transfusion, Int J Clin Transfus Med, 4, 109, 10.2147/IJCTM.S96918 Moreira Júnior, 1996, Red blood cell alloimmunization in sickle cell disease: the influence of racial and antigenic pattern differences between donors and recipients in Brazil, Am J Hematol, 52, 197, 10.1002/(SICI)1096-8652(199607)52:3<197::AID-AJH11>3.0.CO;2-D Olujohungbe, 2001, Red cell antibodies in patients with homozygous sickle cell disease: a comparison of patients in Jamaica and the United Kingdom, Br J Haematol, 113, 661, 10.1046/j.1365-2141.2001.02819.x Chou, 2013, High prevalence of red blood cell alloimmunization in sickle cell disease despite transfusion from Rh-matched minority donors, Blood, 122, 1062, 10.1182/blood-2013-03-490623 Pirenne, 2021, How to avoid the problem of erythrocyte alloimmunization in sickle cell disease, Hematol Am Soc Hematol Educ Program, 2021, 689, 10.1182/hematology.2021000306 Davis, 2017, Guidelines on red cell transfusion in sickle cell disease. Part I: principles and laboratory aspects, Br J Haematol, 176, 179, 10.1111/bjh.14346 Compernolle, 2018, Red blood cell specifications for patients with hemoglobinopathies: a systematic review and guideline, Transfusion, 58, 1555, 10.1111/trf.14611 Desai, 2015, Alloimmunization is associated with older age of transfused red blood cells in sickle cell disease, Am J Hematol, 90, 691, 10.1002/ajh.24051 Hod, 2010, Transfusion of red blood cells after prolonged storage produces harmful effects that are mediated by iron and inflammation, Blood, 115, 4284, 10.1182/blood-2009-10-245001 Donadee, 2011, Nitric oxide scavenging by red blood cell microparticles and cell-free hemoglobin as a mechanism for the red cell storage lesion, Circulation, 124, 465, 10.1161/CIRCULATIONAHA.110.008698 Xiong, 2011, Red blood cell microparticles show altered inflammatory chemokine binding and release ligand upon interaction with platelets, Transfusion, 51, 610, 10.1111/j.1537-2995.2010.02861.x Ackfeld, 2022, Blood transfusion reactions-a comprehensive review of the literature including a swiss perspective, J Clin Med, 11 Goel, 2019, Noninfectious transfusion-associated adverse events and their mitigation strategies, Blood, 133, 1831, 10.1182/blood-2018-10-833988 Rajesh, 2015, Effects of prestorage leukoreduction on the rate of febrile nonhemolytic transfusion reactions to red blood cells in a tertiary care hospital, Ann Med Health Sci Res, 5, 185, 10.4103/2141-9248.157498 Geiger, 2007, Acetaminophen and diphenhydramine premedication for allergic and febrile nonhemolytic transfusion reactions: good prophylaxis or bad practice?, Transfus Med Rev, 21, 1, 10.1016/j.tmrv.2006.09.001 Shah, 2012, Complications of implantable venous access devices in patients with sickle cell disease, Am J Hematol, 87, 224, 10.1002/ajh.22230 Otrock, 2018, Vascular access for red blood cell exchange, Transfusion, 58, 569, 10.1111/trf.14495 Salawu, 2022, Totally implantable venous access ports and associated complications in sub-Saharan Africa: a single-centre retrospective analysis, Ecancermedicalscience, 16, 1389, 10.3332/ecancer.2022.1389 Beverung, 2015, Health-related quality of life in children with sickle cell anemia: impact of blood transfusion therapy, Am J Hematol, 90, 139, 10.1002/ajh.23877 Maxwell, 2019, Health-related quality of life in children with sickle cell disease undergoing chronic red cell transfusion therapy, J Pedia Hematol Oncol, 41, 307, 10.1097/MPH.0000000000001376 Hawkins, 2020, Patient and family experience with chronic transfusion therapy for sickle cell disease: a qualitative study, BMC Pedia, 20, 172, 10.1186/s12887-020-02078-w Carden, 2019, Emerging disease-modifying therapies for sickle cell disease, Haematologica, 104, 1710, 10.3324/haematol.2018.207357 DʼAlessandro, 2020, Hypoxic storage of red blood cells improves metabolism and post-transfusion recovery, Transfusion, 60, 786, 10.1111/trf.15730 Yoshida, 2019, Red blood cell storage lesion: causes and potential clinical consequences, Blood Transfus, 17, 27