SICKLE CELL DISEASE TO TREAT OR

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1 SICKLE CELL DISEASE TO TREAT OR NOT TO TREAT COHEM Barcelona September 8, 2012 Sujit Sheth, M.D. Pediatric Hematology Oncology

2 Disclosures None

3 Outline Morbidity and mortality Definitive therapies Risk benefit analysis

4 Scope of Issue Worldwide >275,000 births/yr 1 1% of all births in Africa, ~6M affected 1 Wide phenotypic variability Decreased life expectancy Significant morbidity, poor QOL Enormous cost treatment, QALYs 3 1. Modell B et al. Bull WHO 2008 Jun;86(6): Grosse SD et al. Am J Prev Med 2011;41(6S4):S398 S Braithwaite et al., Medical Care 2008;46:

5 Survival (US) Genotype Sex Median Survival (yrs) SS M SS F SC M 60 SC F 68 Platt et al, NEJM 1994

6 Age at Death (US) In Mean age at death was 39yrs, 35% alive at age >45 yrs Am J Prev Med 2010;38(4S):S512 S521

7 Data sparse Mortality non-us 3.4% under-5 mortality, 6.4% in Africa 1 Excess childhood mortality in Africa ~ 50-90% (HbSS) 2 -? most undiagnosed Infection, splenic sequestration, stroke (Poor data to support malaria as cause) 1. Modell B et al. Bull WHO 2008 Jun;86(6): Grosse SD et al. Am J Prev Med 2011;41(6S4):S398 S405

8 Morbidity Childhood Infarctive Stroke Growth Pain crisis Acute chest syndrome Splenic sequestration Infectious risk Avascular necrosis Progressive organ dysfunction Adulthood Hemorrhagic stroke Pulmonary hypertension Avascular necrosis Sickle hepatopathy Sexual dysfunction/infertility Eye Leg ulcers Organ failure

9 Quality of Life Episodic / chronic pain Pulmonary disease CNS complications Repeated hospitalizations Organ dysfunction Reduced life expectancy Socioeconomic issues cost, productivity

10 Health Related QOL Review 39 studies, only 5 prospective Generic HRQL instrument not specific Adults and children with SCD have significantly impaired HRQL that is comparable to or worse than other chronic diseases.even in their baseline state of health. Panepinto JA and Bonner M. Pediatr Blood Cancer 2012; 59:

11 Effects on the Brain Prevalence of silent infarcts 1 27% by age 6yrs, 37% by 14 yrs Overt Stroke infarctive, hemorrhagic 2-5% by 6yrs preventable in children 2 Loss of global intellectual function 3 Worse if overt stroke Not preventable by supportive therapies 1. DeBaun MR et al, Blood 2012; 119(20): Adams RJ et al. N Engl J Med.1998;339(1): Wang W et al, J Pediatr 2001; 139(3):

12 Pulmonary disease Acute Chest Syndrome High mortality (>adults), recurrent Lung disease (obstructive and restrictive) Chronic hypoxia Pulmonary hypertension 1 20% of adults Mostly lethal within 3 yrs No prevention or effective treatment 1. Gladwin MT. N Engl J Med 2004; 350:

13 Supportive Therapy Infection prophylaxis Pain management Transfusion when indicated Treatment of complications Will not prevent ongoing disease effect on organs and tissues

14 Success of Supportive Therapy Hassell KL. Am J Prev Med 2010;38(4S):S512 S521

15 Definitive Therapy Regular transfusions / stroke prevention Hydroxyurea Surgery Hip coring EDAS (encephaloduroarteriosynangiosis) Stem cell transplantation Gene therapy

16 Transfusion therapy Benefit for overt stroke Unclear for silent infarct Complications alloimmunization, iron overload Fails to prevent vascular disease if overt stroke or Moya-Moya 1 If discontinued, stroke risk goes back up 2 High cost - $40,000/yr in Bishop S et al. Blood Cells Mol Dis. 2011;47(2): Abboud MR et al. Blood 2011; 118(4): Wayne AS et al. Blood 2000; 96(7):

17 Hydroxyurea Reduction in severity and frequency of complications improves QOL 1,2 Safe to start early 1 Acceptable long-term safety profile 3 Reduced mortality 4? Beneficial effect on organ function? Role in stroke, PHT 1. Wang AC et al. Lancet 2011; 377: Thornburg CD et al. J Pediatr Hematol Oncol 2011; 33(4): Steinberg MH et al. Am J Hematol : Voskaridou E et al. Blood 2010; 115(12):

18 Stem Cell Transplantation Only cure, prevents disease progression Excellent results when matched sibling donor Short term mortality risk, longer term morbidity risk steadily improving Prevention of organ damage if done early, no reversal if done late Advances Non-ablative / reduced intensity preparation Alternative donor MUD, UCB, haplo-identical Overall, long term cost benefit

19 SCT - survival Study Vermylen 1998 Walters 2000 Locatelli 2003 Panepinto 2007 Bernaudi n 2007 Lucarelli 2011 Hsieh * Bhatia 2012* # n Overall Survival Disease Free Survival Rejection Lucarelli G et al. Cold Spring Harb Perspect Med Hsieh MM et al. NEJM 2009; 361(24): * Matched sibling donors only # Unpublished data, , children only

20 Emerging therapies Approach Examples Rees DC et al. Lancet 2010; 376:

21 Cost In US 113,000 admissions/yr 1 $488 million costs (hospitalization only) 1 As life expectancy increases More admissions Higher costs of treating organ failure Loss of productivity Adults - 30% on disability, 50% unemployed 2 1. Steiner C, Miller J. HCUP Statistical Brief #21. December Ballas SK et al. J Natl Med Assoc 2010; 102: 993

22 Quality-Adjusted Life Years (QALYs) 5 yr old with abnormal TCD lifelong transfusion and chelation adds 16 QALYs? HU? Stem cell transplant? QALYs from supportive care 1 QALY = $203,000 ($109,000-$297,000) Braithwaite et al., Medical Care 2008;46:

23 Reasons TO TREAT High morbidity and mortality limited effect of supportive therapy Poor QOL, particularly related to CNS Reduced life expectancy Cost to family and society Good rates of success with HU Improved success rates of curative therapy stem cell transplantation

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