1. Sickling smoothly through life… Educating patients about transition Nirmish Shah, MD Assistant Professor Director of Sickle Cell Transition Program Duke University

2. Outline  Introduction  Challenges facing transitioning SCD patients  Model and key components for a successful transition to adulthood  ‘Who, What, Where’ – levels of patient understanding 2

3. Who  Medicine-Pediatrics residency at ECU – 2000-2004 – Goal was to do med/peds hem/onc…. ….then reality kicked in and hematology seemed more realistic and interesting

4. What  Med/Peds Hematologist – Joint appointment between medicine and pediatrics – Have outpatient clinics in both medicine and pediatrics – Do inpatient consults for adult hematology – Majority of effort in research

5. Where  ‘Where do I go’… – Goal to perform combined pediatric and adult research – Improve transition guidelines and protocols – Advocate co-management of SCD with primary care providers

6. Introduction  Sickle Cell Disease (SCD) is the most common inherited disease worldwide  Recent advances include prophylactic PCN, improved immunizations, and increased use of hydroxyurea

7. Survival Deaths due to sickle cell anemia or sickle cell trait in 1967 Scott, R. Health Care Priority and Sickle Cell Anemia, JAMA, 1970, 214(4)

8. Recent Developments Have Greatly Improved Patient Management and Survival 1 8 1. Quinn CT, Rogers ZR, McCavit TL, Buchanan GR. Blood. 2010;115(17):3447–3452. 2. National Heart, Lung, and Blood Institute. Progress in Heart, Lung, and Blood Research: Reducing the burden of Sickle Cell Disease. August, 2011. https://www.nhlbi.nih.gov/news/spotlight/success/sicklecell.pdf. Accessed April 21, 2014. 3. Elmariah H, Garrett ME, De Castro LM, et al. Am J Hematol. 2014;89(5):530-535. 4. Zhou Z, Behymer M, Guchhait P. Anemia 2011;2011:918916. 5. Lusher JM, Haghighat H, Khalifa AS. Am J Hematol. 1976;1(2):265-273. 6. National Heart, Lung, and Blood Institute. The Management of Sickle Cell Disease. 4th ed. Bethesda, MD: National Institutes of Health; 2002. NIH publication 02-2117. http://www.nhlbi.nih.gov/health/prof/blood/sickle/sc_mngt.pdf. Accessed April 9, 2014. 7. Wang WC, Ware RE, Miller ST, et al. Lancet. 2011;377(9778):1663–1672. 8. Ware RE, Helms RW. Blood. 2012;119(17):3925-3932. BABY HUG, Pediatric Hydroxyurea Phase III Clinical Trial; NIH, National Institutes of Health; STOP, Stroke Prevention Trial in Sickle Cell Anemia. 60 50 40 30 20 10 Life expectancy for patients with SCA (years) 2,3 19101950197019801990200020101960192019301940 1910 - Discovery of SCD 4 1986 – Antibiotic prophylaxis in infants 6 1949 – Identification of HbS 4 1976 – RBC transfusion for treatment of secondary stroke 5 1987 – NIH recommends newborn screening 6 1998 – STOP trial 6 2011 – BABY HUG trial 7 2012 – SWiTCH trial 8 1995 – MSH Trial 6

9. CHALLENGES FACING TRANSITIONING SCD PATIENTS

10. Transitioning Poses Many Challenges for Patients 10 1. Treadwell M, Telfair J, Gibson RW, et al. Am J Hematol. 2011;86(1):116-120. 2. Paulukonis S, Neumayr L, Coates TD, et al. Blood. 2010;116:Abstract 254. 3. McDonnell WM, Kocolas I, Roosevelt GE, Yetman AT. Arch Pediatr Adolesc Med. 2010;164(6):572-576. 4. National Heart, Lung, and Blood Institute. The Management of Sickle Cell Disease. 4th ed. Bethesda, MD: National Institutes of Health; 2002. NIH publication 02-2117. http://www.nhlbi.nih.gov/health/prof/blood/sickle/sc_mngt.pdf. Accessed August 23, 2013. Challenges of Particular Concern for SCD Patients 1-4 Increased socioeconomic challenges Large number of older SCD patients, placing strain on the system Adult "transition patients" ending up in the pediatric emergency room Lack of a medical home with an adult hematologist –Result: suboptimal care with poor tracking of medication and transfusion histories

11. Ineffective use of healthcare system Hemker BG, Brousseau DC, Yan K, Hoffmann RG, Panepinto JA. When children with sickle-cell disease becomes adults: lack of outpatient care leads to increased use of the emergency department. Am J Hematol. 2011 Oct;86(10):863-5. doi: 10.1002/ajh.22106. Epub 2011 Aug 3 During 5 year study: Children = <18 Transition = turned 19 Young Adults = 19-30 Adults = >30

12. Readmission Rates Also Spike After Transition 12 Brousseau DC, Owens PL, Mosso AL, et al. JAMA. 2010;303(13):1288-1294.  Approximately 80% of rehospitalizations were to the same hospital –94% of pediatric readmissions –77% of adult readmissions

13. The Transition Years Are A Vulnerable Time in Life For Many SCD Patients  Deaths in patients 18 years of age or older in the 940-patient Dallas newborn cohort –There were 7 deaths between 2002 and 2007, all of which occurred in transition-age patients –Mean time between transition and death was 1.8 years 13 Quinn CT, Rogers ZR, McCavit TL, Buchanan GR. Blood. 2010;115(17):3447–3452. N/A, not applicable. *Transition was delayed beyond 18 years due to chronic renal failure.

14. Complications of SCD Increase As Patients Transition and RBC Transfusions Decrease 14 Blinder MA, Vekeman F, Sasane M, et al. Pediatr Blood Cancer. 2013;60(5):828–835. Any Complications Mean Transfusions Pain Infection 5 4 3 2 1 0 Mean Number with > 1 Diagnosis Per Patient Per Quarter 0 50 45 40 35 30 25 20 15 10 5 N=(555)(600)(662)(656)(505)(339)(274)(239)(172)(90) Age 0.6 0.4 0.3 0.2 0.1 0 0.5 Mean Number of Transfusions Events and Prescriptions Per Patient Per Quarter Increased frequency of complications correlates with a decrease in the utilization of RBC transfusions and hydroxyurea

15. Risk factors for unsuccessful transition  Associated with successful transition (1 adult visit): –Transferring to adult care <21 (p=0.008) –Distance <20 miles (p=0.01) –Severe phenotype - SS/Sβ 0 (p=0.04) –Being on chronic transfusions (p=0.02) 32% did not transfer successfully Andemariam et al. Identification of Risk Factors for an Unsuccessful Transition from Pediatric to Adult Sickle Cell Disease Care. Pediatr Blood Cancer 2014;61:697–701

16. Duke Experience  A retrospective database query was conducted among all Duke patients aged 18-23 years with a SCD outpatient encounters through Dec 2013 –Transfer was defined as having clinic visit in pediatrics followed by a subsequent visit in the adult clinic. –Successful transfer was defined as having follow up care in adult hematology clinic for 1 year post- transfer. Hill, S; Maslow, G; Walker, L; Johnson, S; Shah, N. Growing Pains – Determination of Transfer and Transition from Pediatrics to Adult Outpatient Clinics for Patients with Sickle Cell Disease (SCD). ASH annual meeting 2014.

17. Duke Experience Database results Hill, S; Maslow, G; Walker, L; Johnson, S; Shah, N. Growing Pains – Determination of Transfer and Transition from Pediatrics to Adult Outpatient Clinics for Patients with Sickle Cell Disease (SCD). ASH annual meeting 2014.

18. Duke Experience Validation  Chart review of these patients –26 (8%) had missing data on outpatient SCD encounters –48 (14%) SCD patients not followed by Duke Hematology –65 (19%) did not have a SCD diagnosis. _______________________________________ Total: –139 (41%) records were excluded –203 patients followed at Duke

19. Duke Experience  Classification: –Continued care in pediatrics –First visit in adults –Transferred to a non-Duke provider –Documented transfer to Duke adult provider (with continued visits for 1 year)  “Lost to follow-up” status as greater than 6 months overdue

20. Duke Experience  5 (2%) are deceased (all <10 yo) Continued care in pediatrics - Less than 6 months overdue for f/u 28 (14%) First visit in adults14 (6%) Transferred to a non-Duke provider19 (9%) Transferred to adult hematology60 (30%) Lost to follow up78 (38%)

21. Duke QI  Lost to follow up –Communicate with patient/family –Document status –PCP –Specialty provider –Barriers to care –Insurance

22. An Educational Model to Promote Successful Transition to Adulthood

23. Blum RW, Garell D, Hodgman CH, et al. J Adolesc Health. 1993;14(7):570-576. A Definition of Transition  “Transition is a multifaceted, active process that attends to the medical, psychosocial, and educational/vocational needs of adolescents as they move from the child-focused to the adult- focused health-care system. Health transition facilitates transition in other areas as well (e.g., work, community, and the school).”  “Transition proceeds at different rates for different individuals and families (and programs)” 23

24. Components of a Transition Program  Comprehensive Approach –Must be a pre-defined program  Policies –Age = 18yo and graduation from high school –20yo if delayed graduation –1-2 years in peds if attending college –Education = must complete level 1 and 2 education in pediatrics –mastery >80% on knowledge questionnaires 24 Schwartz M, Tuchman LK, Hobbie WL, Ginsberg JP. Child Care Health Dev. 2011;37(6):883-895.

25. Components of a Transition Program  Components: –Medical Care – Adolescent/young adult Health Focus –Support - Case Management, Social, Psychological, volunteers/educators –Education - Patient, Family, Providers 25 Schwartz M, Tuchman LK, Hobbie WL, Ginsberg JP. Child Care Health Dev. 2011;37(6):883-895.

26. Levels of understanding  Level I (WHO) – Know who you are  Level II (WHAT) – Know what to do in different situations  Level III (WHERE) – Know where you are going Level I 13-14 year old Level II 15-16 year old Level III 17-18 year old

27. How much do you know about your Sickle Cell?

28. Planning for the Future: Levels of Patient Understanding 28 Level I (WHO) Know who they are Level II (WHAT) Know what to do in different Level II (WHERE) Know where they are going Level I 13 to 14 years old Level II 15 to 16 years old Level III 17 to 18 years old

29. Level I (WHO) Know who they are WHO Level I of Understanding 29 Level I 13 to 14 years old “I am _____, and I have sickle cell type ____.”

30. Level I Understanding Patients Aged 13 to 14 Years Basic points to understand  Shape of a sickle cell  Genetics  Types of sickle cell  How sickle cell causes complications  ‘Stressors’ that cause sickling 30 Level I 13 to 14 years old Level II 15 to 16 years old Level III 17 to 18 years old

31. What causes more ‘Sickling’?  Any stress on your body – Dehydration – Infection – Not breathing well – Temperature changes

32. Body happy = Blood happy!

33. WHAT Level II of Understanding 33 Level II (WHAT) Know what to do in different Level II 15 to 16 years old “I am _____, and I have sickle cell type ____. Sickle cell is when your blood looks like a banana and can cause blood to have problems going places. Stress to the body can cause issues. I know what I need to do when ____ happens with sickle cell.”

34. Level II Understanding Patients Aged 15 to 16 Years Basic points to understand  Types of blood cells  Management of pain  Medications taken daily  Warning signs for complications –Fever, shortness of breath, neurologic symptoms  Complications –Acute chest syndrome, stroke, infection, priapism  Sports and hydration 34 Level I 13 to 14 years old Level II 15 to 16 years old Level III 17 to 18 years old

35. Patients Should Understand Implications of Their Lab Results 35 Hemoglobin  Carries Oxygen  ‘Energy Cells’ White Blood Cells Transfusions, SF, LIC Reticulocyte Count Platelets MCV  How big  Reflects fetal hemoglobin  HU effect  Reflect risk of chronic iron overload  How much blood is made  Stop bleeding  Fight infection MCV, mean corpuscular volume. Level I 13 to 14 years old Level II 15 to 16 years old Level III 17 to 18 years old

36. What Patients Need to Know About: 36 Level I 13 to 14 years old Level II 15 to 16 years old Level III 17 to 18 years old

37. Level II (WHERE) Know where they are going WHERE LEVEL III of Understanding 37 Level III 17 to 18 years old “I am _____, and I have sickle cell type ____. Sickle cell is when your blood looks like a banana and can cause blood to have problems going places. Stress to the body causes issues. I know what I need to do when ____ happens with sickle cell. I am going to ___ college/ university and/or I am going to work ____. I know about what is bad for me and who/what are bad influences.”

38. Level III understanding Patients Aged 17 to 18 Years Basic points to understand  Health Maintenance  School and/or work (limits and resources)  Insurance  Adult provider –Coordination of transfer –Inpatient management differences –Transfusions/exchange transfusion 38 Level I 13 to 14 years old Level II 15 to 16 years old Level III 17 to 18 years old

39. Important Screenings for Health Maintenance 39 Routine dental exams Dilated eye exam Urine microalbumin Vaccinations Level I 13 to 14 years old Level II 15 to 16 years old Level III 17 to 18 years old

40. What Patients Need to Know About: 40 Level I 13 to 14 years old Level II 15 to 16 years old Level III 17 to 18 years old

41. Make Sure Patients Understand Their Insurance Options  Patients need to understand that seeking treatment without insurance will result in large medical bills –Leading cause for young adults to end up bankrupt is large, unexpected medical bills  Patients should talk to a social worker or regional public health sickle cell educators about resources and options. 41 Level I 13 to 14 years old Level II 15 to 16 years old Level III 17 to 18 years old

42.  Inpatient care  Medications  Transfusions/exchange transfusion decisions and tracking  Screening and monitoring  Resources Coordination of Care With the Adult Provider is Crucial  Agree upon the transfer process and time necessary for transition should be agreed upon by both  Outline the coordination of care before and after 42 Adult provider (hematologist) Pediatric provider Level I 13 to 14 years old Level II 15 to 16 years old Level III 17 to 18 years old

43. Know your Sickle Cell 43 Level I 13 to 14 years old Level II 15 to 16 years old Level III 17 to 18 years old I am ____ and I have sickle cell type ____. WHO:WHAT: I know what to do when ___ happens. WHERE: I know where I am going, to ___ college/ university and/or working at ___

44. Evidence of knowledge  Quizzes –Level 1 - Who, median 9 (range 7-10) –Level 2 - What, median 10 (range 8-10) –Level 3 - Where, median 8 (range 4-10)  Working on ‘All star’ Quizzes

45. Questions??? Transition to adulthood