SCD Young Adult Transition

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1 SCD Young Adult Transition Julie Kanter, MD Director, Sickle Cell Disease Research Assistant Professor, Pediatric Heme-Onc Medical University of South Carolina

2 Disclosure MUSC receives funding from multiple pharmaceutical companies for enrollment of patients on sickle cell disease related clinical trials I have served on the advisory committee for: MAST therapeutics Apopharma Bluebird Bio I have served as an expert in SCD for: Eli Lilly Pharmaceuticals Steering Committee Drug therapeutics in SCD NONE OF THAT AFFECTS MY PRESENTATION TODAY

for: MAST therapeutics Apopharma Bluebird Bio I have served as an expert in SCD for: Eli Lilly

3 It is not a transition-but a metamorphosis

4 GOOD NEWS: patients are living into adulthood

5 PREVENTION=IMPROVEMENTS Sickle cell-related death among AA children under 4 years of age fell by 42% during 1999 to Relative to , the mortality rate for patients with SCD decreased by: 68% at age 0 to 3 years 39% at age 4 to 9 years 24% at age 10 to 14 years Most children with SCD live to >18 years of age. Many still die before they are 40 years of age. Life expectancy has increased from mean of 14 yrs

Relative to 1983-1986, the mortality rate for patients with SCD 1999-2002 decreased by: 68% at age 0 to 3

6 What has happened to improve outcomes in childhood? Newborn Screening Parent education Provider education Comprehensive Care Pediatric and Specialty Care Research Enhanced Disease Understanding New preventative tools

Comprehensive Care Pediatric and Specialty Care

7 Key Research Studies Research Studies in Review STOP STUDY: Transfusion greatly reduces the risk of a first stroke in children with sickle cell anemia who have abnormal results on transcranial Doppler ultrasonography. (N Engl J Med 1998;339:5-11.) SIT STUDY: Silent infarcts in patients with sickle cell disease ASTHMA: Patients with SCD AND asthma have an increased incidence of vaso-occlusive pain MSH: Multicenter Study of Hydroxyurea HUG and BabyHUG studies of Hydrxoyurea

8 Stroke in SCD Clinically evident stroke in 8-10% Incidence 220x higher than stroke in children without SCD Silent infarction in 20-35% SI is defined as a lesion on MRI c/w infarction w/o focal neurologic deficit lasting longer than 24 hrs. NOT Silent associated with: Significant cognitive impairment Decrement in intellectual abilities Poor academic attainment Increased risk of new or progressive SI Increased risk of overt stroke DeBaun et al, 1998, Armstrong et al., 1996, Schatz et al, 2001, Pegelow et al, 1999, Miller et al, 2000

NOT Silent associated with: Significant cognitive impairment Decrement in intellectual abilities Poor academic attainment Increased

9 SCD: What happens as patients grow up?

10 PEDIATRICS Under the Sea Golden years Episodic acute pain Opioids acceptable (expected) Goal - pain free Fewer social problems Fewer emotional problems Goal to avoid mortality

11 Above ground is A whole new world ADULT Turbulent years Chronic unremitting pain Opioids BAD (stigmatized) Goal - adjust to pain Progressive social problems Chronic organ dysfunction Goal is symbiosis with disease

12 SCD Complications Pediatric Adult Splenic infarct and/or sequestration 1 Vascular occlusion 1 Primary and recurrent stroke 1 ACS 1 Osteomyelitis 1 Hyperhemolysis 1 Severe recurrent pain 2 Pulmonary hypertension 1 12 Osteonecrosis 1 Papillary necrosis 1 Renal failure 1 Chronic heart failure 2 Complicated pregnancy 3 Vitamin D Deficiency Mental Health Pulm Hypertension Osteoporosis Iron overload

Pulmonary hypertension 1 12 Osteonecrosis 1 Papillary necrosis 1 Renal failure 1 Chronic heart

13 What is transition? Transition: a. passage from one state, stage, subject, or place to another : change b : a movement, development, or evolution from one form, stage, or style to another Medical Transition: a. the process of acquiring medical maturity b. equip persons with chronic diseases with knowledge and self-care skills necessary to function independently.

development, or evolution from one form, stage, or style to another Medical Transition: a.

14 A Tale of Two Environments PEDIATRIC Nurturing/protective Attractive environment Parent centered Family insurance Paternalistic Centralized Available providers ADULT Informing Patient centered Underfunded Employment based insurance Total patient autonomy Fragmented Few providers

Centralized Available providers ADULT Informing Patient centered

15 Education Education of Patients Education of Parents Education of PROVIDERS Education of HOSPITALS Education of the healthcare SYSTEM

16 Research Advocacy Community Alliance Determination Persistence

17 CASE DISCUSSION

18 Case #1: Quella 25 year old woman, history of abnormal TCD at 11 years of age Started on transfusion therapy (simple transfusions) and followed by pediatrics until 18 years and transferred to adult provider in the same hospital Adult provider continued transfusion therapy, chelation with Desferal Works (night hours), lives with her sister (who also has SCD and history of stroke) Occasional pain, ferritin is >10,000

provider in the same hospital Adult provider continued transfusion therapy, chelation with Desferal

19 Case #2 Amy-Lee 18 year old female HbSS disease, history of acute chest, no strokes College-bound, on Hydroxyurea, intermittent VOC Meets a boy, moves, and decides to manage her disease independently Multiple hospitalizations Transfused in local hospital at each visit Returns to pediatric clinic 22 years old Chronic Pain Did not complete college On disability

her disease independently Multiple hospitalizations Transfused in local hospital at each

20 Case #3 Don 27 yo male, history of stroke x 2 Followed by pediatric hematology-on transfusion until 18 years of age No provider-jail at 19 years of age (no treatment-stroke #2) Presented with chronic pain, mild hemiparesis, neurocognitive delay ER 3x/week, multiple medications

19 years of age (no treatment-stroke #2) Presented with chronic pain,

21 Case #4 Loris 29 year old woman, from rural small town, SC Scattered pediatric care, doesn t know what type of sickle cell disease she has Has a PCP but doesn t want to give me pain meds ER 3-4x/week, hospitalized intermittently, gets shots AVN, repaired hip, chronic pain

22 Case #5 Jonny 25 year old male, HbSS disease Started on hydroxyurea at 18 years of age Prior to hydroxyurea, VOC 1x/month, with frequent hospitalizations After hydroxyurea-minimal pain Followed by pediatrics as a child and now sees an adult internist who continues HU at same dose

23 Case #6 Myrtle 41 year old female, HbSB+ We follow her 13 year son with hbss disease who is on monthly exchange transfusions Saw pediatric hematology as a child, now has a family practice physician who says I don t know how to help you Chronic back pain-on Cymbalta No relief Works in Cafeteria

24 More than just transition Transition is just the beginning Transition of the patient Communicate with the provider Educate the system Patient-focused care Physicians + Community = Effective

South Carolina Sickle Cell Network Beaufort Pediatric Sickle Cell USC Pediatric Sickle Cell Georgetown PCMH Beaufort PCMH Columbia PCMH Upstate PCMH MUSC

25 South Carolina Sickle Cell Network Beaufort Pediatric Sickle Cell USC Pediatric Sickle Cell Georgetown PCMH Beaufort PCMH Columbia PCMH Upstate PCMH MUSC Comprehensive Sickle Center MUSC (UIM) PCMH James R. Clark Case management State SCD program Greenville Pediatric Sickle Cell Florence PCMH Sumpter Adult Heme- Onc

Therapeutic Treatment Options: Chronic Blood Transfusions Bone Marrow Transplantation. Marianne E. McPherson Yee, MD, MSc

Therapeutic Treatment Options: Chronic Blood Transfusions Bone Marrow Transplantation Marianne E. McPherson Yee, MD, MSc Sickle Cell Treatment Options Supportive Care Newborn Screen PCN Immunizations Education