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Presented by Gretchen Vaughn, RN, MSN, CPNP Immunology / Bone Marrow Transplant Nurse Practitioner Cincinnati Children’s

                Reduced Intensity Conditioning Regimen for Bone Marrow Transplant in Children with Immune Deficiency: Managing Complications and Improving Outcomes                 . Presented by Gretchen Vaughn, RN, MSN, CPNP

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Presented by Gretchen Vaughn, RN, MSN, CPNP Immunology / Bone Marrow Transplant Nurse Practitioner Cincinnati Children’s

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  1.     Reduced Intensity Conditioning Regimen for Bone Marrow Transplant in Children with Immune Deficiency: Managing Complications and Improving Outcomes      Presented by Gretchen Vaughn, RN, MSN, CPNP Immunology / Bone Marrow Transplant Nurse Practitioner Cincinnati Children’s Hospital Medical Center Cincinnati, Ohio, USA

  2. Cincinnati Children’s Hospital Medical Center

  3. BMT for Immune Disorders 2006-2008 • SCID - 7 • NEMO – 4 • CGD - 8 • CID - 8 • WAS - 9 • HLH - 14 • XLP – 6 • Others (IPEX, LCH, ALPS) - 7

  4. Hemophagocytic Lymphohistiocytosis HLH: is a condition of abnormal cytotoxic functions which result in excessive and prolonged immune activation, usually fatal if untreated  There are many genetic causes of HLH Familial HLH: PRF1, Munc 13-4, STX-11 XLP: SH2D1A, BIRC4  HCT is the only cure for these disorders

  5. Background  Results of ‘conventional’ myeloablative chemotherapy pre-HCT for HLH are suboptimal  Patients with active diseaseat the time of HCT fare poorly  Early treatment related mortality (as defined by death within 100 days of transplant) is a major cause of treatment failure

  6. Background  CONVENTIONAL APPROACH (Bu/Cy/+ VP-16)  HLH94, n=108 * - 70% DFS at 5 years with Matched Sibling and Matched Unrelated Donor - 50% DFS at 5 years with Mismatched Unrelated Donor - Most fatalities occurred within the first 100 days  CIBMTR Analysis, n=53** - 35% rate of mortality by day 100 *Horne et al, BJM, 2006 **Baker et al, ASH, 2006

  7. Background For The Use of Reduced Intensity Conditioning  Pilot data for use of RIC (Campath/Flu/Mel) Great Ormond Street Hosp., UK -12 pts, mixed diagnoses; 8 in clinical remission - 9 survive – 3 are mixed chimeras * Cooper et al, Blood, 2006

  8. Treatment  Campath 1-H*: subQ or IV on 4 consecutive days “proximal” - doses revised October, 07; -timing changed May, 2008 to start day -22 “distal” as an option  Fludarabine*: 30 mg/m2 IV on 5 consecutive days, -8 to -4  Melphalan*: 140 mg/m2 IV once, on day -3  GvHD Prophylaxis: CsA/FK506 and steroids 1mg/kg/day *Doses adjusted per kg if patient < 10 kg

  9. Patient Characteristics – General

  10. Patient Characteristics – General 2

  11. Post HCT Patient Outcomes

  12. Post HCT Patient Outcomes 2

  13. Post HCT Patient Outcomes

  14. Post HCT Patient Outcomes 2

  15. Outcomes Summary - CCHMC Experience Engraftment: total range 21-100% donor  10/14 HLH patients with failure to maintain engraftment received donor lymphocyte infusions (DLI) 1/6 XLP patients received DLI

  16. Outcomes Summary CCHMC Experience  GvH skin - - 1/20 grade 3 without DLI - 3/20 grade 2-3 post DLI  GI GvH - 2/20 with grade 3 post DLI

  17. Outcomes Summary CCHMC Experience  Minimal organ toxicity for all Survival – 19/20 alive 4-30 months post transplant Immune reconstitution – 1 year post: - most patients have normal T cell numbers and function (mitogens) - most patients remain on IVIG replacement with good progress toward B cell reconstitution

  18. Future Implications  Long term monitoring of donor versus recipient lymphocyte populations is needed as well as determination of “functional engraftment” How much engraftment is enough?

  19. Acknowledgements and Appreciation  Alexandra Filipovich, MD Jack Bleesing, MD  Michael Jordan, MD Rebecca Marsh, MD  Nursing Colleagues, Data Managers, and Secretarial Support

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