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Presented by Gretchen Vaughn, RN, MSN, CPNP Immunology / Bone Marrow Transplant Nurse Practitioner - PowerPoint PPT Presentation


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                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

    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



Bmt for immune disorders 2006 2008
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


Hemophagocytic lymphohistiocytosis
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


Background
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


Background1
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


Background for the use of reduced intensity conditioning
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


Treatment
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








Outcomes summary cchmc experience
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


Outcomes summary cchmc experience1
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


Outcomes summary cchmc experience2
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


Future implications
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?


Acknowledgements and appreciation
Acknowledgements and Appreciation

 Alexandra Filipovich, MD

Jack Bleesing, MD

 Michael Jordan, MD

Rebecca Marsh, MD

 Nursing Colleagues, Data Managers,

and Secretarial Support