Transplantation For Scleroderma: Data from the Phase I/II Pilot Multicenter Study (FHCRC protocol 1019)

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Transplantation For Scleroderma Data from the
Phase I/II Pilot Multicenter Study (FHCRC
protocol 1019)
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TRANSPLANT REGIMEN
CD34 AUTO PBSC
CTX 60mg/kgx2
TBI 2Gy x 4 8 Gy
G-CSF
DAY
- 5 - 4 - 3 -2 -1 0
1 3 5

• PBSC mobilized with G-CSF at 16 ug/kg/d
• TBI administered with lung shielding (lung
  dose 200 cGy) for last 26 pts
• Prednisone with Equine ATG then 0.5 mg/kg/day
  day 6-30, taper to day 60

McSweeney, Blood, 2002
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Patient Characteristics
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Decrease in Rodnan Skin Score after SCT
GEE plt0.0001
Mean Value
Nash, Blood 2007
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Resolution of Dermal Fibrosis Patient 11 Skin
Biopsy
AB Pre-HCT grade 5 low high power CD 1
year post-HCT grade 3 low high power EF 5
years post-HCT grade 0 low high power
Nash, Blood 2007
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HEALTH ASSESSMENT (mHAQ)
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Pulmonary Function after SCT for SSc
GEE, p0.002 Mean Change 2.11, p .50
GEE, p0.40 Mean Change -7.07, p.02
Forced Vital Capacity
DLCO (corrected for Hgb)
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Autologous Hematopoietic Cell Transplantation
Survival (n34)

• Progression (from SCOT)
• 1. Respiratory failure-decrease of DLCO gt30 or
  FVC gt20.
• 2. Renal failure- dialysis or kidney
  transplantation.
• 3. Clinical heart failure or LVEF lt30.

5 yr overall survival 64 5 yr PFS 64
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Overall Survival SCOT-Comparable Patients and
Treatment (n22)
Excluded No lung shielding -8 Rabbit ATG
-1 Eject F. (47) -1 FVC (27)
-1 Renal failure/PHT -1
n23
3 year OS 87
3 year OS 91 5 year OS 78