Transplantation For Scleroderma: Data from the Phase I/II Pilot
Multicenter Study
(FHCRC protocol 1019)
Transplantation For Scleroderma: Data from the Phase I/II Pilot
Multicenter Study
(FHCRC protocol 1019)
TRANSPLANT REGIMEN*TRANSPLANT REGIMEN*
TBI 2Gy x 4‡ = 8 Gy
CTX60mg/kgx2
- 5 - 4 - 3 -2 -1 0 1 3 5
CD34+AUTOPBSC
DAY
G-CSF
Equine ATG 15 mg/kg/dose§
* 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
Patient Characteristics
N=33 Median Range
Female (N =26)
Age (yrs) 41 23-61
Primary Organ involvement
lung=33; kidney=1; heart=8; GI=22
Skin score (mRSS): 30 3-48
DLCO adjusted for Hgb (mm/Hg) 62 40-95
FVC % 72 27-103
Serum Creatinine (mg/dL) 0.7 0.4-1.4
Disease Duration (months) 20 4-51
Decrease in Rodnan Skin Score after SCTDecrease in Rodnan Skin Score after SCT
GEE; p<0.0001
Skin
Mean, Baseline (n=34)
Mean Change,
Final (n=25)
mRSS (0-51)
30.12 -22.08, p<.0001
Mean Value
Nash, Blood 2007
Resolution of Dermal Fibrosis Patient 11 Skin Biopsy
A+B : Pre-HCT: grade 5; low + high power
C+D : 1 year post-HCT: grade 3; low + high power
E+F: 5 years post-HCT: grade 0; low + high power
Nash, Blood 2007
HEALTH ASSESSMENT (mHAQ)HEALTH ASSESSMENT (mHAQ)
Pulmonary Function after SCT for SScPulmonary Function after SCT for SSc
Forced Vital Capacity DLCO (corrected for Hgb)
GEE, p=0.002Mean Change +2.11, p= .50
GEE, p=0.40Mean Change -7.07, p=.02
Autologous Hematopoietic Cell Transplantation: Survival (n=34)
Autologous Hematopoietic Cell Transplantation: Survival (n=34)
Progression (from SCOT):1. Respiratory failure-decrease of DLCO >30% or FVC >20%.2. Renal failure- dialysis or kidney transplantation.3. Clinical heart failure or LVEF <30%.
Progression (from SCOT):1. Respiratory failure-decrease of DLCO >30% or FVC >20%.2. Renal failure- dialysis or kidney transplantation.3. Clinical heart failure or LVEF <30%.
5 yr overall survival= 64%5 yr PFS= 64%
Overall Survival: SCOT-Comparable Patients and Treatment (n=22)
Overall Survival: SCOT-Comparable Patients and Treatment (n=22)
n=23 Excluded:No lung shielding -8Rabbit ATG -1Eject F. (47%) -1FVC (27%) -1Renal failure/PHT -1
3 year OS= 87%
3 year OS= 91%5 year OS= 78%