Pilot Study LTFU

							Transplantation For Scleroderma:
   Data from the Phase I/II Pilot
        Multicenter Study

     (FHCRC protocol 1019)
                      TRANSPLANT REGIMEN*

                                      CD34+
   TBI 2Gy      CTX                   AUTO                       G-CSF
  x 4‡ = 8 Gy 60mg/kgx2               PBSC


DAY
      -5       -4     -3      -2     -1      0      1              3
      5

                           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 SCT

             GEE; p<0.0001
              Mean Value

                                                   Mean
                                       Mean,
                             Skin                Change,
                                      Baseline
                                                   Final
                                       (n=34)
                                                  (n=25)

                                              -22.08,
                             mRSS
                                        30.12 p<.0001
                             (0-51)



                                      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)
Pulmonary Function after SCT for SSc




      GEE, p=0.002                        GEE, p=0.40
      Mean Change +2.11, p= .50           Mean Change -7.07, p=.02




 Forced Vital Capacity            DLCO (corrected for Hgb)
Autologous Hematopoietic Cell Transplantation: Survival
                       (n=34)

                                         Progression   (from SCOT):
                5 yr overall survival=
                                         1. Respiratory failure-
                64%                      decrease of DLCO >30% or
                5 yr PFS= 64%            FVC >20%.
                                         2. Renal failure- dialysis or
                                         kidney transplantation.
                                         3. Clinical heart failure or
                                         LVEF <30%.
Overall Survival: SCOT-Comparable Patients and Treatment
                         (n=22)


                                n=23 Excluded:
                                     No lung shielding    -8
                                     Rabbit ATG           -1
                                     Eject F. (47%)      -1
        3 year OS= 87%               FVC (27%)            -1
                                     Renal failure/PHT    -1
               3 year OS= 91%
               5 year OS= 78%