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Table 2 Overview of the literature on immunomodulation in classic infantile Pompe patients after antibodies have formed

From: Effects of immunomodulation in classic infantile Pompe patients with high antibody titers

Study Pt CRIM Age at start of ERT Age at start of IM IM protocol IM duration Current ERT dosea Follow-up since start of IM Alive Vent. free Walks at study end Titer at start of IM Number of RTX infusions B-cell recovery Last known titer (time since B-cell recovery)
Messinger 2012 [19] 1 Neg 7 w 0.5 y 1 40 m 20 eow 4.6 y Yes No No 1:1600 15 Yes 0 (20 m)
2 Neg 16 d 2 m 1 IVIG ongoing 40 w 3 y Yes Yes Yes 1:12,800 14 Yes 0 (10 m)
Banugaria 2012 [25] 1 Neg 4.2 m 8.8 m 2 RTX twice variable 2.5 y No No No 1:25,600 6 No 1:102,400 (before recovery)
Markic 2013 [27] 1 Pos 5 m 17.5 m 1 46 w 20 eow 3 y Yes No No 1:6400 7 Yes 0 (unknown)
Deodato 2013 [26] 1 Neg 7 m 13 m 3 3 w 20 eow 22 m Yes No No 1:3200c 1 Yes 1:100 (16 m)
Stenger 2015 [20] 1 Pos 23 d 11 m 4 Ongoing 20 eow 13 m Yes No No 1:204,800 11 No 1:1200 (no recovery)
Kazi 2016 [28] 1 Pos 6.0 m 2.4 y 5A 3 y 40 w 5.5 y Yes No No 1:204,800 19 Yes 0 (2.5 y)
2 Neg 4.2 m 2 y 5B Ongoing 40 eow 6.9 y Yes No No 1:819,200 52 Yes 0 (4 w)
This study 1 Pos 2.4 m 6.6 y 6 Rap/IVIG ongoing 40 w 2.5 y Yes Yes Nob 1:156,250 3 No 1:31,250 (before recovery)
2 Neg 5.8 m 3.5 y 6 Rap/IVIG ongoing 40 w 2.1 y Yes Yes Yes 1:156,250d 3 Yes 1:31,250 (2 y)
3 Neg 1.9 m 2.3 y 6 Rap/IVIG ongoing 40 w 1.5 y Yes Yes Yes 1:781,250 3 Yes 1:156,250 (1.5 y)
  1. aExcluding Banugaria 2012 (one patient) and the patients in our study, all patients started ERT dosed at 20 mg/kg every other week
  2. bPatient did learn to walk, but lost the ability at the age of 6 years
  3. cTiter was previously 1:25,400
  4. dTiter was previously 1:800,000
  5. Pt Patient, CRIM cross-reactive immunologic material, Pos Positive, Neg Negative, ERT enzyme replacement therapy, w weeks, m months, y years, IM immunomodulation, eow every other week, RTX Rituximab, Vent. free ventilator-free survival, MTX Methotrexate, IVIG intravenous immunoglobulin, Rap Rapamycin
  6. Immunomodulation (IM) protocol used per study:
  7. 1. RTX 375 mg/m2/dose for 4 weekly iv doses followed by maintenance doses; MTX 0.5 mg/kg weekly oral doses; IVIG 500 mg/kg/month.
  8. 2. Cyclophosphamide 15 mg/kg iv on day 1 followed by 2 mg/kg/day iv for 9 days, IVIG 400 mg/kg day 5 through 9; Plasmapheresis day 1, 3 and 5 in week 20, 34 and 56. Between week 34 and 56 oral Cyclophosphamide 2 mg/kg was given. Followed by iv RTX 375 mg/m2/week in weeks 99 through 102 and in weeks 140 and 141.
  9. 3. Plasmapheresis on days 1, 3 and 5. RTX 375 mg/m2 iv once on day 7, directly followed by IVIG (dose not mentioned), with 4 extra IVIG doses over the following 8 months.
  10. 4. RTX 375 mg/m2/dose iv followed by 10 maintenance doses; Bortezomib 1 .3mg/m2/dose in 2 sessions of 4 iv doses. MTX 0.5 mg/kg for 27 oral doses; IVIG 500 mg/kg for 5 doses.
  11. 5. A Cyclophosphamide 250 mg/m2 iv twice; RTX 375 mg/m2/dose in 2 sessions of 4 doses followed by 11 maintenance doses; Bortezomib 1 .3mg/m2/dose in 3 sessions of 4 iv doses; MTX 15 mg/m2 oral doses; IVIG 400-500 mg/kg/month B RTX 375 mg/m2/dose for 4 iv doses followed by RTX maintenance doses 70 weeks later; Bortezomib 1 .3mg/m2/dose in 4 sessions of 4 iv doses; MTX 15 mg/m2 oral doses; IVIG 400-500 mg/kg/month.
  12. 6. RTX 375 mg/m2/dose for 3 iv doses; Bortezomib 1 .3mg/m2/dose for 6 iv doses. Rapamycin daily according to body weight from week 4 onwards; IVIG 500 mg/kg/month.