Table 2 Specific statements

Statement 4

To continue treatment, patients must show improvements or stabilization of disease as measured by clinical scales, laboratory markers, and patient-reported outcomes

Validation of treatment should be shown in at least 1 clinical and 1 patient-reported outcome (a and b):

a) clinical

 Improvement in 6MWT distance or the timed 25-foot (7.6 m) walk (T25FW) of ≥ 10% over baseline or stabilization after 10% improvement

 Any improvement in FVC or FEV1 over baseline or stabilization after 1 year

 Decline in LVEF < 10% from baseline or stabilization after 1 year

 Decline of uKS of ≥ 20% from baseline

b) PROs

 no adverse change in numerical value of 1 out of 3 of the following:

  Eq-5D-5L OR MPS-HAQ

  Beck Depression Score (≥ 13 yrs)

  BPI pain severity score

100

Statement 5

Due to the clearly defined baseline, follow-up assessments and the definition of “stabilization and improvement” (see statement 4), all adult patients with MPS IVA should have early access to treatment with elosulfase alfa

18

(this statement was not approved as it was considered redundant)

Statement 6

In patients with MPS IVA in a very advanced stage of disease, treatment of elosulfase alfa must be discussed individually

100

Statement 7

Treatment with elosulfase alfa in MPS IVA patients could be stopped if stabilization or improvement (as described in statement 4) is not fulfilled after 3 years of treatment or upon physician or patient decision

94

Statement 8

Treatment with elosulfase alfa in MPS IVA patients should be stopped if severe unmanageable infusion related reactions or an additional life-threatening disease occur

100

Statement 9

Treatment with elosulfase alfa in MPS IVA patients could be stopped if the patient is not compliant regarding follow-up assessments

100

Statement 10

Treatment with elosulfase alfa in MPS IVA patients could be stopped if the patient missed ≥ 20% of their scheduled elosulfase alfa infusions, excluding surgeries or other severe medical conditions, problems in drug delivery or organizational issues of the hospital

100