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Table 1 Recommendations for the evaluation of patients with SMA

From: SMArtCARE - A platform to collect real-life outcome data of patients with spinal muscular atrophy

 

Patient visits (days)

1

14a

28a

63a

180

Every 6 months (every 4 monthsa)

Baseline data including genetic test results

X

 

Current medical history and clinical examination

- including motor milestones in children < 12 years of age

X

X

X

X

X

X

Administration of nusinersen a

X

X

X

X

X

X

Physiotherapeutic assessments:

CHOP INTEND

• All children < 2 years of age

• All patients > 2 years of age without ability to sit

HFMSE

• All patients > 2 years of age with ability to sit

• If CHOP INTEND score > 50: CHOP INTEND and HFMSE

• If CHOP INTEND score > 60: HFMSE instead of CHOP INTEND

RULM

• All patients > 2 years of age with ability to sit (in a wheelchair)

6-MWT

• All ambulant patients > 3 years of age

X

  

X

X

X

ALS Functional Rating Scale (in adult patients)

X

 

X

X

X

Pulmonary function b

X

 

X

X

Documentation of adverse events

Ongoing

  1. aAdditional recommendations under treatment with nusinersen
  2. bIf the patient is sufficiently cooperative due to age