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Table 4 Imaging assessments (amyloid load based on SAP scan)

From: An observational, non-interventional study for the follow-up of patients with amyloidosis who received miridesap followed by dezamizumab in a phase 1 study

Subject number

Amyloid type

SAP scan output

1st value post diagnosis

Value at baseline*

Value at 1st follow-up

Value at 2nd follow-up

Value at 3rd follow-up

Value at 4th follow-up

Value at 5th follow-up

Value at 6th follow-up

Value at 7th follow-up

Sustained responders

012

AA

Liver

Normal

Abnormal

  

Spleen

Abnormal

Abnormal

Abnormal

  

Kidney

Abnormal

Abnormal

Abnormal

  

Adrenals

Abnormal

Abnormal

Abnormal

  

Overall

Moderate

Moderate

Moderate

  

Δ from prior visit

Stable

Stable

107

AL

Liver

Abnormal

Abnormal

Abnormal

Abnormal

  

Spleen

Abnormal

Abnormal

Abnormal

Abnormal

  

Kidney

Obscured

Obscured

  

Adrenals

Obscured

Obscured

  

Overall

Large

Moderate

Moderate

Moderate

  

Δ from prior visit

Better

Stable

108

AL

Liver

Abnormal

Abnormal

Abnormal

Abnormal

Normal

Normal

  

Spleen

Abnormal

Abnormal

Abnormal

Abnormal

Abnormal

Normal

Normal

  

Kidney

Abnormal

Abnormal

Abnormal

Abnormal

Abnormal

Abnormal

Abnormal

  

Adrenals

Obscured

Abnormal

Normal

Normal

  

Overall

Large

Moderate

Large

Moderate

Small

Small

Small

Small

Small

  

Δ from prior visit

Better

Better

Stable

Better

Stable

Stable

Stable

110

AL

Liver

Abnormal

Abnormal

Abnormal

Abnormal

Abnormal

Abnormal

  

Spleen

Abnormal

Abnormal

Abnormal

Abnormal

Abnormal

Abnormal

  

Kidney

Obscured

Abnormal

Obscured

Obscured

Normal

Normal

  

Adrenals

Obscured

Obscured

Normal

Normal

  

Overall

Large

Large

Large

Large

Large

Large

  

Δ from prior visit

Stable

Stable

Stable

Stable

113

AL

Liver

Abnormal

Abnormal

Abnormal

  

Spleen

Abnormal

Abnormal

  

Kidney

Obscured

Equivocal

Equivocal

  

Adrenals

Obscured

Normal

  

Overall

Large

Large

Small

Small

  

Δ from prior visit

Better

116

AL

Liver

Abnormal

Abnormal

Abnormal

Abnormal

Abnormal

Abnormal

Abnormal

  

Spleen

Abnormal

Abnormal

Abnormal

Abnormal

Abnormal

Abnormal

Abnormal

  

Kidney

Obscured

Abnormal

Abnormal

Abnormal

Normal

Obscured

  

Adrenals

Obscured

Abnormal

Obscured

Normal

  

Overall

Large

Large

Moderate

Large

Moderate

Large

Large

  

Δ from prior visit

Stable

Better

Better

Stable

Stable

Stable

121

AL

Liver

Normal

  

Spleen

Abnormal

Abnormal

Equivocal

Equivocal

  

Kidney

Normal

  

Adrenals

Normal

  

Overall

Large

Moderate

  

Δ from prior visit

Better

Better

Declining responders

102

AFib

Liver

Normal

Normal

  

Spleen

Abnormal

Abnormal

Abnormal

Abnormal

Abnormal

  

Kidney

Abnormal

Abnormal

Abnormal

Abnormal

Abnormal

  

Adrenals

Obscured

Obscured

  

Overall

Moderate

Small

Small

Small

Small

  

Δ from prior visit

Stable

Stable

Stable

104

AFib

Liver

Normal

Normal

Normal

Normal

  

Spleen

Abnormal

Abnormal

Abnormal

Abnormal

  

Kidney

Abnormal

Abnormal

Abnormal

Abnormal

  

Adrenals

Obscured

Obscured

Normal

Obscured

  

Overall

Small

Small

Small

Moderate

  

Δ from prior visit

Stable

Stable

105

AFib

Liver

Normal

  

Spleen

Normal

Abnormal

  

Kidney

Normal

Abnormal

  

Adrenals

Normal

  

Overall

None

Small

  

Δ from prior visit

Stable

106

AFib

Liver

Normal

Normal

  

Spleen

Abnormal

Abnormal

  

Kidney

Equivocal

Abnormal

Abnormal

  

Adrenals

Normal

Normal

  

Overall

Small

Moderate

Small

  

Δ from prior visit

Stable

Stable

109

ApoA1

Liver

Abnormal

Abnormal

Abnormal

Abnormal

Abnormal

Abnormal

  

Spleen

Abnormal

Abnormal

Abnormal

Abnormal

Abnormal

Abnormal

Abnormal

  

Kidney

Obscured

Obscured

Obscured

Not functioning

Abnormal

  

Adrenals

Obscured

Obscured

Normal

  

Overall

Large

Large

Large

Large

Large

Large

Large

  

Δ from prior visit

Worse

Stable

Worse

Stable

111

AL

Liver

Abnormal

Abnormal

Abnormal

  

Spleen

Abnormal

Abnormal

Abnormal

  

Kidney

Obscured

Obscured

  

Adrenals

Obscured

  

Overall

Large

Large

Large

  

Δ from prior visit

Stable

114

AL

Liver

Abnormal

Abnormal

Abnormal

Abnormal

Abnormal

Abnormal

  

Spleen

Abnormal

Abnormal

Abnormal

Abnormal

Abnormal

Abnormal

  

Kidney

Abnormal

Abnormal

Abnormal

Abnormal

Abnormal

Abnormal

  

Adrenals

Obscured

  

Overall

Large

Moderate

Moderate

Moderate

Large

  

Δ from prior visit

Better

Better

Worse

Stable

Better

115

AL

Liver

Normal

Abnormal

Abnormal

Abnormal

Abnormal

  

Spleen

Abnormal

Abnormal

Abnormal

Abnormal

Abnormal

  

Kidney

Obscured

Obscured

Obscured

  

Adrenals

Obscured

Obscured

Obscured

  

Overall

Large

Large

Large

Large

  

Δ from prior visit

Stable

Stable

Stable

Better

117

AFib

Liver

  

Spleen

Abnormal

Abnormal

Abnormal

Abnormal

  

Kidney

Abnormal

Abnormal

Abnormal

Abnormal

  

Adrenals

  

Overall

Small

Small

Small

Small

  

Δ from prior visit

Stable

118

AL

Liver

Normal

Normal

Normal

  

Spleen

Abnormal

Abnormal

Abnormal

Abnormal

  

Kidney

Normal

Normal

Normal

  

Adrenals

Normal

Normal

Normal

  

Overall

Moderate

Small

Small

Small

Small

  

Δ from prior visit

Stable

Stable

Stable

Stable

Non-responder

           

119

AL

Liver

Abnormal

Abnormal

Abnormal

Abnormal

Abnormal

  

Spleen

Abnormal

Abnormal

Abnormal

Abnormal

Abnormal

  

Kidney

Abnormal

Abnormal

Abnormal

Abnormal

  

Adrenals

Obscured

  

Overall

Large

Large

Large

Large

Large

  

Δ from prior visit

Stable

Stable

Stable

120

AL

Liver

Normal

  

Spleen

Abnormal

Abnormal

Abnormal

  

Kidney

Abnormal

Abnormal

Abnormal

  

Adrenals

Abnormal

Abnormal

Abnormal

  

Overall

Moderate

Moderate

Moderate

  

Δ from prior visit

Better

Non-therapeutic dose in FIHS

           

001

AA

Liver

Normal

Normal

  

Spleen

Abnormal

Abnormal

Abnormal

Abnormal

Abnormal

Abnormal

  

Kidney

Abnormal

Abnormal

Abnormal

Abnormal

Abnormal

Abnormal

  

Adrenals

Obscured

Obscured

  

Overall

Moderate

Moderate

Small

Small

  

Δ from prior visit

Better

Stable

Better

  1. SAP scans not conducted in patients with ATTR (123, 124 and 125). Follow-up visits are presented in chronological order per parameter as given in the database. These visits will therefore occur at different times relative to baseline for each patient and parameter. Therefore, it is not possible to directly compare values at a particular visit between patients or between parameters within a patient
  2. *The latest value in the database pre-baseline. The baseline date was defined as the date of first pharmacologically-active administration of dezamizumab (i.e. 200 mg in the session) in the FIHS. For subjects who only received a non-pharmacologically-active dose (i.e. < 200 mg in the session), their baseline date was defined as the date of first administration of a non-pharmacologically active dose of dezamizumab
  3. 1st value post diagnosis and value at baseline were measured at the same study visit
  4. AA, serum amyloid A; AFib, fibrinogen A alpha-chain; AL, immunoglobulin light chain; ApoA1, apolipoprotein A-I; ATTR, transthyretin; FIHS, first-in-human study; SAP, serum amyloid P