The clinical features and outcomes of systemic light chain amyloidosis with hepatic involvement

Figures & data

Table 1. Selection criteria for autologous stem cell transplantation.

	Inclusion criteria	Exclusion criteria
Age (years)	18–70	< 18 or >70
ECOG-PS	0–2	> 2
LVEF (%)	> 45	≤45
TBIL (mg/dl)	≤2	> 2
Creatinine(mg/dl)	≤2	> 2
Active infection	No	Yes

ECOG-PS: Eastern Cooperative Oncology Group performance status; LVEF: left ventricular ejection fraction; TBIL: total bilirubin.

Table 2. Baseline characteristics.

	Characteristics (n = 81)
Age at presentation (years)	55 (47, 63)
Male (%)	65 (73.9)
ECOG-PS (%)
0	0 (0.0)
1	33 (37.5)
2	36 (40.9)
3	15 (17.0)
4	4 (4.5)
Organ involvement (%)
Renal	87 (98.9)
Liver	88 (100)
Cardiac	66 (75.0)
Hypotension	28 (31.8)
MAP (mmHg)	85 ± 14
AKP (U/L)	415 (308, 574)
AKp ≥ 500 U/L	30 (34.1)
TBIL (umol/L)	9.7 (6.3, 15.4)
TBIL ≥ 34.2 umol/L	9 (10.2)
AST (U/L)	48 (36, 71)
ALT (U/L)	40 (25, 57)
LDH (U/L)	266(218, 357)
Total liver span (mm)	135 (119, 159)
TC (mmol/L)	8.6 ± 5.1
Platelets (×10^9/L)	305 ± 132
Platelets ≥ 500 × 10^9/L)	8 (9.1)
PT (s)	12.3 ± 1.8
cTnT (ng/ml)	0.048 (0.028–0.139)
cTnT ≥ 0.025 ng/ml	54
cTnT ≥ 0.035 ng/ml	30
NT-proBNP (pg/ml)	1630 (536–4127)
NT-proBNp ≥ 1800 pg/ml	31 (35.2)
Mayo 2004 stage I/II/III	14/23/51
Mayo 2012 stage I/II/III/IV	18/28/31/11
IVST (mm)	12 ± 3
LVEF (%)	64 ± 8
dFLC (mg/L) (n = 80)	94.0 (33.4–139.3)
dFLC ≥ 180 mg/L	15
BMPC (%)	2.5 (0.5–5)
Uric acid (umol/L)	423 ± 145
Albumin (g/L)	26.0 ± 6.3
Creatinine (mg/dl)	1.10 (0.84, 1.90)
24 h urine protein (g)	4.40 (2.50, 7.51)

Values are given as mean ± SEM, median (interquartile range) or percentage. ECOG-PS: Eastern Cooperative Oncology Group performance status; MAP: mean arterial pressure; AKP: alkaline phosphatase; TBIL: total bilirubin; AST: aspartic aminotransferase; ALT: alaninetransaminase; LDH: lactate dehydrogenase; TC: total cholesterol; PT: prothrombin time; cTnT: cardiac troponin T; NT-proBNP: N-terminal natriuretic peptide type B; IVST: interventricular septum thickness; LVEF: left ventricular ejection fraction; dFLC: difference between involved and uninvolved free light chains; BMPC: bone marrow plasma cells.

Table 3. Haematologic and organ responses.

	HDM/ASCT	Bortezomib-based regimen	Other chemotherapy regimen^a
	n = 15	n = 24	n = 9
Haematologic response ≥ PR (%, n)	92.9(14/15)	62.5(15/24)	22.2(2/9)
Median haematologic response time (month)	1	2	1
Liver response (%, n)	60.0(9/15)	37.5(9/24)	11.1(1/9)
Median liver response time (month)	7.5	16	3
Kidney response (%, n)	73.3(11/15)	50.0(12/24)	11.1(1/9)
Median kidney response time (month)	6	6	7
Heart response (%, n)	77.8(7/9)	33.3(8/24)	14.3(1/7)
Median heart response time (month)	8	3	8

HDM/ASCT: high-dose intravenous melphalan and autologous stem cell transplantation; PR: partial response.

^aOther chemotherapy agents include melphalan, thalidomide-based chemotherapy.

Figure 1. (A) The OS of all patients. (B) The OS between patients who had total TBil ≥ 34.2 umol/L and TBil < 34.2 umol/L. (C) The OS between patients who had haematologic response and no haematologic response patients. (D) The OS between patients who had NT-ProBNP ≥ 1800 pg/ml and NT-ProBNP <1800 pg/ml.

Table 4. Results of univariate and multivariate analysis for all-cause mortality.

Variable	HR (95% CI)	p value
Univariate
Age ≥ 55岁	1.59 (0.99–2.53)	.053
ECOG-PS	1.23 (0.91–1.64)	.175
Hypotension	1.37 (0.84–2.23)	.200
TBIL ≥ 34.2umol/L	3.38 (1.64–6.99)	.001
AKp ≥ 500 U/L	1.48 (0.91–2.41)	.112
NT-proBNp ≥ 1800 pg/ml	2.40 (1.27–4.52)	.007
c-TnT ≥ 0.025 ng/ml	1.70 (0.76–3.78)	.196
dFLC ≥ 94 mg/L	0.78 (0.49–1.25)	.297
Multivariate
TBIL ≥ 34.2umol/L	3.29 (1.07–10.07)	.037
NT-proBNp ≥ 1800 pg/ml	2.30 (1.19–4.44)	.013

HR: hazard ratio; CI: confidence interval; ECOG-PS: Eastern Cooperative Oncology Group performance status; TBIL: total bilirubin; AKP: alkaline phosphatase; NT-proBNP: N-terminal natriuretic peptide type B; cTnT: cardiac troponin T; dFLC: difference between involved and uninvolved free light chains.

Data availability statement

All data generated within this study are available from the corresponding author on request.