Abstract 599

Purpose:

This study was undertaken to develop selection guidelines for transplant centers to determine eligibility for stem cell transplantation in patients with light chain amyloidosis.

Patients and Methods:

Patients with biopsy-confirmed immunoglobulin light chain amyloidosis who underwent stem cell transplantation between March 8, 1996, and December 31, 2011, were reviewed in 2 cohorts: those who underwent transplantation between March 8, 1996, and June 30, 2009, and those who underwent transplantation between July 1, 2009, and December 31, 2011 (table 1). A second comparison was undertaken among patients who died before posttransplant day 100 to determine features predictive of early death (table 2).

Results:

A total of 499 patients were identified, 410 in the earlier group and 89 in the later group. After July 1, 2009, significantly fewer transplant recipients had Mayo stage III cardiac involvement. Mortality before posttransplant day 100 was 10.5% (43/410) in the earlier group and 1.1% (1/89) in the later group. In the earlier group, one-quarter of transplant recipients with N-terminal pro-brain natriuretic peptide (NT-proBNP) levels higher than 5,000 pg/mL died by 10.3 months (fig 1, left). When the serum troponin T level was > 0.06 ng/mL, 25% died at 3.7 months (fig 2, right).

Conclusion:

The Mayo staging system is highly predictive for overall survival but not useful for selecting transplant recipients. Patients with serum troponin T levels higher than 0.06 ng/mL or NT-proBNP levels >5,000 pg/mL (not on dialysis) should not be considered acceptable candidates for stem cell transplantation because of unacceptable early mortality.

Table 1.

Comparison of Patients Receiving Transplants Before and After July 1, 2009

Characteristic ComparedTransplant Before 7/1/09 (n=410)Transplant After 7/1/09 (n=89)P Value
Mayo stage, No. (%)a   .02 
104/281 (37) 44/85 (52)  
II 115/281 (41) 31/85 (36)  
III 62/281 (22) 10/85 (12)  
Male, No. (%) 245 (60) 54 (61) .78 
Heart involved, No. (%) 208 (51) 31 (35) .006 
Kidney involved, No. (%) 285 (70) 61 (69) .90 
Albumin, median (IQR), g/dL 2.7 (1.9–3.3) 2.6 (2.1–3.3) .43 
Creatinine, median (IQR, mg/dL) 1.8 (0.9–1.3) 1.0 (0.8–1.2) .002 
 (10%>1.8) (10%>1.5)  
Marrow PC, median (IQR), % 7 (3–13) 8.5 (4–14) .04 
Septal thickness, median (IQR), mm 12 (10–14) 11 (10–14) .08 
EF, median (IQR), % 65 (60–69) 64 (61–67) .31 
Age, median (IQR), y 57.5 (51–63) 58.4 (54–63) .18 
Troponin T, median (IQR), ng/mL 0.01 (0.01–0.03) 0.01 (0.01–0.02) .1 
iFLC, median (IQR), mg/dL 15.5 (7.3–37.4) 11.4 (5.8–29.8) .05 
NT-proBNP, median (IQR), pg/mL 662 (166–2,766) 322 (125–1,100) .02 
 (10%>6,537) (10%>4,023)  
Characteristic ComparedTransplant Before 7/1/09 (n=410)Transplant After 7/1/09 (n=89)P Value
Mayo stage, No. (%)a   .02 
104/281 (37) 44/85 (52)  
II 115/281 (41) 31/85 (36)  
III 62/281 (22) 10/85 (12)  
Male, No. (%) 245 (60) 54 (61) .78 
Heart involved, No. (%) 208 (51) 31 (35) .006 
Kidney involved, No. (%) 285 (70) 61 (69) .90 
Albumin, median (IQR), g/dL 2.7 (1.9–3.3) 2.6 (2.1–3.3) .43 
Creatinine, median (IQR, mg/dL) 1.8 (0.9–1.3) 1.0 (0.8–1.2) .002 
 (10%>1.8) (10%>1.5)  
Marrow PC, median (IQR), % 7 (3–13) 8.5 (4–14) .04 
Septal thickness, median (IQR), mm 12 (10–14) 11 (10–14) .08 
EF, median (IQR), % 65 (60–69) 64 (61–67) .31 
Age, median (IQR), y 57.5 (51–63) 58.4 (54–63) .18 
Troponin T, median (IQR), ng/mL 0.01 (0.01–0.03) 0.01 (0.01–0.02) .1 
iFLC, median (IQR), mg/dL 15.5 (7.3–37.4) 11.4 (5.8–29.8) .05 
NT-proBNP, median (IQR), pg/mL 662 (166–2,766) 322 (125–1,100) .02 
 (10%>6,537) (10%>4,023)  
Table 2.

Survival (Before/After Posttransplant Day 100) in 410 Patients Who Received Transplants Before July 1, 2009

Characteristic ComparedDied Before Day 100 (n=43)Survived After Day 100 (n=367)P Value
Mayo stage, No. (%)a   .002 
    I 4/28 (14) 100/253 (40)  
    II 13/28 (46) 102/253 (40)  
    III 11/28 (39) 51/253 (20)  
Kidney involved, No. (%) 33 (77) 252 (69) .30 
Heart involved, No. (%) 32 (74) 191 (52) <.001 
Albumin, median (IQR), g/dL 2.3 (1.7–3.0) 2.7 (2.0–3.3) .02 
Creatinine, median (IQR), mg/dL 1.2 (1.0–1.7) 1.1 (0.9–1.3) .001 
Marrow PC, median (IQR), % 6 (4–12) 7 (3–13) .90 
Septal thickness, median (IQR), mm 14 (12–16) 12 (10–14) <.001 
EF, median (IQR), % 66 (56–70) 65 (60–69) .97 
Age, median (IQR), y 56 (49–65) 57 (51–62) .60 
Troponin T, median (IQR), ng/mL 0.03 (0.01–0.08) 0.01 (0.01–0.06) <.001 
 (10%>0.125) (10%>0.05)  
iFLC, median (IQR), mg/dL 21.3 (15–43.7) 15.2 (6.4–36.5) .004 
NT-proBNP, median (IQR), pg/mL 2,752 (666–4,645) 600 (170–1,969) <.001 
 (10%>10,753) (10%>5,383)  
Characteristic ComparedDied Before Day 100 (n=43)Survived After Day 100 (n=367)P Value
Mayo stage, No. (%)a   .002 
    I 4/28 (14) 100/253 (40)  
    II 13/28 (46) 102/253 (40)  
    III 11/28 (39) 51/253 (20)  
Kidney involved, No. (%) 33 (77) 252 (69) .30 
Heart involved, No. (%) 32 (74) 191 (52) <.001 
Albumin, median (IQR), g/dL 2.3 (1.7–3.0) 2.7 (2.0–3.3) .02 
Creatinine, median (IQR), mg/dL 1.2 (1.0–1.7) 1.1 (0.9–1.3) .001 
Marrow PC, median (IQR), % 6 (4–12) 7 (3–13) .90 
Septal thickness, median (IQR), mm 14 (12–16) 12 (10–14) <.001 
EF, median (IQR), % 66 (56–70) 65 (60–69) .97 
Age, median (IQR), y 56 (49–65) 57 (51–62) .60 
Troponin T, median (IQR), ng/mL 0.03 (0.01–0.08) 0.01 (0.01–0.06) <.001 
 (10%>0.125) (10%>0.05)  
iFLC, median (IQR), mg/dL 21.3 (15–43.7) 15.2 (6.4–36.5) .004 
NT-proBNP, median (IQR), pg/mL 2,752 (666–4,645) 600 (170–1,969) <.001 
 (10%>10,753) (10%>5,383)  
Disclosures:

No relevant conflicts of interest to declare.

Author notes

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