145 Autologous Hematopoietic Stem Cell Transplant (aHSCT) Is a Safe and Reasonable Treatment in Patients with Primary Systemic Amyloidosis (AL amyloidosis)

Track: Contributed Abstracts
Wednesday, February 13, 2013, 6:45 PM-7:45 PM
Hall 1 (Salt Palace Convention Center)
Akshata Pandit, B.S , The Ohio State University College of Medicine, OH
Lai Wei, Ph.D , Center for Biostatistics, The Ohio State University, Columbus, OH
Patrick Elder, MS , Blood and Marrow Transplantation Program, Arthur G. James Cancer Institute, Columbus, OH
William Falk, B.S , The Ohio State University, OH
Megan Sell, MS, CNP , The Ohio State University, OH
Craig C Hofmeister, MD , Division of Hematology, Ohio State University Medical Center, Columbus, OH
Don M Benson, MD, PhD , Division of Hematology, The Ohio State University, Columbus, OH
Sam Penza, MD , Division of Hematology, The Ohio State University, Columbus, OH
Leslie A Andritsos, MD , Division of Hematology, The Ohio State University, Columbus, OH
Rebecca Klisovic, MD , Division of Hematology, The Ohio State University, Columbus, OH
Sumithira Vasu, MBBS , Division of Hematology, Ohio State University Medical Center, Columbus, OH
William Blum, MD , Division of Hematology, The Ohio State University, Columbus, OH
Steven M Devine, MD , Division of Hematology, The Ohio State University, Columbus, OH
Samantha Jaglowski, MD , Division of Hematology, Ohio State University Medical Center, Columbus, OH
Yvonne Efebera, MD , Division of Hematology, The Ohio State University, Columbus, OH

Background: There is no current standard of care for patients with newly diagnosed AL amyloidosis.  Autologous HSCT is a potential option, but has been limited in its use due to increasd treatment-related mortality (TRM) (38% from one randomized study).  Two large retrospective analyses showed improved overall survival (OS) (70% at 4 yrs and 47% at 5 yrs) of AL amyloid patients undergoing aHSCT compared to control (40% at 4 yrs) with TRM of 13%.

Methods: We retrospectively analyzed the outcomes of 29 newly diagnosed AL amyloidosis patients who underwent aHSCT between 10/1998 and 5/2012.  Hematologic responses were evaluated, along with post-transplant survival and TRM.  Progression-free survival (PFS) and (OS) were determined using the Kaplan-Meier method.   

Results:  Of the patients transplanted, 13 were female and 16 were male. Median age at aHSCT was 56 (range 26-71). Eleven (38%) had involvement of at least 2 organs. Median brain natriuretic peptide and troponin available in 20 patients were 109 pm/ml (range 24-502) and 0.02ng/ml (range 0.01-1.17).  Twenty-one patients (72%) received high dose Melphalan 200 mg/m2. Median CD34+ infused stem cells was 5.00 x 106/kg.  No patients received filgrastim or other colony stimulating factor.  Time to neutrophil and platelet engraftment were 12 and 17 days, respectively.  Three months hematologic response was available in 22 patients and showed complete response, partial response, and stable disease in 15(68%), 2 (10%) and 5(22%), respectively.  The 1, 3, and 5 year PFS were 78%, 68% and 41%, respectively. One, 3, and 5 year OS from diagnosis and from aHSCT were 81, 66, and 66% and 89, 66 and 66% respectively (Table 1).  The 100-day and 1 year TRM were 3.4% (1 patient) and 6.9% (2 patients), respectively. 

 Conclusion: Our results show that autologous HSCT is a reasonable option for patients with newly diagnosed AL amyloidosis. The 100 day and 1 year TRM compares favorably to multiple myeloma patients undergoing autologous HSCT.

Table 1

N

Censored

1 yr survival rate (%)

3 yr survival rate (%)

5 yr survival rate (%)

Median (months)

95% CL (months)

PFS

29

18

78

68

41

44.7

(17.3, NA)

OS-HSCT

29

20

81

66

66

112.0

(13.9, NA)

OS-DX

29

20

89

66

66

117.2

(18.7, NA)

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