: Autologous Stem Cell Transplantation in AL amyloidosis in two centers from Latin America

MARIA LOURDES POSADAS MARTINEZ

Simposio; XVIIIth International Symposium on Amyloidosis; 2022

Contribution ID : 429Track / Type : ISA 2022 Abstract SubmissionFormat : Undecided (Oral or Poster Presentation)Title : Autologous Stem Cell Transplantation in AL amyloidosis in two centers from Latin AmericaAuthor(s) : Riva, Eloísa; Brulc, Erika; Arbelbide, Jorge; Carretero, Marcelina; Posadas-Martínez, Lourdes; Aguirre, Adela; Muxí, Pablo; Nucifora, ElsaAutologous Stem Cell Transplantation in AL amyloidosis in Latin AmericaRiva Eloísa1,3, Brulc Erika B2, Arbelbide Jorge2, Garrido David3 Posadas-Martinez Lourdes2 Carrerero Marcelina2,Aguirre Adela2, Muxí Pablo1, Nucifora Elsa21Hospital Británico, Montevideo, Uruguay2Hospital Italiano de Buenos Aires 3Hospital de Clínicas Dr. Manuel Quintela, Montevideo, UruguayBackground: in Light-chain amyloidosis (AL) the depth of the hematologic response is a predictive marker of survival. Autologous hematopoietic stem cell transplantation (ASCT) is a useful strategy for disease control. Only 25% of patients with newly diagnosed AL are candidates for ASCT. However, in those who are candidates, deeper and more durable responses with improved OS and PFS have been reported after ASCT.Objective: To describe the characteristics of patients with AL Amyloidosis who received an ASCT in centers of Latin America, evaluate the pre- and post transplant response, overall survival (OS) and progression-free survival (PFS).Material & Methods: a retrospective cohort of all consecutive patients with newly diagnosed AL amyloidosis based on the Latin American Multiple Myeloma Study Group (GELAMM) registry, from January 2004 to March 2021. Categorical variables were described as percentages and absolute frequency. Quantitative variables were described as the median and interquartile range (IQR). Results: Thirty-three patients from the entire registry (n=212, 15.5%) received ASCT, 6 were excluded from the present analysis due to insufficient data. Patients' characteristics are described in table 1. The median age was 59 (IQR 50-65) and the main organs involved were kidney (51%) and heart (48%). Thirteen patients had cardiac involvement at diagnosis, 4 were in NYHA functional class III, of whom 2 received a heart transplantation prior to chemotherapy, and another 2 received chemotherapy treatment with improvement in cardiac function, which made the subsequent ASCT possible. A single patient required dialysis prior to transplantation, subsequently receiving a kidney transplant.The median number of treatment lines before ASCT was one, (IQR 1-1) being CYBORD the most used chemotherapy regimen (70%) . Only one patient received ASCT as the only frontline therapy.Regarding ASCT, the median time to engraftment was 12 days (IQR 11-14), the median time to hospital discharge was 22 days (IQR 18-29), 3 patients required intensive care admission, and one patient died in the first 100 days due to sepsis. Sixteen patients deepened the response achieved prior to transplantation, 13 of them achieving complete remission. No patient underwent subsequent maintenance.The median follow-up of the entire cohort was 60 months (IQR 21-90). Nine patients relapsed, the median time to relapse was 86 months (73-124 months) and the incidence rate was 0.6/100 patients-months. Relapse-free survival at 12 months was 100%, at 24 months was 95% (69-99), and at 120 months was 29% (5-58) (Figure 1). Median progression free survival (PFS) was 59 months (22-86). PFS at 12 months was 96% (75-99), at 24 months 68% (46-81), and at 120 months 8% (2.24) (Figure 2). The incidence rate of PFS was 1.6/100 patients-months. The median overall survival (OS) was 65 months (22-101). OS at 12 months 96% (IC 76-99), at 24 68% (IC 46-82), at 48 60% (38-76) and at 120 months 23% (IC 8-45%) (Figure 3). There were 8 deaths, 5 due to disease progression.Summary & Conclusion: ASCT in AL Amyloidosis allows deepening responses and prolonging OS and PFS, with a low rate of complications, provided it is performed in adequately selected patients. In this cohort, patients receiving upfront ASCT had long survival, even without maintenance.References: 1- Gertz M. Biol blood marrow transplant 21 (2015) 1339e1342 2- Huang, X., Wang, Q., Chen, W. et al. BMC Med 12, 2 (2014). https://doi.org/10.1186/1741-7015-12-2 3- D'Souza, A. et al. Journal of Clinical Oncology 2015 33:32, 3741-3749 4- Kumar, S. et al. Journal of Clinical Oncology 2012 30:9, 989-995 https://doi.org/10.1200/JCO.2011.38.5724Table 1ASCT in Light-chain amyloidosis (N=27)Clinical CharacteristicsAge (median, IQR)59 (50-65)Sex (M/F)16/11AL kappa/lambda6/21Charlson (median, IQR)3 (2-5)P. Status (ECOG) (median, IQR)1 (1-2)Bone Marrow infiltration %,(median, IQR)11% (5-20)N. Organs involved (median, IQR)2 (1-2)Organ involvement- Heart13 (48%)NYHA class (III/IV)4/16MAYO Score12293243- Kidney14 (51%)- Renal insuficiency (creatinine > 2 mg/dl)3 (11%)-Liver2 (7%)- Gastrointestinal5(18%)- Peripheral Neuropathy9 (33%)- Dysautonomia10 (37%)Frontline therapy (pre-ASCT)CyBorD19Len/dex1Daratumumab/Bortezomib2Other3None1Number of lines before transplant (median)1 (RIC 1-1)Pre-ASCT response (N=24) Post ASCT response (N=25)PR 12PR 1VGPR 7VGPR 8CR 3CR 16 Stable disease 2Stable disease 0Figure 1. Kaplan-Meier progression free survival (1) and overall survival (2) estimates in AL patients with ASCT (n=27).