The Role of Autologous Hematopoietic Stem Cell Transplantation in the Therapy of Systemic AL Amyloidosis

AUTOIMMUNE THROMBOCYTOPENIA

OV Pirogova1, OV Kudyasheva1, AG Smirnova1, VV Porunova1, SV Tolstova1, KR Kalimulina1, MV Chernous1, YuYu Vlasova1, IS Moiseev1, VA Dobronravov2, AD Kulagin1

1 RM Gorbacheva Scientific Research Institute of Pediatric Oncology, Hematology and Transplantation; IP Pavlov First Saint Petersburg State Medical University, 6/8 L’va Tolstogo ul., Saint Petersburg, Russian Federation, 197022

2 Scientific Research Institute of Nephrology; IP Pavlov First Saint Petersburg State Medical University, 6/8 L’va Tolstogo ul., Saint Petersburg, Russian Federation, 197022

For correspondence: Olga Vladislavovna Pirogova, MD, PhD, 6/8 L’va Tolstogo ul., Saint Petersburg, Russian Federation, 197022; Tel.: +7(921)441-90-16; e-mail: bmt.myeloma@gmail.com, dr.pirogova@gmail.com

For citation: Pirogova OV, Kudyasheva OV, Smirnova AG, et al. The Role of Autologous Hematopoietic Stem Cell Transplantation in the Therapy of Systemic AL Amyloidosis. Clinical oncohematology. 2023;16(2):128–36. (In Russ).

DOI: 10.21320/2500-2139-2023-16-2-128-136

ABSTRACT

Aim. To assess the outcomes of autologous hematopoietic stem cell transplantation (auto-HSCT) in systemic AL Amyloidosis patients treated at the R.M. Gorbacheva Scientific Research Institute of Pediatric Oncology, Hematology and Transplantation.

Materials & Methods. In the period from 2005 to 2022, auto-HSCT was performed in 33 patients with systemic AL Amyloidosis. In 7 of them, auto-HSCT was not preceded by the induction therapy “upfront”. From 2012 all patients received induction therapy prior to transplantation. The median age of patients was 54 years (range 38–68 years); among them there were 17 women and 16 men.

Results. The 3-year follow-up period showed hematological response rate of 76 % (95% confidence interval [95% CI] 50–90 %), heart response rate of 27 % (95% CI 6–55 %), renal response rate of 76 % (95% CI 41–93 %), and hepatic response rate of 26 % (95% CI 8–50 %). The 5-year overall (OS) and progression-free (PFS) survivals were 71 % (95% CI 49–85 %) and 53 % (95% CI 32–71 %), respectively. The OS parameters in the group with delayed auto-HSCT, i.e., after induction therapy, were better than in the “upfront” group: 82 % (95% CI 60–93 %) vs. 43 % (95% CI 10–73 %) (p = 0.03). The OS parameters were affected by health status (p = 0.03), reduced left ventricular ejection fraction < 60 % (p = 0.006), stage of heart disease (p = 0.016), and stage III kidney disease (p = 0.007). The PFS parameters depended on ECOG performance status (p = 0.004) and stage of heart disease (p = 0.041).

Conclusion. The presented data confirm the results of the studies emphasizing the importance of induction therapy prior to auto-HSCT in the treatment of systemic AL Amyloidosis. More stringent parameters of renal function, left ventricular ejection fraction, and ECOG performance status can be used as criteria for auto-HSCT eligibility. Reduced melphalan doses, as conditioning regimen, can be administered to patients with pronounced comorbidity.

Keywords: systemic AL Amyloidosis, autologous hematopoietic stem cell transplantation, melphalan, bortezomib.

Received: October 24, 2022

Accepted: March 15, 2023

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