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Vol. 42. Issue S2.
Pages 271-272 (November 2020)
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Vol. 42. Issue S2.
Pages 271-272 (November 2020)
451
Open Access
PITFALLS TO DIAGNOSE AND TREAT LIGHT CHAIN AMYLOIDOSIS IN A UNIVERSITY REFERENCE CENTER: 10 YEARS OF EXPERIENCE IN A PUBLIC HEALTH SYSTEM
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R.S. Szora, G.A. Martineza, F.S. Seguroa, F. Fernandesa, A.M.M. Linoa, L.B. Jorgea, J.B. Castellia,b, E.M. Regoa, V.A. Feitosaa, V. Rochaa
a Universidade de São Paulo (USP), São Paulo, SP, Brazil
b Laboratório Fleury, São Paulo, SP, Brazil
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Background: Amyloidosis is caused by tissue deposition of misfolded protein aggregates, leading to organ dysfunction. Light chain amyloidosis (AL) is the most common subtype. Epidemiological data are scarce in Latin America. Objectives: To describe clinical and laboratory characteristics and treatments of patients with systemic AL amyloidosis in a reference center. Methods: Retrospective cohort study of patients with biopsy-proven systemic AL amyloidosis diagnosed from 2009 to 2018 at the Hospital das Clinicas, University of Sao Paulo. Primary endpoint was overall survival (OS). Results: From 83 cases revised, 75 patients met the eligibility criteria. 53% were male with median age of 62 years. Before diagnosis 56% were seen by ≥3 physicians. Referrals to the hematologist were made mainly by nephrologists (46%), cardiologists (23%) and general practitioners (14%). Median time between symptom onset and diagnosis was 9.1 months. In 75% of patients ECOG was ≤ 2 at diagnosis. Initial clinical presentations were: 61% renal disorders, 43% consumptive syndrome, 39% heart disease, 25% gastrointestinal symptoms, 20% neuropathy, 19% fatigue, 15% skin lesions, 12% macroglossia, 11% hepatic disorders and 5% periorbital purpura. Mean number of biopsies performed per patient was 2.5. In 67% a method to subtype amyloid on biopsy was performed: 68% indirect immunofluorescence, 36% immunohistochemistry and mass spectrometry in 1 case. Free light chain was assessed in 49% of cases. 75% were λ subtype. The mean number of organs involved was 2.8 (11% 1 organ, 37% 2, 52% ≥ 3). Main affected organs were: 81% heart, 63% kidney, 52% soft tissue. 34% had coexisting multiple myeloma. Standard Mayo Clinic (SMC) staging was evaluated in 56% of cases: 59% stage III, 31% stage II, 10% stage I. Stage III patients were assessed by European staging: 32% IIIa, 48% IIIb, 20% IIIc. Revised Mayo Clinic staging was available in 21% of patients: 25% in each stage I to IV. Renal staging showed 81% stages I/II, 19% stage III. 81% of patients were treated with chemotherapy (54% melphalan, 43% cyclophosphamide, 10% bortezomib and 18% thalidomide). Median number of cycles was 4. 12% underwent autologous stem cell transplantation (ASCT). 3 patients received doxycycline, 12% only supportive measures and 1 patient underwent kidney transplantation. 40 patients had hematological response assessed: 30% PR, 12.5% VGPR, 17.5% CR, 25% no response and 15% disease progression. Median follow-up time of survivors was 66.3 months and estimated OS was 17% in 5 years. Statistical difference was observed in median OS of SMC stage I-II and III: 51.6 and 14.4 months respectively (p = 0.023). Discussion: The broad clinical spectrum of AL presentation reflects the complexity of diagnostic approach: patients are seen by different specialists, require more than 2 biopsies and are diagnosed late in advanced stages with markers of poor prognosis. Moreover, proteasome inhibitor is not widely available in public system and few patients are eligible for ASCT. Altogether may explain poor outcomes of AL patients in our center. Conclusions: Diagnosis of systemic AL amyloidosis is a challenge in Brazil. Medical education, better tools for diagnosis, establishment of a multidisciplinary team and a registry, availability of disease-modifying drugs and ASCT may improve outcomes.

Idiomas
Hematology, Transfusion and Cell Therapy
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