Amiloidosis cardiaca por cadenas ligeras y por transtirretina: características clínicas, historia natural y predictores pronósticos

[Abstract] Introduction and objectives. Light-chain amyloidosis (AL-CA) and transthyretin amyloidosis (ATTR-CA) are the most common types of cardiac amyloidosis (CA). We sought to study the clinical characteristics and prognosis of both diseases. Methods. We conducted a single-centre, retrospective review of all patients diagnosed with CA between 1998 and 2018. Clinical characteristics, complementary tests, survival and other adverse clinical events were studied. Results. We identified 105 patients with CA, 65 ATTR-CA and 40 AL-CA. Mean age was 74.4 years; 24.8% were women. In both groups, heart failure was the most frequent clinical presentation (55.2%). The most prevalent electrocardiographic findings were the pseudoinfarct pattern (68.5%) and a Sokolow-Lyon index <1.5 mV (67.7%), with no differences between the two subtypes of CA. One-year, 3-year, and 5-year survival was 43.3%, 40.4% and 35.4%, respectively, in AC-AL patients, and 85.1%, 57.3% and 31.4% in AC-ATTR patients (p = .004). AL-CA subtype (HR 3.41, CI95% 1.45−8.06, p = .005), previous admission for heart failure (HR 4.25, 95% CI 1.63−11.09, p = .003) and a NYHA class III-IV (HR 2.76, 95% CI 1.09−7.03, p = .033) were independent predictors of mortality, while beta-blocker therapy was associated with longer survival (HR .23, 95% CI .09–.59, p = .002). Conclusions. Differences exist between the clinical presentation of AL-CA and ATTR-CA patients. Both diseases, particularly AL-CA, are associated with poor life prognosis.[Resumen] Antecedentes y objetivos. La amiloidosis cardiaca (AC) por cadenas ligeras (AC-AL) y por transtirretina (AC-ATTR) son los dos subtipos más frecuentes de la enfermedad. Nos propusimos caracterizar clínicamente estas entidades y analizar su pronóstico. Material y métodos. Realizamos una revisión retrospectiva de todos los pacientes diagnosticados de AC entre 1998 y 2018 en un centro español. Además de recoger las características clínicas y los resultados de las pruebas complementarias al diagnóstico, analizamos la supervivencia y la incidencia de desenlaces clínicos adversos. Resultados. Identificamos 105 pacientes con AC, 65 AC-ATTR y 40 AC-AL. La edad media era de 74,4 años; el 24,8% eran mujeres. En ambos grupos la insuficiencia cardiaca (IC) fue la forma de presentación clínica más frecuente (55,2%). Los hallazgos electrocardiográficos más prevalentes fueron el patrón de pseudoinfarto (68,5%) y un índice de Sokolow-Lyon <1,5 mV (67,7%), sin diferencias entre los dos subtipos. La supervivencia a 1, 3 y 5 años fue del 43,3%, 40,4% y 35,4%, respectivamente, en pacientes con AC-AL y de 85,1%, 57,3% y 31,4% en pacientes con AC-ATTR (p = 0,004). El subtipo AC-AL (HR 3,41, IC95% 1,45-8,06, p = 0,005), el ingreso previo por IC (HR 4,25, IC95% 1,63-11,09, p = 0,003) y una clase NYHA III-IV (HR 2,76, IC95% 1,09-7,03, p = 0,033) fueron predictores independientes de mortalidad, mientras que el tratamiento betabloqueante se asoció con una mayor supervivencia (HR 0,23, IC95% 0,09-0,59, p = 0,002). Conclusiones. Existen ciertas diferencias en la presentación clínica de los pacientes con AC-AL y AC-ATTR. Ambas entidades, y muy especialmente la AC-AL, presentan un pobre pronóstico vital

Differentiation syndrome in patients with acute promyelocytic leukemia treated with all- trans retinoic acid and anthracycline chemotherapy: Characteristics, outcome, and prognostic factors

Differentiation syndrome (DS) can be a life-threatening complication in patients with acute promyelocytic leukemia (APL) undergoing induction therapy with all- trans retinoic acid (ATRA). Detailed knowl- edge about DS has remained limited. We present an analysis of the incidence, char- acteristics, prognostic factors, and out- come of 739 APL patients treated with ATRA plus idarubicin in 2 consecutive trials (Programa Espanol de Tratamientos en Hematologíc [PETHEMA] LPA96 and LPA99). Overall, 183 patients (24.8%) ex- perienced DS, 93 with a severe form (12.6%) and 90 with a moderate form (12.2%). Severe but not moderate DS was associated with an increase in mortality. A bimodal incidence of DS was observed, with peaks occurring in the first and third weeks after the start of ATRA therapy. A multivariate analysis indicated that a WBC count greater than 5 x 109/L and an abnor- mal serum creatinine level correlated with an increased risk of developing severe DS. Patients receiving systematic pred- nisone prophylaxis (LPA99 trial) in con- trast to those receiving selective prophy- laxis with dexamethasone (LPA96 trial) had a lower incidence of severe DS. Pa- tients developing severe DS showed a reduced 7-year relapse-free survival in the LPA96 trial (60% vs 85%, P = .003), but this difference was not apparent in the LPA99 trial