ABSTRACT
Pure red cell aplasia (PRCA) is a rare bone marrow disorder characterized by a severe reduction or absence of erythroid precursor cells, without affecting granulocytes and megakaryocytes. Immunosuppressive therapies, particularly cyclosporine, have demonstrated efficacy as a primary treatment. This study aims to develop a predictive model for assessing the efficacy of cyclosporine in acquired PRCA (aPRCA). This retrospective study encompasses newly treated aPRCA patients at the General Hospital of Tianjin Medical University. Diagnosis criteria include severe anemia, and absolute reticulocyte count below 10 × 109/L, with normal white blood cell and platelet counts, and a severe reduction in bone marrow erythroblasts. Cyclosporine therapy was administered, with dose adjustments based on blood concentration. Response to cyclosporine was evaluated according to established criteria. Statistical analysis involved logistic multi-factor regression, generating a predictive model. The study included 112 aPRCA patients with a median age of 63.5 years. Patients presented with severe anemia (median Hb, 56 g/L) and reduced reticulocyte levels. Eighty-six patients had no bone marrow nucleated erythroblasts. Primary PRCA accounted for 62 cases (55.4%), and secondary PRCA accounted for 50 cases (44.6%). Univariate analysis revealed that ferritin, platelet to lymphocyte ratio (PLR), and CD4/CD8 ratio influenced treatment response. Multivariate analysis further supported the predictive value of these factors. A prediction model was constructed using ferritin, PLR, and CD4/CD8 ratio, demonstrating high sensitivity and specificity. The ferritin, PLR, and CD4/CD8-based nomogram showed good predictive ability for aPRCA response to cyclosporine. This model has potential clinical value for individualized diagnosis and treatment of aPRCA patients.
Subject(s)
Cyclosporine , Nomograms , Red-Cell Aplasia, Pure , Humans , Cyclosporine/therapeutic use , Red-Cell Aplasia, Pure/drug therapy , Red-Cell Aplasia, Pure/blood , Middle Aged , Female , Male , Retrospective Studies , Aged , Adult , Immunosuppressive Agents/therapeutic use , Treatment Outcome , Aged, 80 and overABSTRACT
Se describe el caso de una paciente con insuficiencia renal crónica terminal en programa de hemodiálisis y en tratamiento con eritropoyetina recombinante humana (epoetina alfa) por vía subcutánea, que a los 8 meses de tratamiento presentó una aplasia pura de la serie roja secundaria al desarrollo de anticuerpos neutralizantes anti-eritropoyetina. A pesar de la retirada del tratamiento y de haberse ensayado tratamiento inmunosupresor con corticoides y gammaglobulinas la paciente sigue presentando unos requerimientos transfusionales muy elevados y niveles circulantes de eritropoyetina indetectables. La eritroblastopenia secundaria al tratamiento con eritropoyetina recombinante humana es una complicación rara pero grave en pacientes urémicos. En los últimos años se ha descrito un aumento de su incidencia, especialmente en pacientes tratados con epoetina alfa por vía subcutánea. Por ello, recientemente la Agencia Española del Medicamento ha contraindicado administrar epoetina alfa por vía subcutánea en pacientes con insuficiencia renal crónica
We report the case of a woman with end-stage renal disease on maintenance hemodialysis and treated with recombinant human erythropoietin (epoetin alfa) administered subcutaneously, who developed a pure red cell aplasia secondary to the development of neutralizing antibodies anti-erythropoietin after 8 months of treatment. Despite epoetin withdrawal and immunosuppressive treatment with corticosteroids and gammaglobulins the patient still has high red blood cell transfusion requirements and undetectable plasma erythropoietin levels. Pure red cell aplasia secondary to the development of neutralizing antibodies anti-erythropoietin is a rare but severe complication associated with the use of recombinant human erythropoietin in uremic patients. In recent years, the incidence of this complication has sharply increased, specially associated with the use of epoetin alfa administered subcutaneously. For this reason, the Spanish Drug Agency has recently contraindicated treating uremic patients with epoetin alfa administered subcutaneously
Subject(s)
Female , Adult , Humans , Autoantibodies/blood , Epoetin Alfa/adverse effects , Epoetin Alfa/immunology , Red-Cell Aplasia, Pure/blood , Uremia/drug therapy , Case-Control Studies , Renal Insufficiency, Chronic/drug therapy , Red-Cell Aplasia, Pure/classification , Uremia/blood , Uremia/complicationsABSTRACT
El trasplante alogeneico de células progenitoras hematopoyéticas (TCPH) con incompatibilidad ABOentre el donante y el receptor puede en ocasiones asociarse a trastornos en la progenie eritroide de-sarrollada a partir de la médula ósea trasplantada, caracterizado por un funcionamiento tardío, inadecuado e in-completo de la misma. En este contexto, la aplasia pura de serie roja es la complicación más severa. Se han inten-tado tratamientos para la aplasia pura de serie roja post-TCPH con eritropoyetina o plasmaféresis, con relativo éxito. Algunos autores han informado también la utilización de globulina antilinfocitaria, asumiendo que dicha aplasia se-lectiva de la serie roja en la médula ósea trasplantada es mediada por un mecanismo inmune. En este trabajo se describe un paciente portador de una leucemia aguda en quien se realizó un TCPH alogeneico (ABO incompatible con su donante). Teniendo niveles bajos de aglutininas contra el grupo sanguíneo de la donante, desarrolló una aplasia pura de serie roja post - TCPH. La misma no mejoró con tratamiento con eritropoyetina o con un refuerzo de progenitores hematopoyéticos de sangre periférica de la misma donante ( boost), resolviéndose totalmente lue-go de un tratamiento exitoso con globulina antilinfocitaria de origen equino (AU)