Comparison of Cyclosporine A and Cyclosporine A Combined with Corticosteroid in the Treatment of Acquired Pure Red Cell Aplasia / 中国实验血液学杂志

OBJECTIVE@#To evaluate the efficacy, safety and relapse of cyclosporine A (CsA) and CsA combined with corticosteroid (CS) as the frontline therapy for patients with newly diagnosed acquired pure red cell aplasia (aPRCA).@*METHODS@#The clinical features, treatment responses, relapses and clinical outcomes of patients with newly diagnosed aPRCA in Peking Union Medical College Hospital (PUMCH) from January 2015 to May 2020 were analyzed retrospectively. All the enrolled patients had been treated with either CsA or CsA+CS for at least 6 months and had been followed up for at least 12 months, with complete clinical data and consent forms.@*RESULTS@#96 patients including 72 treated with CsA and 24 treated with CsA+CS were enrolled. With comparable baseline characteristics and follow-up periods, patients treated with CsA or with CsA+CS had similar overall response rates (ORRs) and complete response rates (CRRs) at the 3rd, 6th and 12th month and at the end of follow-up (P>0.05). Meanwhile, no significant difference was found between the two groups in the optimal ORR, optimal CRR, time to response or time to complete response. CsA+CS and CsA groups had similar adverse event (AE) rates, but CsA+CS group had higher CS-related infection rate (P <0.05). One patient in CsA+CS group died of multiple infections. As for the relapse, the two groups had compatible relapse rates at different time points, time to relapse, overall relapse rate and relapse-free survival (P>0.05). CsA exposure time, rather than different therapy regimens, was the only influence factor for either ORR or relapse rate (P <0.05).@*CONCLUSION@#CsA monotherapy has similar efficacy, AE rate and relapse rate as compared with CsA+CS for patients with newly diagnosed aPRCA, and shows less CS-related AEs such as infection.

Pure red cell aplasia and anti-erythropoietin antibodies in patients on hemodialysis: a report of two cases and a literature review / Aplasia pura de células vermelhas e anticorpo antieritropoietina em pacientes de hemodiálise: relato de dois casos e revisão da literatura

ABSTRACT Introduction: Anemia is a frequent multifactorial complication of CKD seen in patients on dialysis derived mainly from impaired erythropoietin (EPO) production. A less common cause of anemia in individuals with CKD is pure red cell aplasia (PRCA) secondary to the production of anti-EPO antibodies. Objective: This paper aimed two describe two cases of PRCA secondary to the production of anti-EPO antibodies including choice of treatment, patient progression, and a literature review. Materials: This study included the cases of two patients with CKD on hemodialysis with severe anemia in need of specific investigation and management. Results: Patient 1 with CKD secondary to hypertension treated with EPO for 7 months showed persistent decreases in hemoglobin (Hb) levels despite the subcutaneous administration of increasing doses of EPO; the patient required recurring blood transfusions. Workup and imaging tests were negative for the main causes of anemia in individuals with CKD on dialysis. Patient 2 with CKD secondary to adult polycystic kidney disease had been taking EPO for 2 years. The patient developed severe abrupt anemia the month he was started on HD, and required recurring transfusions to treat the symptoms of anemia. Workup and imaging findings were inconclusive. Specific laboratory tests confirmed the patients had anti-EPO antibodies. After six months of immunosuppressant therapy (corticosteroids + cyclosporine) the patients were stable with Hb > 9.0 g/dl. Conclusion: PRCA is a rare condition among patients on dialysis treated with rhEPO and should be considered as a possible cause of refractory anemia. Treating patients with PRCA may be challenging, since the specific management and diagnostic procedures needed in this condition are not always readily available.

RESUMO Introdução: Anemia é complicação frequente da Doença Renal Crônica (DRC) em pacientes dialíticos. Apresenta caráter multifatorial principalmente pela insuficiente produção de eritropoietina (EPO). Situação rara causadora de anemia na DRC é Aplasia Pura de Células Vermelhas (APCV), em decorrência da produção de anticorpos anti-EPO. Objetivo: Descrever 2 casos de APCV com formação de anticorpos anti-EPO, sua abordagem clínica, evolução e revisão de literatura. Métodos: Dois pacientes em hemodiálise que desenvolveram anemia grave, necessitando investigação e manejo específico. Resultados: Paciente nº 1: feminina, 75 anos, DRC secundária à hipertensão arterial. Após 7 meses com EPO desenvolveu queda persistente em valores de hemoglobina (Hb) mesmo com incremento em doses EPO SC, necessitando transfusões de sangue recorrentes. Extensa investigação laboratorial e de imagem resultou negativa para principais causas de anemia. Paciente nº 2: masculino, 66 anos, DRC secundária à DRPA, há 2 anos em uso de EPO. No mês de entrada em HD desenvolveu anemia severa, também exigindo transfusões recorrentes para tratamento da anemia sintomática. Extensa investigação laboratorial e por imagem, sem chegar a uma conclusão definitiva. Em ambos os casos a presença de anticorpos anti-EPO foi confirmada por exames laboratoriais específicos. Terapia imunossupressora resultou em estabilização do quadro e Hb > 9,0 g/dl em ambos os pacientes, 6 meses após início do tratamento. Conclusão: APCV é condição rara entre pacientes dialíticos que recebem EPOHuR e deve ser lembrada como causa de anemia refratária. Seu manejo específico e diagnóstico laboratorial nem sempre acessível, tornando desafiadora a condução dos casos para o nefrologista.

Mesotelioma y aplasia pura de serie roja / Malignant mesothellioma and pure red cell aplasia

La aplasia pura de serie roja (APSR) es un síndrome clínico definido por la disminución de precursores eritroides maduros. Los pacientes con esta enfermedad presentan reticulopenia pero su cuenta de plaquetas y granulocitos es normal. La patofisiología es heterogénea y puede ser congénita o adquirida. En su forma secundaria, se encuentra asociada con distintos tipos de tumores como los timomas, las leucemias y los linfomas. El interés del caso que presentamos es la presencia de una aplasia pura de serie roja en un paciente con un mesotelioma. El tratamiento utilizado para esta aplasia consistió en el uso de prednisona y eritropoyetina recombinante humana. Ninguno de los tratamientos utilizados mejoró la anemia, por lo que el paciente fue mantenido a base de transfusiones periódicas. Por otra parte, el mesotelioma es una neoplasia muy agresiva con una vida media de tan solo dos a ocho meses. El paciente presentó una sobrevida de 18 meses y un estado físico aceptable.

"Steroid responsive pure red cell aplasia".

A young female with pure red cell aplasia with brisk response to prednisolone therapy is described. The patient has remained in remission on a small dose of prednisolone for 10 months.

Aplasia pura de la serie eritroide en el lupus eritematoso sistémico (LES): a propósito de un caso / Pure red-cell aplasia in a patient with sle.report of one case and review or the literature

Nosotros reportamos un caso de LES que desarrolla aplasia pura de la serie eritroide, hallazgo infrecuente en esta enfermedad, que respondió favorablemente al tratamiento administrado con prednisona y ciclosfosfamida (EV). Se revisa la literatura al respecto

Utilidad de los bolos de metilprednisolona en aplasia pura de serie rija (APSR) adquirida, recurrente y refrectaria / Utility of methylprednisolone bolus in acquired pure red aplasia, recurrent and refractory

Un enfermo con aplasia pura de serie roja (APSR) adquirida de 106 meses de evolución, presentó una remisión con ciclo fosfamida y en la recaída fue refractario a numerosas medidas terapéuticas: corticoesteroides, ciclofosfamida (dosis total acumulativa de 38 gr.), azatioprina (dosis total acumulativa de 9 gr.); la esplenectomía y timecetomía, así como plasmaféresis intensiva. Después obtuvo cifras normales de hemoglobina con tratamiento a base de metilprednisolona, administrado a una dosis total de 9 gr.; respuesta que continúa por 10 meses sin necesidad de algún tratamiento. Esta experiencia indica que por su patogenia autoinmune, los casos de APSR recurrentes y refractarios pueden ser candidatos a este enfoque terapéutico