Corticosteroids/erythropoietin
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Lack of efficacy in bone marrow necrosis: case report A 21-year-old patient [sex not stated] exhibited lack of efficacy with erythropoietin and unspecified corticosteroids during their treatment for bone marrow necrosis (BMN). The patient, who had haemoglobin SS Disease, presented to the emergency room due to knee and back pain consistent with previous episode of vaso-occlusive crisis. The patient’s medical history included right hip avascular necrosis, recurrent acute chest syndrome and sickle hepatopathy. The patient’s home medications included glutamine [L-glutamine], folic acid and voxelotor and had never received blood products. Eleven weeks prior to presentation, the patient started receiving voxelotor resulting in improved haemoglobin level. On current admission, liver function test revealed elevated ALT and AST with a total bilirubin of 2 g/dL. Voxelotor was not available in hospital, therefore, the patient was off voxelotor until admission day 3 when home supply was received. On admission day 4, a repeat laboratory investigations revealed a dramatic onset of marrow dysfunction with elevated AST and ALT. Urinary microalbumin level was 30 mg/dL. Viral aetiology for aplastic crisis was unrevealing. On admission day 6, the patient developed new fevers, worsening dyspnoea and tachycardia. A chest radiograph revealed bibasilar opacities. Peripheral smear revealed numerous blister cells. Therefore, the patient was started on unspecified broad-spectrum antibacterials. A PCR ruled out COVID-19. Subsequently, the patient’s condition worsened with haemoglobin level of 3 g/dL elevated troponin and non-specific ST changes on ECG. Owing to life-threatening condition, the patient received one dose of erythropoietin at 40 000IU. On admission day 7, the patient received three packed RBC units resulting in transient improvement. On admission day 9, the patient required two more on RBC transfusions. The patient received unspecified corticosteroids as unsuccessful adjunctive therapy to plasmapheresis for hyperhemolysis. Two plasmapheresis were performed with little benefit on admission day 9 and 10. However, the patient developed severe thrombocytopenia by admission day 10. A repeat peripheral smear showed red blood cell fragments raising the suspicion of thrombotic microangiopathy. Therefore, platelets were transfused over the following 5 days with little benefit. On the same day, a bone marrow assessment revealed extensive BMN. Because of refractory treatment course during admission, a complement-mediated mechanism was considered. Therefore, the patient started receiving eculizumab on admission day 13. The platelet count, haemoglobin and absolute reticulocyte count normalised within 5 days after eculizumab initiation. The patient was discharged home on admission day 17. Complement testing was not performed prior to eculizumab. On day 18 of clinical course, total complement (CH50) was low. Twelve days post-eculizumab (one week after discharge), C3 and C4 were found to be elevated. Two weeks after discharge, the patient’
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