Caplacizumab prevents platelet adhesion and has been approved for acquired thrombotic thrombocytopenic purpura (aTTP). This study was retrospective, including all patients diagnosed with aTTP and treated with caplacizumab since commercial availability in 2019 until 28 February 2021 at a single academic hospital with no exclusion criteria. Results used definitions for outcomes in aTTP from the International Working Group Consensus. Ten patients with aTTP received caplacizumab. The median age was 52 years. Six (60%) patients had refractory aTTP while 4 (40%) had newly diagnosed aTTP. The median laboratory values prior to therapy demonstrated: platelet count (PC) 29/uL, LDH 518 U/L (182–1850), ADAMTS13 activity 3% and ADAMTS13 inhibitor 1.4 BU. Everyone received glucocorticoids, rituximab, therapeutic plasma exchange (TPE) and caplacizumab. The median number of TPE was 12 days. Caplacizumab was started at a median of 5 days after the first TPE and the median treatment duration was 31 days. Normalization of PC, LDH and ADAMTS13 activity in days were 5, 3.5, and 32.5, respectively. Six (60%) patients achieved complete response, 3 (30%) had refractory disease and 1 (10%) had relapsed aTTP. No subject suffered abnormal bleeding, or thrombotic event. There were no deaths. Caplacizumab with TPE, glucocorticoids and rituximab was a safe and effective therapy for aTTP.

Mixed phenotype acute leukemia (MPAL) is a rare group of acute leukemias with blasts that co-express antigens of more than one lineage or separate populations of blasts of different lineages. Though treatment guidelines are not well established, the standard of care in treating MPAL remains the acute lymphoblastic leukemia (ALL)-derived chemotherapeutic regimen of hyper-cyclophosphamide, vincristine, doxorubicin (also known by its trade name, Adriamycin), and dexamethasone (CVAD) followed by allogeneic stem-cell transplant (ASCT). Beyond induction chemotherapy, evidence-based treatments remain to be investigated, especially regarding patients who relapse prior to ASCT. This case report illustrates a patient with relapsed MPAL following induction hyper-CVAD who was not immediately eligible for ASCT. After brief treatment with gilteritinib alone, the patient was started on gilteritinib and azacitidine as salvage therapy and achieved and maintained complete remission with incomplete count recovery (CRi) for eight months. Targeted therapy is a novel approach to improve survival rate, but unfortunately, there have been very few studies in the context of MPAL. We report a patient with relapsed FLT3-mutant MPAL who achieved remission using a combination approach with targeted therapy.

Amyloidosis is an underappreciated medical condition with symptoms camouflaging as common medical comorbidities leading to its underdiagnosis due to its systemic involvement. Despite common misconceptions, amyloidosis and its systemic comorbidities are more prevalent and treatable than previously acknowledged by the medical community. There are two major forms of amyloidosis: amyloid light-chain and transthyretin amyloidosis. Each of these have a distinct pathophysiology, diagnostic work-up, treatment, and prognosis. The patient described in this study was diagnosed with transthyretin cardiac amyloidosis months after presenting with heart failure of unknown etiology. Usually, clinicians presume that heart failure results from common comorbidities such as hypertension, diabetes, and hyperlipidemia. Here, the correct etiology was transthyretin cardiac amyloidosis. The patient had five admissions for heart failure symptoms prior to a physician identifying the etiology as cardiac transthyretin amyloidosis. After initiating the transthyretin stabilizer tafamidis, the patient did not experience another heart failure exacerbation. This vignette provides an example of the clinical presentation, diagnostic work-up, and treatment of a patient with cardiac transthyretin amyloidosis. The review of the literature focuses on the epidemiology, and clinical symptoms that should prompt an evaluation for cardiac amyloidosis as well as the diagnostic and therapeutic options are available. Transthyretin cardiac amyloidosis is a rare and underdiagnosed disease, while heart failure is a highly prevalent condition. This clinical vignette seeks to provide education and awareness to an overlooked medical disorder.

Mucor is a well-known opportunistic organism; however, it rarely infects the gastrointestinal (GI) tract. Although known to transiently colonize healthy immunocompetent individuals, the incidence of mucormycosis is increasing in patients with hematologic malignancies, which can have serious consequences in an immunocompromised host. This abstract presents 3 patients from a single institution with Mucor colonization of their stool. Each patient had a hematologic malignancy and was neutropenic when diagnosed with Mucor colonization of the stool. The duration of neutropenia ranged from 1 to 76 days with median of 31 days at the time of diagnosis. Patients were observed for symptoms of GI tract invasion, and they received antimold prophylaxis while neutropenic. Despite GI shedding, no mold sinusitis or pneumonia or gastroenteritis was noted. Gastrointestinal mucormycosis was never noted during their hospital course. This case series emphasizes the importance of not dismissing mold colonization of the stool in a patient with prolonged neutropenia who is at high risk of invasive GI tract mucormycosis. When stool cultures grow Mucor in patients with prolonged neutropenia, anti-Mucor therapy is indicated and may prevent invasive mold infection during the period of risk.