Sequential Use of Romiplostim after Eltrombopag for Refractory Thrombocytopenia in Hydrocarbon-Induced Myelodysplasia

We describe the clinical course of a 65-year-old male patient who suffered from hydrocarbon-induced myelodysplasia and was successfully treated with the thrombopoietin receptor agonist (TPO-RA), romiplostim. Myelodysplastic syndromes (MDS) are characterized by ineffective hematopoiesis, cytopenias, and increased risk of leukemic transformation. Here, we present a clinical vignette of MDS-associated thrombocytopenia refractory to first-line drugs as well as the TPO-RA, eltrombopag. To date, romiplostim is an U.S. Food and Drug Administration (FDA)-approved drug for idiopathic thrombocytopenic purpura and thrombocytopenia secondary to liver disease. Of note, currently the FDA advises against its use in MDS based on previous long-term safety concerns. Since the therapeutic options for thrombocytopenia in MDS patients are sparse, repurposing and reassessing romiplostim in this setting have been the focus of recent studies. At the time of writing, no published double-blind randomized clinical trials have conducted a head-to-head comparison between romiplostim and eltrombopag in thrombocytopenic MDS patients. To the best of our knowledge, for a thrombocytopenic patient in the setting of MDS, this is the first documented report of refractory clinical response after a 2-year use of eltrombopag in which replacement of treatment with romiplostim resulted in sustained physiological counts of thrombocytes within four weeks.

When Should We Think of Myelodysplasia or Bone Marrow Failure in a Thrombocytopenic Patient? A Practical Approach to Diagnosis

Thrombocytopenia can arise from various conditions, including myelodysplastic syndromes (MDS) and bone marrow failure (BMF) syndromes. Meticulous assessment of the peripheral blood smear, identification of accompanying clinical conditions, and characterization of the clinical course are important for initial assessment of unexplained thrombocytopenia. Increased awareness is required to identify patients with suspected MDS or BMF, who are in need of further investigations by a step-wise approach. Bone marrow cytomorphology, histopathology, and cytogenetics are complemented by myeloid next-generation sequencing (NGS) panels. Such panels are helpful to distinguish reactive cytopenia from clonal conditions. MDS are caused by mutations in the hematopoietic stem/progenitor cells, characterized by cytopenia and dysplasia, and an inherent risk of leukemic progression. Aplastic anemia (AA), the most frequent acquired BMF, is immunologically driven and characterized by an empty bone marrow. Diagnosis remains challenging due to overlaps with other hematological disorders. Congenital BMF, certainly rare in adulthood, can present atypically with thrombocytopenia and can be misdiagnosed. Analyses for chromosome fragility, telomere length, and germline gene sequencing are needed. Interdisciplinary expert teams contribute to diagnosis, prognostication, and choice of therapy for patients with suspected MDS and BMF. With this review we aim to increase the awareness and provide practical approaches for diagnosis of these conditions in suspicious cases presenting with thrombocytopenia.

Mucormycosis as the Elusive Cause of an Aortic Thrombus and Tissue-Obliterating Abscess

Invasive mucormycosis is an increasingly common cause of morbidity and mortality in hematologic malignancy patients. Early consideration of the diagnosis is essential in at-risk patients, exhibiting suggestive signs and symptoms. A 56-year-old female with acute myeloid leukemia initially presented with neutropenic fever before subsequently developing dense hemiplegia due to septic emboli to the spine and multifocal abscesses. These findings were later determined to be a result of a disseminated mucor infection and represented a rare manifestation of the disease. Despite the disseminated nature of the infection, identification of the causative organism was initially impeded by limitations in obtaining a tissue sample in a severely thrombocytopenic patient, as is common among hematologic malignancy patients. As a result of this limitation, diagnosis was ultimately made via PCR on bronchiolar lavage fluid. Early consideration of the diagnosis with prompt initiation of treatment is of utmost importance in this invasive infection. Further research is needed to identify and validate rapid, minimally invasive strategies for early diagnosis of mucormycosis.

Retrospective Cohort Analysis of Pedal Procedures in the Thrombocytopenic Patient

Thrombocytopenia is an important medical condition to understand prior to performing procedures in the foot and ankle. We have set forth to highlight factors a physician should take into consideration before performing procedures in the thrombocytopenic patient. A retrospective cohort analysis at a large academic institution was undertaken utilizing a cohort discovery tool to discover incidence and management strategies for patients with foot-related conditions that require in-office procedures. We demonstrate that a full history and physical are important to guide treatment along with complete blood count testing prior to intervention. We included all patients at the institution that underwent a foot and ankle procedure in-office with podiatric surgery over 10 years where thrombocytopenia was demonstrable via complete blood count within 3 months of the procedure. Patients’ charts were reviewed for 1 year following podiatric intervention and outcomes were recorded. The cohort reveals that patients with thrombocytopenia have many advanced comorbidities but performing procedures in this cohort is safe. Complications from procedures included erythrocyte transfusion, ulcer recurrence, need for formal surgical intervention, infection, falls, and death. We then provide a brief discussion about the etiology and management options available for thrombocytopenia.

Platelet transfusion goals in oncology patients

Despite the advances in platelet component preparation and transfusion support over the years, platelet products remain a limited resource due to their short (5 day) shelf life, and therefore their optimal use in the non-bleeding thrombocytopenic patient continue to draw much attention. There have been a number of national and international guidelines for platelet transfusion therapy in patients with hematologic diseases, some within the last 1-2 years that have incorporated key randomized controlled trials (RCTs) which address issues, such as the optimal platelet dose, the most appropriate threshold for prophylactic platelet transfusions, and whether prophylactic platelet transfusions are superior to therapeutic-only platelet transfusion practices for the prevention life-threatening bleeding in patients with hypoproliferative thrombocytopenia. This review highlights key RCTs and recent systematic reviews focused on optimal platelet transfusion therapy in adult and pediatric patients with hypoproliferative thrombocytopenia secondary to chemotherapy or hematopoietic stem cell transplant (HSCT), discuss how recent innovations in platelet component processing may affect transfusion efficiency, and introduce renewed concepts on adjuvant therapies to prevent bleeding in the hypoproliferative thrombocytopenic patient.