Successful treatment of refractory secondary immune thrombocytopenia (antiphospholipid antibody syndrome-associated) with the combination of rituximab and romiplostim at the cost of severe bone pain: A case report and review of literature.

INTRODUCTION: Immune thrombocytopenia is an autoimmune disorder associated with increased thrombocyte destruction and impaired production in the bone marrow. Proposed mechanisms include an antibody or autoreactive T-cell-associated autoimmunity and thrombopoietin deficiency among others. Clinical manifestations are predominantly mucocutaneous hemorrhages including petechiae, purpura, mucosal bleeding in the urinary or the gastrointestinal tracts, menorrhagia, and epistaxis. The purpose of the treatment is to prevent bleeding rather than normalizing the platelet counts. First-line treatments include corticosteroids ± intravenous immunoglobulin and Anti-D which mainly decrease antibody-mediated platelet destruction and increase the number of peripheral Tregs. Second-line and subsequent therapies include splenectomy, chimeric anti-CD20 antibody (rituximab), which eliminates B cells and act as an immunomodulatory agent, and Thrombopoietin receptor agonists (romiplostim), which promote platelet production. CASE REPORT: We describe a 40-year-old male patient diagnosed with immune thrombocytopenia that was refractory to first-line corticosteroid and intravenous immunoglobulin and second-line romiplostim monotherapy treatments.Management and outcome: The patient was given the romiplostim and rituximab combination which not only successfully treated thrombocytopenia but also resulted in grade 3 bone pains and the patient's subsequent refusal to continue therapy. DISCUSSION: Common adverse effects of rituximab are infusion reactions and prolonged immunosuppression; those of romiplostim include thrombosis, headaches, arthralgia-myalgia, and gastrointestinal symptoms. This case shows that romiplostim has not caused any discernible side effects when given alone, while combination with rituximab resulted in severe bone and joint pains. We hypothesize that this combination regimen shows a synergistic effect both in terms of efficacy and adverse-effect probability and/or severity.

A Near-Complete Response to Treatment with Gemcitabine plus nab (®)-Paclitaxel in a Patient with Metastatic Pancreatic Cancer and Poor Performance Status: A Case Report.

Patients with metastatic pancreatic adenocarcinoma and poor performance status (PS) are typically excluded from clinical trials of new systemic treatments. Due to concerns that such patients cannot tolerate the greater toxicity sometimes associated with combination chemotherapy regimens, the recommended treatment for pancreatic cancer patients with poor PS is gemcitabine monotherapy. We report the case of a 79-year-old female with pancreatic adenocarcinoma metastatic to the lungs, with multiple comorbidities and an Eastern Cooperative Oncology Group PS of 3, who achieved a rapid and prolonged objective response to gemcitabine plus nab (®)-paclitaxel. The patient received a total of 11 cycles of treatment. Although her disease was well controlled with gemcitabine plus nab-paclitaxel, she died just over 11 months after diagnosis as a result of her comorbid conditions compounded by treatment-related hematologic toxicity. This case suggests that patients with metastatic pancreatic adenocarcinoma and poor PS may benefit from first-line combination therapy with gemcitabine plus nab-paclitaxel. Further study of this regimen in such patients is warranted.

Strong and Sustained Response to Treatment with Carboplatin plus Nab-Paclitaxel in a Patient with Metastatic, Triple-Negative, BRCA1-Positive Breast Cancer.

Our case describes a 51-year-old female, diagnosed in April 2008 with a triple-negative, BRCA1-positive, infiltrating ductal carcinoma of the left breast. Initial platinum-based therapy resulted in a complete regression until November 2009, when a recurrence of the disease was detected. As no evidence of metastasis was found, a dose-dense regimen of doxorubicin plus cyclophosphamide was administered, followed by paclitaxel. The patient was actively monitored until March 2012, when brain metastases were discovered and successfully treated with whole-brain radiation therapy. Three months later, the patient experienced severe abdominal pain, and CT scans revealed extensive metastatic disease, including a large mass in the abdomen and more than 20 bilateral pulmonary metastases. Treatment commenced with carboplatin plus nab-paclitaxel. However, carboplatin was stopped after 4 cycles due to persistent neutropenia, and nab-paclitaxel was continued as monotherapy. Whole-body CT scans performed in October 2012 and March 2013 revealed a significant response to therapy, and the patient reported feeling well and being fully mobile. No treatment-related adverse events were observed. A routine brain MRI scan carried out on April 18, 2013, revealed a recurrence of brain metastases; however, CT scans confirmed that disease progression was not systemic, but confined to the central nervous system. Despite the initiation of treatment with irinotecan plus temozolomide on April 24, the patient died on July 2, 2013. The author believes that this case is the first report of a robust response to nab-paclitaxel monotherapy in triple-negative BRCA1-positive breast cancer, and that it supports further studies of nab-paclitaxel in this aggressive indication.