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Rare Diseases Drug Development
Published in Wei Zhang, Fangrong Yan, Feng Chen, Shein-Chung Chow, Advanced Statistics in Regulatory Critical Clinical Initiatives, 2022
Shein-Chung Chow, Shutian Zhang, Wei Zhang
Velcade® (Bortezomib) is an antineoplastic agent (a proteasome inhibitor) indicated for the treatment of multiple myeloma and mantle cell lymphoma (see Table 1.1). Multiple myeloma is the second most common cancer (an incurable cancer) of the blood, representing approximately 1% of all cancers and 2% of all cancer deaths. It is estimated that approximately 45,000 Americans have multiple myeloma with about 15,000 new cases diagnosed each year. Only about percent of multiple myeloma patients survive longer than five years with the disease. Although the disease is predominantly a cancer of the elderly (the average age at diagnosis is 70 years of age) recent statistics indicate both increased incidence and younger age of onset. Thus, multiple myeloma is considered a rare disease and meet the requirement for Fast-Track designation for expedited review.
Haematological malignancy
Published in Peter Hoskin, Peter Ostler, Clinical Oncology, 2020
Symptoms of multiple myeloma typically present in three ways: Bone marrow infiltration causes anaemia; thrombocytopenia is usually not prominent but more commonly there may be a bleeding disorder owing to the effects of macroglobulinaemiaBone destruction results in local pain, pathological fracture or neurological complications such as nerve root or spinal cord compression – bone pain is present in two-thirds of patients presenting with myelomaMetabolic and biochemical disturbance occurs, including: High levels of paraprotein causing hyperviscosity, resulting in confusion and headacheRenal failure, which is present in around one-third of patients as defined by a raised blood urea and creatinine causing nausea, vomiting, malaise, fluid retention or itchingHypercalcaemia, which is present in around one-third of patients who present with myeloma resulting in thirst, polyuria, dyspepsia, nausea, vomiting, constipation or confusion
Differential Diagnosis in Forensic Entomology: Mites versus Pathologies and Taphonomy
Published in Heather M. Garvin, Natalie R. Langley, Case Studies in Forensic Anthropology, 2019
César Sanabria-Medina, Luz Elena Cifuentes, Maria Alexandra Lopez-Cerquera
The general morphology of bone lesions initially suggested they were taphonomic, but the specific agents and mechanism were unknown, leaving some uncertainty. Therefore, the pathologist and forensic anthropologists investigated additional hypotheses. The possibility of multiple myeloma was considered. Multiple myeloma is a type of cancer of the bone marrow with an abnormal proliferation of plasma cells. The myeloma cells also interfere with the normal production of osteoclasts and osteoblasts: the myeloma cells produce a substance that signals the osteoclasts to accelerate bone resorption, causing the bone undergoing remodeling to resorb without new bone formation to replace it (American Cancer Society, 2018). Macroscopically, the lytic processes of the myeloma tend to have sharp edges. In this case, however, they were rounded or polished, although it is not possible to establish if the polish was caused by taphonomic effects (Ortner, 2003). Multiple myeloma generally affects people over 40 years of age, but has been reported in younger individuals (Kasenda et al., 2011). Still, it would be exceptionally rare to see such disease progression in a 19-year-old male. Furthermore, according to his relatives, the man had no medical history of this disease. Samples were sent to the histopathology laboratory where the microscopic diagnosis definitively ruled out multiple myeloma.
The diagnostic and prognostic value of haematologic parameters in multiple myeloma patients
Published in Hematology, 2023
Fuyan Han, Nan Sheng, Chenchen Sheng, Jing Meng
In this study, 151 patients with MM newly diagnosed in the Department of Haematology in the Second Hospital of Shandong University from 2010 to 2018 were retrospectively analysed. Among them, 58 were females aged 11–78 years and 93 were males aged 15–84 years. All patients were diagnosed according to the 2016 World Health Organization diagnostic criteria for multiple myeloma [10]. Excluding other diseases of the blood system, autoimmune diseases, inflammation-related diseases, malignant tumours and other diseases such as hypertension, diabetes, and cardiovascular disease, all patients did not receive radiotherapy and chemotherapy therapy. A total of 153 age and sex-matched healthy volunteers were collected as the control group. All subjects signed informed consent. We obtained permission from the hospital's research Ethics Committee.
Cell division cycle 37 change after bortezomib-based induction therapy helps to predict clinical response and prognosis in multiple myeloma patients
Published in Hematology, 2023
Wuqiang Lin, Xiuli Chen, Heyong Zheng, Zhenjie Cai
Multiple myeloma patients’ characteristics were gained for study analysis. Bone marrow specimens were taken from multiple myeloma patients after recruitment and after bortezomib-based induction treatment (4-6 cycles), as well as from disease controls and healthy controls after bone marrow examinations. Then, CD138+ plasma cells in bone marrow specimens were isolated, and CDC37 expression in plasma cells was detected by reverse transcription-quantitative polymerase chain reaction (RT-qPCR), and the experiment was triplicated. Total RNA isolations were completed using PureZOL RNA isolation reagent (Bio-Rad, America). cDNA syncretization were performed using the ReverTra Ace® qPCR RT Kit (Toyobo, Japan). Next, qPCR was completed using KOD SYBR® qPCR Mix (Toyobo, Japan). Relative quantification calculation was based on the 2-ΔΔCT method. The primers were: CDC37, F: 5’-TGAAGACGAGACGCACC-3’, R: 5’-TCAGTTTCCTCTGGCACTCG-3’; GAPDH, F: 5’-GAAGGTGAAGGTCGGAGTC-3’, R: 5’-GAAGATGGTGATGGGATTTC-3’ [18].
Prognostic evaluation and staging optimization of the Mayo Additive Staging System (MASS) in real world for newly diagnosed multiple myeloma patients
Published in Hematology, 2023
Yongqin Cao, Yingying Gong, Xin Zhou, Chao Sun
With continuous progress in new immunotargeted drug therapies, the prognosis of multiple myeloma (MM) has improved greatly; however, there is still no cure due to its heterogeneity[1]. Currently, the Revised International Staging System (R-ISS) and the Mayo Myeloma Stratification and Risk Adjusted Therapy Stratification System 3.0 (mSMART3.0) are widely used in clinical practice[2, 3]. However, these two prognostic models have limitations. Therefore, the Mayo Additive Staging System(MASS), which is believed to reflect the compound effect of high-risk disease characteristics better, has recently been proposed internationally[4]. Through a retrospective analysis of the same group of newly diagnosed MM patients, this study discusses the prognostic evaluation value of this model in the real world for newly diagnosed MM patients and further optimizes the application process.