Real-World Registry on the Pharmacotherapy of Multiple Myeloma and Associated Renal and Pulmonary Impairments in the Greater Gulf Region: Protocol for a Retrospective Real-World Data Study.

BACKGROUND: Multiple myeloma (MM) is the second-most common cancer among hematological malignancies. Patients with active disease may experience several comorbidities, including renal insufficiency and asthma, which may lead to treatment failure. The treatment of relapsed or refractory MM (RRMM) has been associated with multiple factors, causing a decline in progression-free survival as well as overall survival with subsequent lines of therapy. Data about the characteristics of this group of patients in the Greater Gulf region are lacking. OBJECTIVE: The primary objective of this study is to describe the disease characteristics and various treatment approaches or regimens used in the management of patients with RRMM in the Greater Gulf region. METHODS: We will conduct a regional, retrospective study collecting real-world and epidemiological data on patients with MM in countries of the Greater Gulf region. Medical records will be used to obtain the required data. Around 150 to 170 patients' records are planned to be retrospectively reviewed over 6 months without any cross-sectional or prospective intervention. Cases will be collected from Saudi Arabia, the United Arab Emirates, Kuwait, Oman, and Qatar. Descriptive as well as analytical statistics will be performed on the extracted data. The calculated sample size will allow us to estimate the percentages of RRMM cases with acceptable precision while complying with the challenges in light of data scarcity. We will obtain a comprehensive description of the demographic profile of patients with MM; treatment outcomes; the proportion of patients with MM with renal impairment and asthma, chronic obstructive pulmonary disease, or both at the time of diagnosis and any subsequent point; and data related to treatment lines, regimens, and MM-associated morbidities. RESULTS: Patient medical records were reviewed between June 2022 and January 2023 for eligibility and data extraction. A total of 148 patients were eligible for study inclusion, of whom 64.2% (n=95) were male and 35.8% (n=53) were female. The study is currently in its final stages of data analysis. The final manuscript is expected to be published in 2024. CONCLUSIONS: Although MM is a predominant hematological disease, data on its prevalence and patients' characteristics in the Greater Gulf region are scarce. Therefore, this study will give us real-world insights into disease characteristics and various management approaches of patients with MM in the Greater Gulf region. INTERNATIONAL REGISTERED REPORT IDENTIFIER (IRRID): DERR1-10.2196/49861.

Prediction of Multiple Clinical Complications in Cancer Patients to Ensure Hospital Preparedness and Improved Cancer Care.

Reliable and rapid medical diagnosis is the cornerstone for improving the survival rate and quality of life of cancer patients. The problem of clinical decision-making pertaining to the management of patients with hematologic cancer is multifaceted and intricate due to the risk of therapy-induced myelosuppression, multiple infections, and febrile neutropenia (FN). Myelosuppression due to treatment increases the risk of sepsis and mortality in hematological cancer patients with febrile neutropenia. A high prevalence of multidrug-resistant organisms is also noted in such patients, which implies that these patients are left with limited or no-treatment options amidst severe health complications. Hence, early screening of patients for such organisms in their bodies is vital to enable hospital preparedness, curtail the spread to other weak patients in hospitals, and limit community outbreaks. Even though predictive models for sepsis and mortality exist, no model has been suggested for the prediction of multidrug-resistant organisms in hematological cancer patients with febrile neutropenia. Hence, for predicting three critical clinical complications, such as sepsis, the presence of multidrug-resistant organisms, and mortality, from the data available from medical records, we used 1166 febrile neutropenia episodes reported in 513 patients. The XGboost algorithm is suggested from 10-fold cross-validation on 6 candidate models. Other highlights are (1) a novel set of easily available features for the prediction of the aforementioned clinical complications and (2) the use of data augmentation methods and model-scoring-based hyperparameter tuning to address the problem of class disproportionality, a common challenge in medical datasets and often the reason behind poor event prediction rate of various predictive models reported so far. The proposed model depicts improved recall and AUC (area under the curve) for sepsis (recall = 98%, AUC = 0.85), multidrug-resistant organism (recall = 96%, AUC = 0.91), and mortality (recall = 86%, AUC = 0.88) prediction. Our results encourage the need to popularize artificial intelligence-based devices to support clinical decision-making.

Acute Myeloid Leukemia in Qatar (2010-2016): Clinical, Biological, and Prognostic Factors and Treatment Outcomes.

The current study retrospectively evaluated cytogenetic profiles, various prognostic factors, and survival outcomes in 128 acute myeloid leukemia (AML) patients (14 ≤ age ≤ 70 years) admitted to the National Center for Cancer Care and Research (NCCCR), Hamad Medical Corporation, Doha, Qatar, between January 2010 and December 2016. The median age at diagnosis was 43 years, and 80% were less than 60 years old; 75% of patients were male. Cytogenetic analysis was integrated into the World Health Organization 2008 classification and showed that the percentages of normal and abnormal karyotypes were similar, accounting for 48.4% of each group of patients. The AML risk stratification based on cytogenetic analysis resulted in the following distribution: 18% in the favorable risk group, 57% in the intermediate-risk group, 24% in the unfavorable risk group, and 1% unknown. Only 88 patients received therapy with curative intent; 67% achieved complete remission, increasing to 81% after inductions 1 and 2. The median overall survival (OS) and disease-free survival (DFS) in AML patients were 26.6 and 19.5 months, respectively. The 3-year OS and DFS were 40 and 36%, respectively. Prognostic factors including age, gender, white blood cell count, and risk stratification were not significantly associated with treatment outcomes, whereas response to treatment vs. failure was significantly associated with the outcome (p = 0.01). The current study supports the importance of cytogenetics as a useful tool in diagnosis, prognosis, and risk assessment in AML treatment.

Acquired pure megakaryocytic aplasia successfully treated with cyclosporine.

Acquired pure megakaryocytic aplasia is a rare hematological disorder characterized by thrombocytopenia with absent or markedly reduced megakaryocytes in the bone marrow. We report a case of a 25-year-old male diagnosed as acquired pure megakaryocytic aplasia. Treatment with prednisone and intravenous immunoglobulin failed, but he was successfully treated with cyclosporine, with complete remission after 90 days and normal platelet count maintained thereafter.