Evaluation of the therapeutic efficacy and tolerability of current drug treatments on the clinical outcomes of paediatric spinal muscular atrophy type 1: A systematic review.

Spinal muscular atrophy (SMA) is a severe hereditary lower motor neuron disorder characterised by degeneration of alpha motor neurons in the spinal cord, resulting in progressive weakness and paralysis of proximal muscles. A systematic literature search was carried out by using PRISMA guidelines and searching through different databases that could provide findings of evidence on the health outcomes of the approved therapies for the management of paediatric SMA type 1 regarding efficacy with follow-up in terms of motor and respiratory functions and the tolerability and incidence of adverse drug reactions (ADRs) post-treatment from real-world publications. Half of the publications (50%) had a prospective observational design. Eight studies (66.7%) assessed nusinersen, and three studies (25%) assessed onasemnogene abeparvovec with a duration of follow-up ranging from 6 months to 3 years to evaluate the motor and respiratory functions using different assessment tools, hospitalisation rates, and the tolerability and incidence of ADRs post-treatment. The three currently approved treatments for SMA type 1 provided good support and health outcomes in terms of motor function, respiratory outcomes, reduction of hospitalisations, and improvement of survival. Nevertheless, uncertainties regarding continued improvement after long-term illness and the generalizability of results are still unknown.

Potential therapeutic effects of different mouthwashes for alleviation and treatment of oral complications of 5-fluorouracil-based chemotherapy in patients with colon cancer.

OBJECTIVE: The primary endpoint of the present study was to assess the potential therapeutic effects of three different mouthwashes for alleviation and treatment of oral complications (OCs). The secondary endpoint was to assess patients' perceptions and daily functional activities after therapy of OCs in patients with colon cancer receiving 5-fluorouracil (5-FU)-based chemotherapy regimens. METHODS: A prospective, randomised controlled study carried out on 90 patients with colon cancer eligible for 5-FU-based chemotherapy regimens at the oncology centre, Istanbul, Turkey. Patients were randomly randomised into three groups (30 patients in each group) and received a single mouthwash. The first group (group A) received benzydamine at a dose of 15 mL; the second group (group B) received sodium bicarbonate at a dose of 1.2-2.4 g in 240 mL of water; and the third group (group C) received glutamine suspension 10 g. Patients were assessed for the occurrence of oral complications based on the WHO scale for oral mucosa evaluation and National Cancer Institute Common Terminology Criteria for Adverse Events version 4.0. Oral pain was assessed using a visual analogue scale alongside assessment of patients' perceptions and daily functional activities based on the Rotterdam Symptom Checklist. RESULTS: A total of 119 oral complications were reported, including mouth dryness (n=56, 47.1%), oral mucositis (n=31, 26.1%) and oral pain (n=32, 26.8%). At the end of the study, patients of group A and group B significantly suffered from mouth dryness (p=0.0001), oral mucositis (p=0.029) and oral pain (p=0.039) compared with patients in group C. Although there was no significant change, group C patients showed a slight improvement in psychological discomfort, activity levels and quality of life at the end of the study. CONCLUSION: The present study showed that benzydamine and sodium bicarbonate mouthwashes were significantly less effective for the alleviation and treatment of oral complications compared with glutamine among patients with colon cancer receiving 5-FU-based chemotherapy regimens.

The potential chemo-preventive roles of Malus domestica against the risk of colorectal cancer: A suggestive insight into clinical application.

Biologically active compounds in fruit-derived ingredients, particularly phytochemicals, have significant potential to modulate and mitigate many pathological processes in the development of disease conditions, including cancer. Colorectal cancer (CRC) remains a major public health issue. Nonetheless, prevention is an important step in lowering the rate of mortality from this cancer. Currently, the link between apple consumption and improved health is becoming remarkably evident and is reported to be beneficial for human health. Phytochemicals, such as flavonoids and other polyphenol compounds extensively available in apple fruits, have well-known positive effects on health outcomes and the potential to combat and prevent the development of CRC, including antioxidant, anti-proliferative, and anti-carcinogenic effects. This review describes the bioactive compounds derived from apple fruits, particularly the polyphenols and flavonoids, their proposed mechanisms responsible for their bioactive properties and health-promoting attributes that could provide potential chemo-preventive effects against the risk of CRC development. The conclusion of the review provides insights into the potential effects of apple-derived bioactive compounds and proposes the need for more clinical studies in large trials for future strategies regarding the valuable effects of apple phytochemicals, which might be therapeutic candidates in the campaign against CRC.

Prognostic factors of ado-trastuzumab emtansine treatment in patients with metastatic HER-2 positive breast cancer.

BACKGROUND: Ado-trastuzumab emtansine is an antibody-drug conjugate that combines the cytotoxic activity of emtansine with human epidermal growth factor receptor 2-targeted antitumor features of trastuzumab. OBJECTIVE: We conducted a study of metastatic breast cancer patients treated with trastuzumab emtansine. By evaluating progression-free survival, overall survival, and response rates, we aimed to find prognostic factors of trastuzumab emtansine treatment. METHODS: Our study is a single-center, retrospective, observational study. We have clinical data from 78 patients treated with trastuzumab emtansine for metastatic breast cancer, from May 2016 through May 2019, at Kartal Dr Lutfi Kirdar Education and Research Hospital, Medical Oncology Department. Our objective is to assess the survival and response rates in trastuzumab emtansine-treated individuals and the factors associated with survival. The factors we analyzed were cancer antigen 15-3 sensitivity, Eastern Cooperative Oncology Group-Performance Status, presence or absence of visceral metastases, presence or absence of cranial metastases, and treatment-associated thrombocytopenia. RESULTS: Among 78 patients, median progression-free survival was 7.8 months, and overall survival was 21.1 months. Twenty of the patients had an objective tumor response. The results showed that trastuzumab emtansine was tolerable with a manageable safety profile and consistent with the results of the previous literature. Mostly seen adverse events were anemia, thrombocytopenia, fatigue, and increased levels of alkaline phosphatase. Patients with Eastern Cooperative Oncology Group-Performance Status = 2 had worse progression-free survival and overall survival compared to ones with Eastern Cooperative Oncology Group-Performance Status < 2; progression-free survival and overall survival are worse in cancer antigen 15-3-sensitive breast cancer patients. According to our findings, treatment-associated thrombocytopenia was a significant prognostic factor for survival. Patients with thrombocytopenia had 12 months progression-free survival, whereas patients without thrombocytopenia had only 4.1 months progression-free survival. In like manner, overall survival was much better in the thrombocytopenia-experienced patients as 29.5 versus 11.8 months. CONCLUSIONS: Trastuzumab emtansine prolongs progression-free survival and overall survival with a manageable safety profile. Thrombocytopenia, Eastern Cooperative Oncology Group-Performance Status, and cancer antigen 15-3 are correlated with progression-free survival and/or overall survival.

Impact of clinical pharmacy recommendations and patient counselling program among patients with diabetes and cancer in outpatient oncology setting.

INTRODUCTION: The simultaneous occurrence of diabetes and cancer may complicate the management of both conditions resulting in poor prognosis and more deterioration of patient-related outcomes. OBJECTIVE: To assess the effective provision of clinical pharmacy services and pharmacist-led counselling program on improving patient-related outcomes among patients with diabetes and newly diagnosed with cancer during chemotherapy administration. METHODS: A single-centre, prospective, randomised, controlled study was carried out on patients with diabetes newly diagnosed with cancer during chemotherapy administration at the outpatient oncology setting. Patients were assigned as a normal care group receiving only normal care by the oncology care providers and an intervention group receiving both normal and clinical pharmacy care through an extensive oral and written patient education, pharmacotherapy optimisation and regular recommendations for diabetic self-care activities with three-month follow-up. RESULTS: Patients within the intervention group showed a better glycaemic control (p = .049), a significant increase in medication adherence (p = .0049), a significant increase in diabetes self-care activities, including diet (p = .037), self-monitoring of blood glucose (p = .027) and foot care (p = .0085) and reported a lower deterioration in quality of life. CONCLUSION: Patients with diabetes and cancer receiving chemotherapy experienced improved patient-related outcomes after clinical pharmacy intervention and counselling program compared to the normal care group.