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Protocol for a randomised controlled trial to study cryoprevention of chemotherapy-induced oral mucositis after autologous stem cell transplantation.

INTRODUCTION: A majority of patients who receive myeloablative therapy prior to hematopoetic stem cell transplantation develop oral mucositis (OM). This adverse cytotoxic effect manifests as oral mucosal erythema and ulcerations and frequently necessitates high doses of morphine for pain alleviation. OM may also interfere with food intake and result in parenteral nutrition, weight loss and impaired quality of life. To date, there have been a few studies of evidence-based interventions for prevention of OM. Cooling the oral mucosa using ice chips in conjunction with chemotherapy is known to red

Crystalline deposits in the cornea and various areas of the kidney as symptoms of an underlying monoclonal gammopathy: a case report.

BACKGROUND: Plasma cell dyscrasias (PCD) are characterized by an abnormal production of intact monoclonal immunoglobulins or parts such as heavy or light chains. In most cases, the monoclonal protein (also termed paraprotein) is produced by a clonal plasma cell population. The production of monoclonal proteins can result in deposits of various types and localization depending on the type, amount, and electrochemical properties of the paraprotein. One histopathologic presentation, albeit rare, are crystalline deposits. They can form in various organs and hence cause a wide spectrum of symptoms.

High-dose single-fraction IMRT versus fractionated external beam radiotherapy for patients with spinal bone metastases: study protocol for a randomized controlled trial.

BackgroundStereotactic body radiation therapy (SBRT)using intensity-modulated radiotherapy (IMRT) can be a safe modality for treating spinal bone metastasis with enhanced targeting accuracy and an effective method for achieving good tumor control and a rigorous pain response.Methods/designThis is a single-center, prospective randomized controlled trial to evaluate pain relief after RT and consists of two treatment groups with 30 patients in each group. One group will receive single-fraction intensity-modulated RT with 1 × 24 Gy, and the other will receive fractionated RT with 10 × 3 Gy. The ta

Patients' perspectives on the derivation and use of organoids.

Organoid research is enhancing understanding of human development and diseases as well as aiding in medication development and selection, raising hopes for even more future therapeutic options. Nevertheless, this work raises important ethical issues and there is a paucity of data regarding patients' perspectives on them. We report on 60 interviews with adult patients or parents of pediatric patients from diverse disease populations who receive medical care at a major academic research institution in the United States. Interviewees expressed broad support for organoid development and use. Howev

Online education for rare genetic diseases: a systematic review.

IntroductionRare genetic diseases are, collectively, not in fact rare. However, educational opportunities focused on rare genetic disease can be limited. The Internet has increased the availability of education related to rare genetic disease and is accessible to a diverse range of people who seek out such information, including healthcare professionals, researchers, students, patients, and the public.PurposeTo assess the potential educational outreach of the Internet, this systematic literature review will appraise the landscape of what education for rare genetic disease is available online,

Factor X deficiency: An overlooked cause of bleeding in the newborn.

Inherited factor X (FX) deficiency is a rare autosomal recessive bleeding disorder, presenting with various bleeding manifestations ranging from nonspecific bruising to life-threatening intracranial hemorrhage. It is often misdiagnosed as hemorrhagic disease of the newborn. It is characterized by prolonged prothrombin time, as well as activated partial thromboplastin time, and needs FX level assay for definitive diagnosis. Here, we describe a case of a newborn with FX deficiency who presented with frank umbilical stump bleeding.

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… Declaration of patient consent The authors certify that they have obtained all appropriate patient consent forms. In the form, the patient(s) has/have given his/her/their consent for his/her/their images and other clinical information to be reported in the journal. …

The MODIFY Study Protocol: An Open-Label, Single-Arm, Multicenter, Prospective Pragmatic Study of Ixazomib-Based Triple-Drug Therapy in Chinese Patients with Multiple Myeloma.

BACKGROUND: Although the bortezomib-based triple-drug therapy is considered as a front-line therapy for multiple myeloma (MM) in Chinese patients, increased level of toxicity leads to treatment dissatisfaction. Treatment with ixazomib, an oral proteasome inhibitor, has demonstrated better efficacy and safety profile without increasing the toxicity. In this study, we investigate the safety and clinical outcomes of Chinese patients with newly diagnosed MM (NDMM) who transitioned from a bortezomib-based triple-drug therapy to an ixazomib-based triple-drug therapy in a real-world clinical setting.

Stereotactic Body radiotherapy and pedicLE screw fixatioN During one hospital visit for patients with symptomatic unstable spinal metastases: a randomized trial (BLEND RCT) using the Trials within Cohorts (TwiCs) design.

BackgroundSpinal metastases can lead to unremitting pain and neurological deficits, which substantially impair daily functioning and quality of life. Patients with unstable spinal metastases receive surgical stabilization followed by palliative radiotherapy as soon as wound healing allows. The time between surgery and radiotherapy delays improvement of mobility, radiotherapy-induced pain relief, local tumor control, and restart of systemic oncological therapy. Stereotactic body radiotherapy (SBRT) enables delivery of preoperative high-dose radiotherapy while dose-sparing the surgical field, al

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… Informed consent forms will be stored in a secure archive of the study site. Plans for collection, laboratory evaluation, and storage of biological specimens for genetic or molecular analysis in this trial/future use {33} Not applicable. No biological specimens will be collected. … The participant information materials and informed consent form are available from the corresponding author on request. Competing interests {28} HMV receives research funding from Elekta, European Commission, Netherlands Organisation for Health Research and Development, Dutch Cancer Foundation. …

Proceedings of the 27th European Paediatric Rheumatology Congress (PReS 2021) : Virtual. 19-21 September 2021

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Life-Sustaining Treatment States in Korean Cancer Patients after Enforcement of Act on Decisions on Life-Sustaining Treatment for Patients at the End of Life.

PurposeIn Korea, the "Act on Hospice and Palliative Care and Decisions on Life-sustaining Treatment for Patients at the End of Life" was enacted on February 4, 2018. This study was conducted to analyze the current state of life-sustaining treatment decisions based on National Health Insurance Service (NHIS) data after the law came into force.Materials and methodsThe data of 173,028 cancer deaths were extracted from NHIS qualification data between November 2015 and January 2019.ResultsThe number of cancer deaths complied with the law process was 14,438 of 54,635 cases (26.4%). The rate of patie

Pediatric Sneddon syndrome presenting with early-onset liver fibrosis: a rare case report.

Sneddon syndrome (SS) is a rare neurocutaneous disorder consisting of livedo racemosa (LR) and cerebrovascular disease with an incidence of 4 per 1 000 000 annually. It may be idiopathic or associated with autoimmune or genetic factors, including deficiency of adenosine deaminase 2 (DADA2). We describe a 17-month-old girl with recurrent fevers, hepatosplenomegaly, LR, and progressive liver fibrosis. A pathogenic ADA2 mutation and LDLR mutation associated with familial hypercholesterolemia (FH) were detected by genetic testing. Despite corticosteroids, etanercept, and immunosuppressants, she co

The role of ixazomib as an augmented conditioning therapy in salvage autologous stem cell transplant (ASCT) and as a post-ASCT consolidation and maintenance strategy in patients with relapsed multiple myeloma (ACCoRd [UK-MRA Myeloma XII] trial): study protocol for a Phase III randomised controlled trial.

BACKGROUND: Multiple myeloma (MM) is a plasma cell tumour with an approximate annual incidence of 4500 in the UK. Therapeutic options for patients with MM have changed in the last decade with the arrival of proteasome inhibitors and immunomodulatory drugs. Despite these options, almost all patients will relapse post first-line autologous stem cell transplantation (ASCT). First relapse management (second-line treatment) has evolved in recent years with an expanding portfolio of novel agents, driving response rates influencing the durability of response. A second ASCT, as part of relapsed diseas

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… Female participants who: (a) Are not of childbearing potential, OR (b) If they are of childbearing potential, agree to practice two effective methods of contraception, at the same time, from the time of signing the informed consent form until 90 days after the last dose of study drug, OR (c) Agree to … Source data verification will involve direct access to participant notes at the participating hospital sites and the central collection of copies of consent forms and other relevant investigation reports. …

Association between the severity of constipation and sarcopenia in elderly adults: A single-center university hospital-based, cross-sectional study.

The aim of the present study was to investigate the association between the severity of constipation and sarcopenia in elderly adults. We conducted a single-center university hospital-based, retrospective cross-sectional study of consecutive outpatients aged ≥65 years from 2017 to 2020. Patients were included in the study if all of the following information were available from medical records: Patient's profile (age, sex, body mass index), laxative/prokinetics use, evaluation of sarcopenia, nutritional status, and questionnaires concerning the severity of constipation [Constipation Scoring Sys

Психосоциальные и поведенческие корреляции ВИЧ-инфицированных мужчин, имеющих сексуальные отношения с мужчинами в Португалии: перекрестное исследование.

Справочная информация/задачи: Мужчины, вступающие в половые отношения с мужчинами (МСМ), несут в Португалии непропорционально большую долю ВИЧ-инфекции, однако социально-психологические характеристики ВИЧ-инфицированных лиц по-прежнему недостаточно описаны. В этом перекрестном исследовании изучалось, как навязчивое сексуальное поведение, интернализованный хомонегатив, психологическое расстройство, трудности с регулированием эмоций и употребление веществ связаны с ВИЧ серостатусом, независимо от социально-демографических характеристик. Методы: Удобная выборка из 480 Cisgender MSM (66 человек, ж

Hemoglobin SS Nigerian Woman First Diagnosed at the Age of 52 years with Manifestation Mimicking Tuberculosis of the Spine.

Sickle cell anemia (SCA) is an inherited disorder of hemoglobin due to the presence of abnormal hemoglobin in a homozygous state. Manifestation is usually in infancy or early childhood due to gradual decrease in hemoglobin F level as age advances. Diagnosis in middle age is unusual. We present a woman who was diagnosed of SCA for the first time at middle age. The aim was to bring to the knowledge of physicians that patients with SCA can also present late so high index of suspicion is required to make diagnosis. A 52-year-old woman presented to orthopedic clinic with complaints of generalized b

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… Declaration of patient consent The authors certify that they have obtained all appropriate patient consent forms. In the form the patient(s) has/have given his/her/their consent for his/her/their images and other clinical information to be reported in the journal. …

Belantamab mafodotin плюс венетоклакс в t()11;14)-положительное повторное или повторное несколько миелом – испытание BELI(E)VE

Множественная миелома (ММ) — широко неизлечимая злокачественная клетка B-клеток, которая в основном затрагивает пожилых людей. 10% от рака гематологов. Несмотря на достижения в области терапии, которые значительно увеличили средний показатель общего выживания (ОС), ММ по-прежнему является клинической проблемой с плохим прогнозом в отношении пациентов, отказывающихся от лечения в нескольких классах лекарственных препаратов. Существует острая потребность в новаторских и индивидуализированных методах лечения, в том числе для пациентов с транслокацией (см.11;14), подгруппу, характеризующуюся особо

Transferability of a US claims-based machine learning model for ATTRwt-CM identification: a retrospective evaluation in a German setting.

Cardiac amyloidosis (CA), a fatal and progressive cardiomyopathy is characterized by amyloid deposition within the myocardium. The main forms of CA include transthyretin amyloidosis (ATTR-CM; distinguished into a hereditary and wildtype form [ATTRv and ATTRwt]) and light-chain amyloidosis (AL). Transthyretin amyloidosis with cardiomyopathy (ATTR-CM) is increasingly recognized among heart failure (HF) patients, despite its underestimated prevalence as a rare disease. ATTR-CM is challenging to diagnose due to its broad phenotypic overlap with HF, yet a timely differentiation of CA is crucial, as

Ход работы сессии 31st Европейский педиатрический конгресс по педиатрии: часть 1.

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Whole-body Computed Tomography Versus Dual Energy X‑ray Absorptiometry for Assessing Heterotopic Ossification in Fibrodysplasia Ossificans Progressiva.

Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare genetic disorder that leads to heterotopic ossification (HO), resulting in progressive restriction of physical function. In this study, low-dose, whole-body computed tomography (WBCT) and dual energy X-ray absorptiometry (DXA) were evaluated to determine the preferred method for assessing total body burden of HO in patients with FOP. This was a non-interventional, two-part natural history study in patients with FOP (NCT02322255; date of registration: December 2014). In Part A (described here), WBCT and DXA scans were individually ass

Ixazomib in combination with carboplatin in pretreated women with advanced triple-negative breast cancer, a phase I/II trial of the AGMT (AGMT MBC-10 trial).

BACKGROUND: Triple-negative breast cancer (TNBC) comprises a heterogeneous group of diseases which are generally associated with poor prognosis. Up to now, no targeted treatment beyond anti-VEGF therapy has been approved for TNBC and cytotoxic agents remain the mainstay of treatment. Ixazomib is a selective and reversible inhibitor of the proteasome, which has been mainly investigated in the treatment of multiple myeloma. In a preclinical study TNBC cells were treated with the first-generation proteasome inhibitor bortezomib in combination with cisplatin and synergistic efficacy was demonstrat