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Medicina (Kaunas, Lithuania) May 2024Spontaneous remissions (SRs) in blastic plasmacytoid dendritic cell neoplasms (BPDCNs) are infrequent, poorly documented, and transient. We report a 40-year-old man...
Spontaneous remissions (SRs) in blastic plasmacytoid dendritic cell neoplasms (BPDCNs) are infrequent, poorly documented, and transient. We report a 40-year-old man presenting with bycitopenia and soft tissue infection. The bone marrow exhibited 3% abnormal cells. Immunophenotyping of these cells revealed the antigens CD45+ (dim), CD34+, CD117+, CD123+ (bright), HLA-DR+ (bimodal), CD56+ (bright), CD33+, CD13+, CD2+, and CD22+ (dim) and the partial expression of the CD10+, CD36+, and CD7+ antigens. All other myeloid, monocytic, and lymphoid antigens were negative. Genetic studies showed a complex karyotype and mutations in the TP53 and KRAS genes. On hospital admission, the patient showed a subcutaneous nodule on the right hand and left lower limb. Flow cytometry multiparameter (FCM) analysis showed the presence of 29% abnormal cells with the previously described immunophenotype. The patient was diagnosed with BPDCN. The patient was treated with broad-spectrum antibiotics for soft tissue infection, which delayed therapy for BPDCN. No steroids or chemotherapeutic or hypomethylating agents were administered. His blood cell counts improved and skin lesions disappeared, until the patient relapsed five months after achieving spontaneous remission. About 60% of abnormal cells were identified. No changes in immunophenotype or the results of genetic studies were observed. The patient underwent a HyperCVAD chemotherapy regimen for six cycles. Consolidation therapy was performed via allogeneic bone marrow transplantation with an HLA-unrelated donor. One year after the bone marrow transplant, the patient died due to the progression of his underlying disease, coinciding with a respiratory infection caused by SARS-CoV-2. In the available literature, SRs are often linked to infections or other stimulators of the immune system, suggesting that powerful immune activation could play a role in controlling the leukemic clone. Nevertheless, the underlying mechanism of this phenomenon is not clearly understood. We hypothesize that the immune system would force the leukemic stem cell (LSC) to undergo a state of quiescence. This loss of replication causes the LSC progeny to die off, resulting in the SR of BPDCN.
Topics: Humans; Male; Adult; Dendritic Cells; Remission, Spontaneous; Immunophenotyping; Hematologic Neoplasms
PubMed: 38792990
DOI: 10.3390/medicina60050807 -
Medicina (Kaunas, Lithuania) Apr 2024The guidelines for chronic urticaria in children contain recommendations that are often based on adult studies. The diagnostic pathway has not been standardized and the... (Observational Study)
Observational Study
The guidelines for chronic urticaria in children contain recommendations that are often based on adult studies. The diagnostic pathway has not been standardized and the effectiveness of anti-H1, omalizumab, montelukast, and systemic glucocorticoids is rarely reported in the pediatric population. There is a wide variation in the rate of remission of chronic urticaria between studies. The aim of this study is to enhance our understanding of pediatric chronic urticaria. This study enrolled 37 children with chronic urticaria aged from 0 to 18 years. Demographic parameters, medical history, clinical features, laboratory data and treatment information were collected. Children were treated with the recommended dosage of second-generation H1-antihistamines, which was increased by up to twofold. Omalizumab was added for refractory anti-H1 patients. A three-day course with systemic glucocorticoids was administered for severe exacerbations. Montelukast was administered to some children. : Wheals without angioedema were common. Chronic urticaria was spontaneous in 32 children (86.48%), inducible in 2 (5.41%), induced by a parasite in 1 and vasculitic in 2. Treatment of the potential causes of chronic urticaria was of no benefit, except for eradication of Dientamoeba fragilis. Chronic urticaria was resolved within three years in 45.9% of cases. Allergic diseases were present in nine children (24.32%) and autoimmune diseases were present in three (8.11%). All children were treated with anti-H1 at the licensed dose or at a higher dose. A partial or complete response to anti-H1 was observed in 29 (78.38%) patients. Montelukast showed no benefit. All children treated with omalizumab responded. Systemic glucocorticoids were successfully used to treat exacerbations. Our findings indicate that laboratory tests should not be routinely performed in children with chronic urticaria without clinical suspicion. However, comorbidities such as thyroid autoimmune disease and coeliac disease are suggested to be monitored over the chronic urticaria course. These clinical conditions could be diagnosed from the diagnostic framework of chronic urticaria. Increasing the dosage of anti-H1 and omalizumab was effective in children resistant to standard treatment but we still need further studies to generate a standard patient-centered treatment.
Topics: Humans; Child; Female; Male; Child, Preschool; Adolescent; Chronic Urticaria; Infant; Sulfides; Cyclopropanes; Quinolines; Acetates; Omalizumab; Histamine H1 Antagonists; Glucocorticoids; Anti-Allergic Agents; Infant, Newborn; Chronic Disease; Urticaria
PubMed: 38792886
DOI: 10.3390/medicina60050704 -
Journal of Clinical Medicine May 2024Benralizumab has been shown to restore good control of severe eosinophilic asthma (SEA). Robust data on benralizumab effectiveness over periods longer than 2 years are...
Benralizumab has been shown to restore good control of severe eosinophilic asthma (SEA). Robust data on benralizumab effectiveness over periods longer than 2 years are scarce. This retrospective multicentric study was conducted on 108 Italian SEA patients treated with benralizumab for up to 36 months. Partial and complete clinical remission (CR) were assessed. Data were analyzed with descriptive statistics or using linear, logistic, and negative binomial mixed-effect regression models. At 36 months, benralizumab reduced the exacerbation rate by 89% and increased the forced expiratory volume in 1 second (FEV) (+440 mL at 36 months, < 0.0001). Benralizumab improved asthma control as well as sinonasal symptoms in patients with chronic rhinosinusitis with nasal polyposis (CRSwNP). Up to 93.33% of patients either reduced or discontinued OCS; benralizumab also decreased ICS use and other asthma medications. Overall, 84.31% of patients achieved partial or complete CR. Benralizumab improved asthma and sinonasal outcomes up to 36 months. These findings support the potential of benralizumab to induce CR, emphasizing its role as a disease-modifying anti-asthmatic drug for the management of SEA. Further research is warranted to expand these findings by minimizing data loss and assessing benralizumab's long-term safety.
PubMed: 38792553
DOI: 10.3390/jcm13103013 -
Biomedicines Apr 2024In its severe form, where possible, asthma is treated using biological drugs in order to reduce, as much as possible, the use of systemic steroids. Mepolizumab is...
BACKGROUND
In its severe form, where possible, asthma is treated using biological drugs in order to reduce, as much as possible, the use of systemic steroids. Mepolizumab is effective for severe asthma based on key outcomes such as exacerbation and steroid dependence. Its efficacy in terms of the criteria for clinical remission in the short and long term has become of interest.
OBJECTIVE
We aimed to evaluate the effect of mepolizumab in the achievement of clinical remission after 3 years of administration.
METHODS
In this study, 71 patients who continued mepolizumab for 3 years were assessed for clinical remission according to six different published sets of remission criteria.
RESULTS
According to the criteria, 39-52% of patients experienced complete remission in the first year, increasing to 51-73% at 3 years. By classifying patients according to partial and complete remission criteria, proposed by the SANI, we observe 22% of patients in partial remission at one year, achieving complete remission after three years. The baseline factors associated with earlier remission were a higher FEV1, if we consider classifications requiring an FEV1 ≥ 80%, a low OCS dose, and low FeNO levels, in the patients requiring FEV1 stabilization.
CONCLUSIONS
Clinical remission is possible for patients treated with mepolizumab. The observations at three years compared with the first year indicated that the factors negatively affecting remission delayed rather than prevented it. Earlier treatment could increase the chances of remission.
PubMed: 38790922
DOI: 10.3390/biomedicines12050960 -
Brain Sciences Apr 2024Major depressive disorder (MDD) is frequently chronic and relapsing. The use of maintenance or continuation transcranial magnetic stimulation (TMS) has received clinical...
BACKGROUND
Major depressive disorder (MDD) is frequently chronic and relapsing. The use of maintenance or continuation transcranial magnetic stimulation (TMS) has received clinical and some research support.
OBJECTIVE
To conduct a case series study to report the outcomes of once-weekly (OW) or once-fortnightly (OF) continuation TMS in a real-life setting.
METHODS
We offered OW or OF TMS sessions to patients with MDD in remission or partial remission/relapse.
RESULTS
Ten patients received OW TMS and four received OF TMS, for 8 to 46 weeks. No patients in either group who were in remission or partial remission at baseline experienced a relapse. Improvements in HAMD6 and CGI-S scores were statistically significant or of borderline significance for the total sample and the OW group.
CONCLUSIONS
This naturalistic, open-label observational study indicates that OW TMS is effective as maintenance therapy in MDD, while also offering some support for OF TMS maintenance in preventing relapse.
PubMed: 38790394
DOI: 10.3390/brainsci14050415 -
Diabetology & Metabolic Syndrome May 2024Mesenchymal stem cell infusion and vitamin D supplementation may have immunomodulatory actions that could prolong the preservation of residual insulin secretion in...
Evaluation of type 1 diabetes' partial clinical remission after three years of heterologous adipose tissue derived stromal/stem cells transplantation associated with vitamin D supplementation.
BACKGROUND
Mesenchymal stem cell infusion and vitamin D supplementation may have immunomodulatory actions that could prolong the preservation of residual insulin secretion in patients with type 1 diabetes (T1D). Intervention with these agents after onset of T1D could favor the development of a remission phase, with potential clinical impact. We aimed to compare the presence of clinical remission (CR), glycemic control and daily insulin requirement at 6, 12, 18, 24 and 36 months after the diagnosis of T1D using IDAA1c in patients who received therapy with adipose tissue-derived mesenchymal stem cell (ASC) infusion and vitamin D supplementation and a control group.
METHODS
This retrospective cohort study analyzed data from the medical records of patients with T1D diagnosed between 15 and 40 years. Partial CR was defined as an IDAA1c index < 9. Patients in the intervention group received an infusion of adipose tissued-derived mesenchymal stem cells (ASCs) within 3 months after diagnosis and supplementation with 2000 IU of cholecalciferol for 1 year, started on the day following the infusion. Partial CR was also determined using the ISPAD criteria, to assess its agreement with IDAA1c.
RESULTS
A total of 28 patients were evaluated: 7 in the intervention group (group 1) and 21 in the control group (group 2). All patients in group 1 evolved with partial CR while only 46.7% of patients in group 2 had this outcome. Group 1 had a higher frequency of CR when evaluated with IDAA1c and ISPAD criteria. The mean duration of CR varied between the two criteria. Although HbA1c was similar between groups during follow-up, group 1 had a lower total daily insulin requirement (p < 0.005) at all time points. At 36 months, group 1 used 49% of the total daily insulin dose used by group 2 with similar glycemic control.
CONCLUSION
The intervention with infusion of ASC + vitamin D supplementation was associated with partial CR at 6 months. Although there were no differences in CR established by the IDAA1c and ISPAD criteria after three years of follow-up, patients who underwent intervention had nearly the half insulin requirement of controls with conventional treatment, with similar glycemic control.
TRIAL REGISTRATION
37001514.0.0000.5257.
PubMed: 38790009
DOI: 10.1186/s13098-024-01302-2 -
Medicine May 2024Inflammatory myofibroblastic tumor (IMT) is a rare invasive soft tissue tumor. Many IMTs are positive for anaplastic lymphoma kinase (ALK) with ALK gene fusion; other...
INTRODUCTION
Inflammatory myofibroblastic tumor (IMT) is a rare invasive soft tissue tumor. Many IMTs are positive for anaplastic lymphoma kinase (ALK) with ALK gene fusion; other gene mutations have also been reported, which indicates a key role for genetic testing and the development of target therapy to optimize treatment strategies.
PATIENT CONCERNS
We report 2 patients who obtained clinical benefits following targeted treatment with ensartinib.
DIAGNOSIS
The first patient was diagnosed as IMT, with TFG-ROS1 fusion gene mutation. The second patient was IMT harboring the ALK-STRN fusion gene mutation.
INTERVENTIONS
We performed gene testing for these 2 patients. According to the test result, both patients received ensartinib 225 mg QD as targeted therapy for a 30-day cycle.
OUTCOMES
The first patient achieved partial remission and maintained a stable state for 14.7 months. The second patient was treated for 10 months and reached complete remission after 5 months and is currently still benefiting from treatment. Treatment-related side effects were mild in both patients.
CONCLUSION
Our cases provided some new insights and approaches for the clinical diagnosis and treatment of IMT.
Topics: Humans; Female; Male; Neoplasms, Muscle Tissue; Adult; Anaplastic Lymphoma Kinase; Middle Aged; Soft Tissue Neoplasms; Antineoplastic Agents
PubMed: 38787978
DOI: 10.1097/MD.0000000000038136 -
Cureus Apr 2024Cranial nerve palsy is common in pituitary disease and depends on the extension of the lesion into the cavernous sinuses. Bilateral cranial nerve palsy was described...
Cranial nerve palsy is common in pituitary disease and depends on the extension of the lesion into the cavernous sinuses. Bilateral cranial nerve palsy was described in pituitary adenomas with apoplexy and in only one case in hypophysitis. We present a case of a 32-year-old female manifesting with headache, diplopia, bilateral sixth nerve palsy, and hypopituitarism. Magnetic resonance imaging (MRI) revealed symmetric expansion of the pituitary gland, with bilateral cavernous sinus invasion and thickening of the pituitary stalk. Hypophysitis was suspected, and after treatment with IV methylprednisolone boluses, a decrease in the pituitary lesion was observed, with complete remission of sixth nerve palsy in the right eye and partial improvement in the left eye. In this case, we report an infrequent form of presentation of hypophysitis, and highlight that steroids are the first line of treatment.
PubMed: 38784347
DOI: 10.7759/cureus.58850 -
Arquivos de Gastroenterologia 2024Spontaneous regression (SR) is defined as the partial or complete disappearance of a tumor, in the absence of a specific treatment. Evidence of the SR in hepatocellular...
BACKGROUND
Spontaneous regression (SR) is defined as the partial or complete disappearance of a tumor, in the absence of a specific treatment. Evidence of the SR in hepatocellular carcinoma (HCC) is rare.
OBJECTIVE
The authors aimed to review all the cases of SR of HCC in two reference centers of Southern Brazil, highlighting the main characteristics.
METHODS
Data of all patients with HCC were retrospectively reviewed looking for the occurrence of SR in patients from two tertiary centers in Southern Brazil, in the last five years. The diagnosis of cirrhosis was established according to clinical, laboratory and imaging data, as well as upper endoscopy or histopathological examination when necessary. The diagnosis of HCC was based on typical findings according to radiologic criteria (LIRADS) or histopathological examination. Spontaneous regression was defined as a partial or complete involution of a HCC in the absence of a specific therapy.
RESULTS
From all cases of HCC in the last 5 years (n=433), there were five cases of SR. Three (60%) were men, the mean age was 62.6 (50.0-76.0) years, and the etiology was HCV in 3 (60%). Complete regression was observed in three patients (60%), one patient (20%) presented partial regression, and one (20%) relapesed and died. The time of follow-up varied between 12 and 21 months. In this presentation, it was highlighted one case of SR observed after COVID-19 infection in a patient with cirrhosis. The possible mechanisms involved in this situation were reviewed, emphasizing the most common like hypoxia and immunological. There were also one patient submitted to a surgical procedure as a possible fator involved and three patients without obvious risk factors.
CONCLUSION
This phenomenon will possibly contribute to a better understanding of the pathophysiological mechanisms of HCC.
Topics: Humans; Carcinoma, Hepatocellular; Liver Neoplasms; Male; Female; Middle Aged; Retrospective Studies; Risk Factors; Aged; Neoplasm Regression, Spontaneous; Liver Cirrhosis; Remission, Spontaneous; Brazil
PubMed: 38775585
DOI: 10.1590/S0004-2803.24612023-151 -
Annals of Palliative Medicine May 2024End-of-life (EOL) care is the part of palliative care intended for persons nearing death. In anorexia nervosa (AN), providing EOL care instead of coercing... (Review)
Review
BACKGROUND
End-of-life (EOL) care is the part of palliative care intended for persons nearing death. In anorexia nervosa (AN), providing EOL care instead of coercing life-sustaining measures is controversial. The existing literature has not been synthesized yet. To clearly delineate differing views and identify open questions as well as areas of possible consensus, we conducted the first-ever synthesis of the existing literature.
METHODS
We searched EMBASE, PubMed, PsycInfo, and Web of Science for scientific publications on forgoing coerced life-sustaining measures and/or providing EOL care for persons with AN who refuse life-sustaining measures, typically artificial nutrition. Palliative care outside of the EOL context and medical assistance in dying were not reviewed. As very little quantitative studies were identified, we qualitatively analyzed conceptual questions, ethical reasoning, legal aspects, stakeholder attitudes, practical aspects, stakeholder needs, and outcome.
RESULTS
We identified 117 eligible publications from 1984 to 2023, mainly case reports (n=26 different cases) and ethical analyses. Conceptualizations of key terms such as terminality, futility, and decision-making capacity (DMC) in AN varied widely and were often value-laden and circular. Ethical reasoning centered on weighing the preservation of life versus quality of life in the context of uncertainty about DMC and likelihood of clinical remission. Studies on stakeholder attitudes reflected this challenge. In some cases, courts ruled against coerced life-sustaining measures and/or in favor of EOL care for persons with AN. While eligibility criteria were contested, recommendations for deliberating about and providing EOL care were consistent. We identified only one study on stakeholder needs and none on outcome. Case reports described quality of life under EOL care as good and death as the most frequent outcome but engagement in voluntary treatment and (partial) clinical remission in some.
CONCLUSIONS
The debate around EOL care in AN needs consented, coherent terminology whose value base is reduced to a minimum and made transparent. While more empirical research into decision-making in AN and predictors of outcome might help reduce uncertainty, fundamental normative questions need to be addressed, for example regarding the ethico-legal significance of treatment refusals, the weighing of quantity versus quality of life and the appropriateness of diagnosis-based ethico-legal exceptionalism such as hard paternalism. More research is needed on outcome of and stakeholder needs in EOL care for persons with AN.
Topics: Adult; Female; Humans; Anorexia Nervosa; Palliative Care; Quality of Life; Terminal Care
PubMed: 38769800
DOI: 10.21037/apm-23-522