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Hemic and Lymphatic Diseases - Top 30 Publications

Gene Therapy in a Patient with Sickle Cell Disease.

Gene Therapy in a Patient with Sickle Cell Disease.

Gene Therapy in a Patient with Sickle Cell Disease.

Mepolizumab or Placebo for Eosinophilic Granulomatosis with Polyangiitis.

Eosinophilic granulomatosis with polyangiitis is an eosinophilic vasculitis. Mepolizumab, an anti-interleukin-5 monoclonal antibody, reduces blood eosinophil counts and may have value in the treatment of eosinophilic granulomatosis with polyangiitis.

Tumor Lysis Syndrome: A Unique Solute Disturbance.

Tumor lysis syndrome (TLS) is a life-threatening disorder that is an oncologic emergency. Risk factors for TLS are well-known, but the current literature shows case descriptions of unexpected acute TLS. Solid tumors and untreated hematologic tumors can lyse under various circumstances in children and adults. International guidelines and recommendations, including the early involvement of the critical care team, have been put forward to help clinicians properly manage the syndrome. Advanced practice nurses may be in the position of triaging and initiating treatment of patients with TLS, and need a thorough understanding of the syndrome and its treatment.

Crizanlizumab in Sickle Cell Disease.

Crizanlizumab in Sickle Cell Disease.

Managing Chemotherapy-Induced Anemia with Erythropoiesis-Stimulating Agents Plus Iron.

Editor's note: This is a summary of a nursing care-related systematic review from the Cochrane Library.

Stabilizing the Mixed Lineage Leukemia Protein.

Eosinophilic esophagitis: unclear roles of IgE and eosinophils.

Eosinophilic esophagitis (EoE) is a chronic inflammatory disease of the oesophagus. Recognized as a distinct entity only two decades ago, the emergence of the disease along with the availability of new technologies has rapidly opened new research avenues and outlined the main features of the pathogenesis of EoE. Yet, each advance in our understanding of the disease has raised new questions about the previous consensus. Currently, new subsets of the disease challenge our diagnostic criteria. For instance, it was believed that EoE did not respond to proton pump inhibitor (PPI) therapy; however, it has now been shown that a substantial proportion of EoE patients indeed respond to PPIs. In addition, a new subset of patients not even presenting eosinophil infiltrates in the oesophagus has also been described. Moreover, approaches for better understanding the heritability of the disease bring into question the dogma of predominant genetic involvement. Furthermore, the specificity and sensitivity of allergy testing for targeted food avoidance is highly controversial, and the production of specific antibodies in EoE now includes IgG4 in addition to IgE. In conclusion, EoE is perceived as 'a moving target' and the aim of this review was to summarize the current understanding of EoE pathogenesis.

SerpinC1/Antithrombin III in kidney-related diseases.

The gene SerpinC1 encodes a serine protease inhibitor named antithrombin III (ATIII). This protease demonstrates both anticoagulant and anti-inflammatory action. ATIII is the most important coagulation factor inhibitor, and even minor changes in ATIII can significantly alter the risk of thromboembolism. ATIII can also suppress inflammation via a coagulation-dependent or -independent effect. Moreover, apart from ATIII deficiency, ATIII and its gene SerpinC1 may also be related to many diseases (e.g. hypertension, kidney diseases). The present review summarizes how ATIII affects the progress of kidney disease and its mechanism. Further studies are required to investigate how ATIII affects renal function and the treatment.

Thromboelastography Utilization in Delayed Recurrent Coagulopathy after Severe Eastern Diamondback Rattlesnake Envenomation.

Venomous snakebites are fairly common in the United States and can present with a wide range of symptoms. A 48-year-old man presented after Eastern Diamondback rattlesnake envenomation. His hospital course was complicated by right leg compartment syndrome and delayed recurrent coagulopathy, requiring multiple doses of Crotalidae Polyvalent Immune Fab (CroFab) antivenom and transfusions. Thromboelastography was used as an adjunct to standard coagulation studies in monitoring his delayed recurrent coagulopathy.

Eltrombopag Added to Standard Immunosuppression for Aplastic Anemia.

Acquired aplastic anemia results from immune-mediated destruction of bone marrow. Immunosuppressive therapies are effective, but reduced numbers of residual stem cells may limit their efficacy. In patients with aplastic anemia that was refractory to immunosuppression, eltrombopag, a synthetic thrombopoietin-receptor agonist, led to clinically significant increases in blood counts in almost half the patients. We combined standard immunosuppressive therapy with eltrombopag in previously untreated patients with severe aplastic anemia.

Sickle Cell Disease.

Mortality is associated with inflammation, anemia, specific diseases and treatments, and molecular markers.

Lifespan is a complex trait, and longitudinal data for humans are naturally scarce. We report the results of Cox regression and Pearson correlation analyses using data of the Study of Health in Pomerania (SHIP), with mortality data of 1518 participants (113 of which died), over a time span of more than 10 years. We found that in the Cox regression model based on the Bayesian information criterion, apart from chronological age of the participant, six baseline variables were considerably associated with higher mortality rates: smoking, mean attachment loss (i.e. loss of tooth supporting tissue), fibrinogen concentration, albumin/creatinine ratio, treated gastritis, and medication during the last 7 days. Except for smoking, the causative contribution of these variables to mortality was deemed inconclusive. In turn, four variables were found to be associated with decreased mortality rates: treatment of benign prostatic hypertrophy, treatment of dyslipidemia, IGF-1 and being female. Here, being female was an undisputed causative variable, the causal role of IFG-1 was deemed inconclusive, and the treatment effects were deemed protective to the degree that treated subjects feature better survival than respective controls. Using Cox modeling based on the Akaike information criterion, diabetes, mean corpuscular hemoglobin concentration, red blood cell count and serum calcium were also associated with mortality. The latter two, together with albumin and fibrinogen, aligned with an"integrated albunemia" model of aging proposed recently.

The lack of BTK does not impair monocytes and polymorphonuclear cells functions in X-linked agammaglobulinemia under treatment with intravenous immunoglobulin replacement.

The lack of BTK in X-linked agammaglobulinemia (XLA) patients does not affect monocytes and polymorphonuclear cells (PMN) phenotype and functions. In this study, we show that XLA patients had an increased frequency of the intermediate monocytes subset and that BTK-deficient monocytes and PMN had a normal expression of receptors involved in the activation and cellular responses. We demonstrate that BTK is not required for migration, phagocytosis and the production of reactive oxygen species (ROS) following engagement of FC gamma receptors (FcγR). XLA monocytes and PMN showed an efficient calcium (Ca2+)-independent activation of oxidative burst, suggesting that oxidative burst is less dependent by Ca2+ mobilization. The phagocytosis was functional and it remained unaltered also after Ca2+ chelation, confirming the independence of phagocytosis on Ca2+ mobilization. Intravenous immunoglobulin (IVIg) infusion exerted an anti-inflammatory effect by reducing the frequency of pro-inflammatory monocytes. In monocytes, the IVIg reduce the oxidative burst and phagocytosis even if these functions remained efficient.

Defining the complex phenotype of severe systemic loxoscelism using a large electronic health record cohort.

Systemic loxoscelism is a rare illness resulting from the bite of the recluse spider and, in its most severe form, can lead to widespread hemolysis, coagulopathy, and death. We aim to describe the clinical features and outcomes of the largest known cohort of individuals with moderate to severe loxoscelism.

Effects of Jianpi Qingchang decoction on the quality of life of patients with ulcerative colitis: A randomized controlled trial.

This study aims to determine the effects of the Jianpi Qingchang decoction (JQD) on the quality of life (QOL) of patients with spleen deficiency and dampness-heat syndrome ulcerative colitis (UC).A total of 120 active UC patients with spleen deficiency and dampness-heat syndrome were enrolled into this study. These patients were randomly divided into 2 groups: test group and control group (n = 60, each group). Patients in the test group were treated with JQD, while patients in control group were treated with 5-amino salicylic acid. After treatment for 8 weeks, differences in inflammatory bowel disease questionnaire (IBDQ) scores, short form-36 health survey questionnaire (SF-36) scores, and Sutherland Disease Activity Index (DAI) values were compared between these 2 groups to assess the QOL of patients.Sutherland DAI scores decreased in both groups after the treatment, but the difference was not statistically significant (P < .05). However, the difference in bowel symptoms, systemic symptoms, total scores of the 4 IBDQ dimensions (physical function, bodily pain, vitality, and mental health), and total scores of the SF-36 questionnaires between these 2 groups were statistically significant (P < .05).JQD can be used as supplementary and alternative therapy to relieve clinical symptoms in patients with mild to moderate active UC, and consequently improve their QOL.

Crystalglobulinemia manifesting as chronic arthralgia and acute limb ischemia: A clinical case report.

Crystalglobulinemia is a rare disease caused by monoclonal immunoglobulins, characterized by irreversible crystallization on refrigeration. It causes systemic symptoms including purpura, arthralgia, and vessel occlusive conditions to be exacerbated by exposure to cold. We report a patient with crystalglobulinemia associated with monoclonal gammopathy of undetermined significance (MGUS) manifesting as chronic arthralgia and recurrent acute arterial occlusion.

Tuberculosis-associated hemophagocytic lymphohistiocytosis with initial presentation of fever of unknown origin in a general hospital: An analysis of 8 clinical cases.

The study aimed to investigate the clinical features and prognoses of patients with tuberculosis (TB) who had secondary hemophagocytic lymphohistiocytosis (HLH).Patients first presenting with fever of unknown origin, who were ultimately diagnosed with TB-associated secondary HLH, were assessed retrospectively. We summarized and analyzed clinical manifestations, laboratory examinations, diagnoses, treatments, and prognoses of patients using clinical data, outpatient follow-up, and telephone follow-up in combination with literature review.Among patients admitted to the hospital with fever of unknown origin in the past 10 years, 371 patients were diagnosed with TB. Among them, 8 cases were diagnosed as tuberculosis-associated HLH (TB-HLH). The proportion of females among TB-HLH patients was higher than the proportion of females among TB patients. Within the same time period, 227 cases met the diagnostic criteria for HLH, among which TB-HLH patients accounted for 3.52% of the cases. None of the 8 TB-HLH patients had underlying diseases, and a majority of them had short symptom durations, rapid progression, along with multisystem and multiorgan dysfunctions. Their clinical manifestations were inconsistent with the typical clinical manifestations and imaging results characteristic of TB. Compared with patients with TB in our hospital during the same period, the 8 TB-HLH patients had a higher proportion of blood-disseminated TB and tuberculous meningitis. Apart from this, the hematological damage in these patients was higher than the common clinical manifestations of TB, and they also had a high proportion of respiratory failure. All 8 TB-HLH patients received antitubercular therapy, and 6 of them were also treated for HLH. However, their morbidity and mortality were significantly higher than that for reported cases of TB-HLH cases, both domestically and abroad, which may be attributed to the fever of unknown origin.Patients with TB-HLH had poor prognoses and no specific clinical manifestations. Therefore, cases of atypical TB and severe TB should be carefully monitored to achieve early diagnosis and early intervention.

Feasibility and safety of emergency laparoscopic partial splenectomy: A retrospective analysis.

The increased awareness of asplenia-related life-threatening complications has led to the development of parenchyma sparing splenic resections in past few years. The aim of this study is to retrospectively analyze the feasibility and safety of laparoscopic partial splenectomy (LPS) in selected emergency patients.From January 2013 to December 2015, there were 46 emergency patients, diagnosed with splenic rupture, admitted in our department. Selection criteria for LPS: (1) Preoperative CT scan revealed single pole rupture without spleen pedicle injury; (2) BP>90/60 mm Hg and heart rates <120 bpm; (3) No sigh of multiple organ injury. Eventually, LPS was performed in 21 patients (Group LPS), while laparoscopic splenectomy (LS) was performed in 20 patients (Group LS).The main cause of splenic rupture was traffic accident, followed by blunt injury and high falling injury. Abdominal CT scan showed the mean longitudinal diameter of spleen of group LPS was 14.2 ± 1.8 cm (range 12-17 cm), while the size of remnant spleen was 5.5 ± 1.2 cm. Between 2 groups, operation time (LPS: 122.6 ± 17.2 min vs LS: 110.5 ± 18.7 minutes, P = .117), and intraoperative blood loss (LPS: 174 ± 22 mL vs LS: 169 ± 29 mL, P = .331) were similar. There were 2 patients suffered subsequent unstable vital sign altering during mobilization when performing LPS. Conversion to LS (2/21, 9.52%) was decided and successfully completed. Although there was no patient suffered postoperative OPSI or thrombocytosis events in both groups after 6-month follow-up, the mean platelets and leukocyte count were significantly lower in group LPS. Splenic regrowth was evaluated in 20 patients of group LPS. And the mean regrowth of splenic volume reached 19% (10%-26%).Due to its minimal invasive effect and functional splenic tissue preservation, LPS may be a safe and feasible approach for emergency patients. And prospective trials with clear inclusion criteria are needed to proof the benefit of LPS.

Interdigitating dendritic cell sarcoma presenting in the sigmoid colon mesentery: A case report and literature review.

Interdigitating dendritic cell sarcoma (IDCS) is an extremely rare disease. It commonly occurs in middle-aged males and mainly involves the lymph nodes. Pathological examination plays an important role in differentiating from other tumors, but far less published literature focuses on the imaging characteristics of IDCS.

Growth characteristics and endocrine abnormalities in 22q11.2 deletion syndrome.

22q11.2 deletion syndrome (22q11.2DS) has a wide range of clinical features including endocrine abnormalities. We aimed to characterize growth patterns, hypoparathyroidism, and thyroid dysfunction of individuals with 22q11.2DS. Anthropometric and laboratory measurements were obtained from the charts of 48 individuals (males=28, 8.0±6.8 visits/participant) followed at a national 22q11.2DS clinic between 2009 and 2016. Age at diagnosis was 4.3±4.9 years and age at last evaluation 11.2±7.2 years. Median height-SDS was negative at all ages. Height-SDS at last visit was correlated to the midparental height-SDS (r=0.52 P=0.002). Yet, participants did not reach their target height, with a difference of 1.06±1.07 SD (P <0.0001). Height-SDS at last visit of participants with a heart defect was lower compared to participants with a normal heart (-1.5±1.4 vs. -0.6±0.8, P=0.036), with lower height-SDS in the subgroup of participants with severe heart defects (-2.1±1.6, P=0.009). Mean IGF1-SDS was low (-0.99±1.68) but was not correlated with height-SDS. Thirteen patients (27%) had hypoparathyroidism: 10 presented during infancy and 3 during adolescence. Five patients (10.4%, female=4) had thyroid abnormalities. In conclusions, individuals with 22q11.2 DS have a distinct growth pattern consisting of growth restriction at all ages, resulting in final adult height in the low-normal range. Hypoparathyroidism is common and may present during the neonatal period as well as later in life. Thyroid abnormalities may present during childhood, adolescence, or adulthood.

Reslizumab in Eosinophilic Asthma: A Review.

Reslizumab (Cinqaero(®); Cinqair(®)) is a humanized monoclonal antibody against interleukin-5 (IL-5), a cytokine mediator of eosinophilic airway inflammation. Reslizumab is indicated as an add-on treatment for severe eosinophilic asthma in adults, on the basis of data from the BREATH phase III clinical trial programme. In three double-blind BREATH studies of up to 52 weeks' duration, adding intravenous reslizumab (3 mg/kg, once every 4 weeks) to the current asthma therapy of patients (aged 12-75 years) with eosinophilic asthma inadequately controlled with inhaled corticosteroids resulted in significant reductions in clinical asthma exacerbation frequency and significant improvements in lung function, asthma control and health-related quality of life relative to adding placebo. Pooled data from the two trials of 52 weeks' duration indicated similar benefits with reslizumab across various patient subgroups, including patients with severe eosinophilic asthma. Reslizumab was generally well tolerated, with very few recipients experiencing severe or serious treatment-related adverse events. Moreover, in an open-label extension study, continued use of reslizumab for up to 2 years was associated with durable lung function benefit, without any new tolerability concerns. Thus, intravenous reslizumab extends the valuable add-on treatment options for adults with severe eosinophilic asthma inadequately controlled with standard therapies.

Anti-MOG antibody-positive ADEM following infectious mononucleosis due to a primary EBV infection: a case report.

Anti-Myelin oligodendrocyte glycoprotein (MOG) antibodies are detected in various demyelinating diseases, such as pediatric acute disseminated encephalomyelitis (ADEM), recurrent optic neuritis, and aquaporin-4 antibody-seronegative neuromyelitis optica spectrum disorder. We present a patient who developed anti-MOG antibody-positive ADEM following infectious mononucleosis (IM) due to Epstein-Barr virus (EBV) infection.

Current Role of FDG-PET in Pediatric Hodgkin's Lymphoma.

Hodgkin's lymphoma is one of the most curable pediatric cancers with long-term survival rates exceeding 90% following intensive treatment. Collaborative group studies worldwide aim on reduction or elimination of radiotherapy to avoid potentially life-limiting late effects especially second cancers and cardiovascular diseases. Large prospective trials have integrated early response FDG-PET scans to identify adequate responders to chemotherapy in whom radiotherapy may safely be omitted. The criteria for interpretation of early response PET have changed during the past years and will be further refined based on trial results. FDG-PET is also systematically used to assess initial disease involvement of pediatric Hodgkin's lymphoma and could replace bone marrow biopsy. This article summarizes the role of FDG-PET in staging and response assessment focusing on large pediatric trials, the criteria for PET interpretation and pitfalls.

The treatment of primary mediastinal large B-cell lymphoma: a two decades monocentric experience with 98 patients.

The purpose of this study is to investigate the most suitable first-line approach and the best combination treatment for primary mediastinal large B-cell lymphoma (PMLBCL) as they have been matter of debate for at least two decades.

Characterization of hydroxypropyl-beta-cyclodextrins used in the treatment of Niemann-Pick Disease type C1.

2-Hydroxypropyl-beta-cyclodextrin (HPβCD) has gained recent attention as a potential therapeutic intervention in the treatment of the rare autosomal-recessive, neurodegenerative lysosomal storage disorder Niemann-Pick Disease Type C1 (NPC1). Notably, HPβCD formulations are not comprised of a single molecular species, but instead are complex mixtures of species with differing degrees of hydroxypropylation of the cyclodextrin ring. The degree of substitution is a critical aspect of the complex mixture as it influences binding to other molecules and thus could potentially modulate biological effects. VTS-270 (Kleptose HPB) and Trappsol® Cyclo™ are HPβCD products under investigation as novel treatments for NPC1. The purpose of the present work is to compare these two different products; analyses were based on ion distribution and abundance profiles using mass spectrometry methodology as a means for assessing key molecular distinctions between products. The method incorporated electrospray ionization and analysis with a linear low-field ion mobility quadrupole time-of-flight instrument. We observed that the number of hydroxypropyl groups (the degrees of substitution) are substantially different between the two products and greater in Trappsol Cyclo than in VTS-270. The principal ions of both samples are ammonium adducts. Isotope clusters for each of the major ions show doubly charged homodimers of the ammonium adducts. In addition, both products show doubly charged homodimers from adduction of both a proton and ammonium. Doubly charged heterodimers are also present, but are more intense in Trappsol Cyclo than in VTS-270. Based on the analytical differences observed between VTS-270 and Trappsol Cyclo with respect to the degree of substitution, the composition and fingerprint of the complex mixture, and the impurity profiles, these products cannot be considered to be the same; the potential biological and clinical implications of these differences are not presently known.

Antigen Discovery and Therapeutic Targeting in Hematologic Malignancies.

Historically, immune-based therapies have played a leading role in the treatment of hematologic malignancies, with the efficacy of stem cell transplantation largely attributable to donor immunity against malignant cells. As new and more targeted immunotherapies have developed, their role in the treatment of hematologic malignancies is evolving and expanding. Herein, we discuss approaches for antigen discovery and review known and novel tumor antigens in hematologic malignancies. We further explore the role of established and investigational immunotherapies in hematologic malignancies, with a focus on personalization of treatment modalities such as cancer vaccines and adoptive cell therapy. Finally, we identify areas of active investigation and development. Immunotherapy is at an exciting crossroads for the treatment of hematologic malignancies, with further investigation aimed at producing effective, targeted immune therapies that maximize antitumor effects while minimizing toxicity.

Spotlight on romiplostim in the treatment of children with chronic immune thrombocytopenia: design, development, and potential place in therapy.

Primary immune thrombocytopenia (ITP) is an autoimmune disorder characterized by isolated thrombocytopenia. In approximately one-third of cases, the duration of thrombocytopenia will extend beyond 12 months consistent with a diagnosis of chronic ITP. Minor bleeding manifestations are common in chronic ITP while severe or life-threatening bleeding complications are uncommon. Moreover, spontaneous resolution occurs in the majority of children with chronic ITP necessitating treatment in only those children with ongoing bleeding manifestations or impairment in health-related quality of life (HRQOL). The characterization of thrombopoietin (TPO) and remarkable advancements in our understanding of the pathophysiology of ITP has led to the development of a new class of agents, the TPO-receptor agonists that have documented efficacy in the amelioration of thrombocytopenia and bleeding manifestations in chronic ITP. Romiplostim is a second-generation TPO-receptor agonist that has undergone limited evaluation in the treatment of chronic ITP in children. Evolving data suggest that romiplostim may be a safe and effective agent in the treatment of chronic ITP in children. Additional data are needed to confirm its ability to increase platelet counts, decrease bleeding manifestation, and improve the HRQOL of children and caregivers impacted by chronic ITP.