Replacement Therapy In Patients Research Articles

Current Practice and Perspectives on Subcutaneous Immunoglobulin Replacement Therapy in Patients with Primary Antibody Deficiency Among Specialized Nurses in Poland

Background/Objectives: Inborn errors of immunity (IEI) encompass various congenital disorders, resulting in immunity defects and recurrent infections. Home-based subcutaneous immunoglobulin replacement therapy (scIgRT) is the best treatment option for those with primary antibody deficiency (PAD). However, the lack of standardized procedures in patient training remains a challenge. Our study investigates nurses’ practice and perspectives, aiming to identify areas for improvement in at-home scIgRT practice. Methods: We prepared a structured survey regarding scIgRT, including needle choice experience and perception of adverse events, and distributed it among qualified nurses involved in patient training and scIgRT supervising. Results: We included 56 nurses with a median age of 50 years. Among them, 67.9% represented adult care providers, while 32.1% supervised IgRT in children. Most respondents (83.9%) used the classic or assisted with hyaluronidase scIgRT preparations. Single-channel needles were administered most commonly (85.7%). The needle length was mostly chosen solely by a nurse (57.1%) or in cooperation with the patient (23.2%). Next, 9 mm and 12 mm needles were used most often (92.9% and 78.6%, respectively). As expected, the 6 mm needle was more frequently applied for children compared to adults (n = 16, 88.9% vs. n = 11, 28.9%, p < 0.001), while 12 mm was primarily used in adults (n = 35, 92.1% vs. n = 9, 50.0%, p < 0.001). Visual skin fold assessment was the basis for the needle selection (58.9%), followed by the injection site rule (26.8%) or a choice between two available needle types for thinner or thicker patients (25.0%). Results of this survey indicate that, according to nurses’ opinions presented in this survey, the needle length could be associated with local scIgRT adverse events, such as side needle leakage or local burning. Yet, it was likely unrelated to general adverse signs, such as headaches or dizziness. Most respondents (66.1%) indicated that, even if local adverse events occur, patients are reluctant to change scIgRT preparation or needle length. Most participants (69.6%) reported that the optimal administration technique needs to be discussed with the patient before and during scIgRT. Conclusions: This study sheds light on scIgRT practice in Poland, emphasizing deficiency in needle selection technique. Future research should focus on standardized training and advanced needle selection procedures on patient outcomes, investigating the correlation between needle strategies and adverse events, as well as the effectiveness of scIgRT.

Switching to extended half-life factor VIII concentrates or non-factor replacement therapies in patients with severe-to-moderate haemophilia A without inhibition: retrospective analysis. Experience of the Galician Thrombosis and Hemostasis Working Group (G2TH-AGHH)

Switching to extended half-life factor VIII concentrates or non-factor replacement therapies in patients with severe-to-moderate haemophilia A without inhibition: retrospective analysis. Experience of the Galician Thrombosis and Hemostasis Working Group (G2TH-AGHH)

Effectiveness of tolvaptan on renal replacement therapy in patients with autosomal dominant polycystic kidney disease: a retrospective cohort study from the TriNetX global collaborative network

Background and hypothesis Autosomal Dominant Polycystic Kidney Disease (ADPKD) is a major genetic contributor to end-stage kidney disease (ESKD). Current evidence on tolvaptan primarily focuses on slowing estimated glomerular filtration rate (eGFR) decline and kidney volume growth. However, direct confirmation of its effectiveness in reducing the need for hemodialysis in ESKD remains limited. Methods We included ADPKD patients aged ≥18 years using TriNetx data from Sep 2, 2018, to Sep 3, 2023. Propensity score matching (PSM) ensured baseline comparability (standardized mean difference (SMD) <0.1). Hazard ratios (HRs) with 95% confidence intervals (CIs) evaluated outcomes, and subgroup analyses were performed. Results After 1:1 PSM, both groups comprised 673 patients. The average age was 45, with generally good health (3–5% diabetes, 2-3% ischemic heart disease). Baseline eGFR averaged ∼55 ml/min/1.732m2. Post-matching, all SMDs were <0.1, indicating successful matching. Tolvaptan users exhibited lower eGFR (51.45 ± 30.09 vs. 57.37 ± 33.65, p < 0.001) and higher risk of stage 4-CKD (HR: 2.436, 95% CI:1.649, 3.599) compared to non-users. However, tolvaptan users showed significantly reduced chances of initiating hemodialysis (HR:0.362, 95%CI:0.176, 0.745), experiencing urinary tract infections (HR:0.581, 95%CI:0.354, 0.956), and all-cause mortality (HR:0.355, 95% CI:0.180, 0.700). Kaplan-Meier curves for hemodialysis initiation indicated higher survival rates among tolvaptan users across age and number of medication refill subgroups. Conclusions This real-world study, employing precise matching, reveals tolvaptan’s role in reducing hemodialysis initiation risk in ADPKD, despite initial hemodynamic-induced lower eGFR.

Investigating Associations between Subclinical Hypothyroidism and Pregnancy Outcomes and Effects of Levothyroxine Therapy on Improving Maternal and Infant Prognosis

Background: Current evidence shows subclinical hypothyroidism (SCH) is associated with increased risk of adverse pregnancy outcomes, though some controversies exist. However, little is known on the impacts and effectiveness of levothyroxine (LT4) therapy on pregnancy outcomes in women with SCH. Present study aims to investigate the associations between SCH and adverse pregnancy outcomes and clinical effects of levothyroxine (LT4) replacement therapy in patients with SCH. Methods: The clinical data of pregnant women (n = 635) with SCH who referred to Huai'an Maternal and Child Health Care Hospital, Huaian, China from June 2018 to December 2018 were retrospectively analyzed. Among them, 147 cases received standard thyroxine replacement therapy, 292 cases did not receive treatment and 150 cases who received irregular treatment or did not achieve the target or were lost to follow-up. 46 cases whose thyroid peroxidase antibody (TPOAb) was not checked during pregnancy were not included in the study. According to the TPOAb test results patients were divided into positive treatment (n = 14), negative treatment (n = 133), positive untreated (n = 19), or negative untreated (n = 273) subgroups. A total of 1876 pregnant women with normal thyroid function (TPOAb positive = 59; TPOAb negative = 1817) who delivered during the same period were selected as the control group. Pregnancy outcomes were assessed and compared between treated and control group, untreated and control group, TPOAb positive treatment subgroup and TPOAb positive and untreated subgroup, TPOAb negative treatment subgroup and TPOAb negative subgroup, and TPOAb positive and TPOAb negative subgroup. Results: Our data showed that the incidences of hypertensive disease, premature delivery, fetal growth restriction and fetal death during pregnancy in the untreated group were significantly higher than in the control group (p &lt; 0.05). The incidence of preterm delivery in the treatment group was significantly lower compared to the untreated group and the control group (p &lt; 0.05). Moreover, the incidence of premature birth in TPOAb positive treatment subgroup was significantly lower than their peers in TPOAb positive and untreated subgroup. The incidence of premature delivery in TPOAb negative treatment subgroup was significantly lower than TPOAb negative untreated subgroup and the difference was statistically significant (p &lt; 0.05). There was no significant difference in the incidence of adverse pregnancy outcomes between TPOAb positive subgroup and TPOAb negative subgroup in the control group (p &gt; 0.05). Conclusions: SCH during pregnancy is a risk factor for hypertensive disease during pregnancy, fetal growth restriction, premature delivery and fetal death. L-T4 replacement therapy improves maternal and infant outcomes in patients with SCH during pregnancy, regardless of whether or not TPOAb is positive.

667P Glycogen response to enzyme replacement therapy in patients with Pompe disease

667P Glycogen response to enzyme replacement therapy in patients with Pompe disease

Exploring Therapeutic Effects of Continuous Kidney Replacement Therapy in Patients with Severe Acidosis Using Deep Learning-Based Causal Inference

Exploring Therapeutic Effects of Continuous Kidney Replacement Therapy in Patients with Severe Acidosis Using Deep Learning-Based Causal Inference

Effectiveness of Tolvaptan on Kidney Replacement Therapy in Patients with Autosomal Dominant Polycystic Kidney Disease: A Retrospective Cohort Study from the TriNetX Global Collaborative Network

Effectiveness of Tolvaptan on Kidney Replacement Therapy in Patients with Autosomal Dominant Polycystic Kidney Disease: A Retrospective Cohort Study from the TriNetX Global Collaborative Network

A new score predicting renal replacement therapy in patients with crush injuries: Analysis of a major earthquake

BackgroundIt is important to predict which patients may require renal replacement therapy (RRT) at the time of initial presentation after crush injuries. There is limited data in the literature examining the predictors of RRT. MethodsThis study was conducted by evaluating 2232 patients who presented to our hospital following two major earthquakes of magnitudes 7.6 and 7.7 Mw that occurred in Kahramanmaras, Turkey, on February 6, 2023. A total of 314 patients who were hospitalized upon being rescued from the rubble and had a creatine kinase (CK) level above 1000 U/L were included in the final analysis. Factors predicting the need for RRT were investigated, and a dialysis score was developed for this prediction. Resultsthe 314 patients included in the study, 95 (30.2 %) developed acute kidney injury (AKI). RRT was performed on 68 (21.6 %) patients. The optimal cut-off value of CK for the prediction of AKI was 23,000 U/L. Multivariate analysis revealed that factors predicting RRT were the number of traumatized sides (odds ratio [OR]: 2.2, 95 % confidence interval [CI]: 1.09–4.39, p = 0.026), albumin (OR:0.11, 95 % CI: 0.04–0.32, p < 0.001), and CK (OR: 1.00, 95 % CI 1.00–1.00, p < 0.001). A dialysis score was developed ranging from 0 to 7 based on the number of traumatized sides, albumin, and CK. The area under the curve (AUC) of the dialysis score in receiver operating characteristic analysis was 0.974. A dialysis score of 4 or higher had a sensitivity of 97.1 % and a specificity of 89.4 % for predicting the need for RRT. ConclusionsThe dialysis score predicts the need for RRT quite well. The simplicity of use and high sensitivity and specificity of this score in earthquake-related crush injuries will greatly facilitate clinicians in patient triage and follow-up.

Incidence of Contrast-Associated Acute Kidney Injury and Need for Renal Replacement Therapy in Patients who have Undergone Elective PCI in the Last 3 Years in a Tertiary Care Hospital

Incidence of Contrast-Associated Acute Kidney Injury and Need for Renal Replacement Therapy in Patients who have Undergone Elective PCI in the Last 3 Years in a Tertiary Care Hospital

Long-term metabolic effectiveness and safety of growth hormone replacement therapy in patients with adult growth hormone deficiency: a single-institution study in Japan.

To elucidate the long-term efficacy and safety of growth hormone replacement therapy (GHRT) in Japanese patients with adult growth hormone deficiency (AGHD). We conducted a retrospective study. A total of 110 patients with AGHD receiving GHRT were enrolled. Clinical and laboratory data were collected annually from the beginning of the study. Statistical analysis was performed using a linear mixed-effects model. Of all patients, 46.4% were males, 70.9% had adult-onset GHD, and follow-up was up to 196 months, with a median of 68 months. The insulin-like growth factor-1 standard deviation score increased after the start of GHRT and remained constant for more than 11 years. Seventeen patients were followed up for more than 11 years. The body mass index increased. Waist circumference decreased in the short term but increased in the long term. The diastolic blood pressure decreased 1-5 years after the start of GHRT, and the systolic blood pressure increased 11 years after GHRT. Moreover, a long-term decrease in low-density lipoprotein cholesterol, an increase in high-density lipoprotein cholesterol, and a decrease in aspartate aminotransferase and alanine aminotransferase levels were observed. The glycosylated hemoglobin level increased after 3 years. The bone mineral density in the lumbar spine and total hip increased significantly 3 years after the start of GHRT. Finally, the number of adverse events was eight. We demonstrated the metabolic effectiveness and safety of GHRT in Japanese patients with AGHD over a long follow-up period of 16 years.

Intracerebroventricular Enzyme Replacement Therapy in Patients with Neuropathic Form of Mucopolysaccharidosis Type II: to Help Practicing Physician

Mucopolysaccharidosis type II (Hunter syndrome, MPS II) is a rare hereditary disease from the group of hereditary metabolic diseases. There are neuropathic and non-neuropathic forms of this disease. The neuropathic form is most common and leads to severe cognitive impairment and progressive damage of central nervous system. Nowadays, early diagnosis and timely initiation of pathogenetic therapy in patients with orphan diseases is the crucial problem of modern pediatrics. Intracerebroventricular administration of idursulfase beta is one of the promising treatment options in patients with neuropathic form of MPS II as it prevents severe complications development. The study of new pathogenetic therapy methods for rare hereditary diseases will help doctors of pediatric specialties to route patient correctly in timely manner to receive all the necessary treatment.

Characteristics of initiation timing and anticoagulation of continuous renal replacement therapy in patients following cardiac surgery: A retrospective analysis of 28 patients.

Continuous renal replacement therapy (CRRT) used in cardiac surgery-associated acute kidney injury (CSA-AKI) may have different characteristics from other diseases. We reviewed the medical records of patients with CSA-AKI requiring CRRT who underwent cardiac surgery from January 2020 to September 2021. Patients with AKI caused by other reasons who received CRRT during the same period were also evaluated. A total of 28 patients with CSA-AKI and 12 patients with AKI caused by other reasons were enrolled in this study. Compared with AKI patients caused by other reasons, patients with CSA-AKI were found to have lower mean arterial pressure, higher level of bilirubin, higher vasoactive-inotropic score, and larger daily diuretic dosage. The patients with CSA-AKI were prescribed CRRT earlier than the patients with AKI caused by other reasons. There was a significant difference in the CRRT anticoagulation method between patients with CSA-AKI and patients with AKI caused by other reasons. Six patients with CSA-AKI were treated with regional citrate anticoagulation (RCA), and the other 22 patients were treated with low molecular weight heparin or without anticoagulants. The timing of CRRT initiation in patients with CSA-AKI is earlier than that in patients with AKI caused by other reasons. Although RCA is recommended as the preferred anticoagulant for patients without contraindications, patients with CSA-AKI often have circulatory dysfunction and severe liver damage, so the risk of citrate accumulation is greater, whether to use RCA should be determined according to the individual condition of the patient.

Quality of life in primary immunodeficiency: Its contribution to shared decision-making and patient outcomes.

Quality of life (QOL) measures have become increasingly important in the management of patients with complex diseases. There are a number of instruments to measure QOL that include broad areas or domains of physical, psychological, social, spiritual, and environmental issues. The number of potential domains plus the large number of items within each domain have led to the development of a large variety of QOL instruments and of different approaches by using both health-related and non-health-related factors. Health-related QOL (HRQOL) measures have been incorporated into clinical trials to assess changes in the patient's perspective on his or her disease and the effects of treatment. An important aspect of these HR-QOL instruments is that these questionnaires are patient reported and usually self-administered. The life-long therapy of intravenous immunoglobulin (IVIG) or subcutaneous immunoglobulin (SCIG) can be a challenge to the patient, his or her family and the physician. A number of patient-reported surveys have been published that examined treatment satisfaction and the comparison between IVIG and SCIG replacement therapy in patients with primary immunodeficiency. This review explores the use of HR-QOL surveys in patient preferences for mode and route of immunoglobulin therapy and site of care. Shared decision-making will be explored to emphasize the importance of this approach in optimizing patient care and compliance.

Long-term cardiovascular outcomes and mortality with enzyme replacement therapy in patients with mucopolysaccharidosis type II.

Mucopolysaccharidosis type II (MPS II) is a rare multisystemic lysosomal disorder in which cardiac issues can lead to serious dysfunction and an increased risk of fatal cardiac failure. However, studies on major adverse cardiac event (MACE) outcomes in MPS II are lacking. This study evaluated the cardiovascular outcomes and impact of enzyme replacement therapy (ERT) in patients with MPS II in South Korea. In this national cohort study, utilizing data from the National Health Insurance Database, we evaluated 127 patients with MPS II over a 14-year period to investigate the effects of ERT on MACE and all-cause mortality. We tracked MACE incidence, defined by hospitalizations for cardiovascular events, from diagnosis and adjusted the hazard ratios for MACE using Cox modeling. Over an average follow-up period of 7.3 years, we identified 16 cases of MACE among patients (17.35 per 1000 person-years; 95% confidence interval, 10.74-26.83). Patients receiving ERT exhibited a significantly lower incidence of MACE than untreated patients, with cumulative incidences showing a marked difference of 8.3 years. Notably, initiating ERT at earlier stages post-diagnosis was associated with improved outcomes, underscoring the importance of timely treatment. The key risk factors for MACE included specific arrhythmias, a history of invasive procedures, and depression. Early ERT significantly reduced MACE risk and increased survival in patients with MPS II. This underscores the importance of prompt treatment initiation and comprehensive care to address key risk factors and advocates for an expanded therapeutic strategy to enhance MPS II outcomes.

Severe respiratory acidosis contributing to refractory hyperkalemia: a case series of COVID-19 patients with renal failure

Introduction: Respiratory acidosis (RA) is not known to cause severe hyperkalemia. However, an exception to this general rule was observed in three patients with severe COVID-19 complicated with renal failure in our center. Objectives: The current study investigated the explanation for hyperkalemia refractory to renal replacement therapy in patients with severe COVID-19 with hypercarbia and renal failure in a tertiary care center. Patients and Methods: In this study, we present a case series of three patients with severe COVID-19 with hypercarbia and dialysis-requiring renal failure who developed persistent hyperkalemia refractory to renal replacement therapy. We studied various causes of persistent hyperkalemia, such as acid‒base disorder, a high turnover state, exogenous administration, rhabdomyolysis, and hypoaldosteronism. Results: All three patients initially presented with episodes of severe hyperkalemia, which were ameliorated after RA was corrected. All of these patients had worsening pulmonary function, after which the hyperkalemia could not be corrected, corresponding to persistent RA. The evidence that there was a contribution of RA to hyperkalemia was further strengthened by the observations of patient 1, in whom the hyperkalemia was only corrected after changing ventilator settings and who was unresponsive to dialysis. We studied the dialysate effluent in patient 3, and the results showed satisfactory removal of potassium with no improvement in the high serum potassium levels. Another interesting finding was that although the dialysate sodium concentration was greater than the serum sodium concentration, there was a decrease in the serum sodium concentration, indicating a reduced movement of sodium into the extracellular space. Conclusion: In our study, metabolic acidosis alone could not explain the high serum potassium levels. Moreover, hyperkalemia was corrected after RA was corrected, indicating that the latter might have been the predominant cause of hyperkalemia in these scenarios. The presence of renal failure and low serum bicarbonate levels might have been the factors contributing to decreased Na+-K+-ATPase activity in the setting of RA, which contributed to severe hyperkalemia in all three patients.

#236 Immediate or delayed initiation of renal replacement therapy in patients with leptospirosis and acute kidney injury: a target trial emulation

Abstract Background and Aims Leptospirosis is a widespread zoonosis responsible for 60,000 deaths annually worldwide [1]. Acute kidney injury (AKI) stands out as a hallmark of the disease. Leptospirosis-induced AKI (AKI-L) has been entertained as a potential cause of chronic kidney injury of unknown etiology, which is a significant burden in tropical rural settings [2]. Anecdotal evidence suggests that early renal replacement therapy (RRT) may improve mortality associated with AKI-L [3]. Conversely, several randomized controlled trials (RCTs) conducted in intensive care units have refuted the positive impact of early RRT on mortality in patients with other causes of AKI and sepsis [4]. Given these contradicting results, the optimal timing for initiating RRT in patients with leptospirosis and severe AKI remains uncertain. In this emulated RCT, we aimed to evaluate the impact of the timing of RRT on mortality and worsening renal failure (WRF) in patients hospitalized with leptospirosis, stage 3 AKI on admission, and no immediate need for RRT. Method We conducted a retrospective cohort study in two academic centers in Réunion island between 2010 and 2020. During this period, we included adult patients with documented leptospirosis and stage 2 or 3 AKI. Then we selected patients with stage 3 AKI to perform a target trial emulation, adapted from the AKIKI trial's design. Patients were categorized according to the allocated treatment strategy. Patients assigned to the “early” group received RRT within 48 hours following hospital admission while patients in the “delayed” group did not. A propensity score was derived for each imputed dataset using multivariate logistic regression. The propensity score estimates the probability of treatment allocation as a function of covariates assessed upon hospital entry. These covariates included those used in the SAPSII score and patients' baseline renal function. The primary outcome was a composite outcome assessed at one year: death or WRF. Results The cohort included 380 patients. We selected 295 patients with stage 3 AKI to perform the emulated RCT. Eight two (28%) patients were started on RRT within 48 hours of admission and were assigned to the early group. The delayed group included the remaining 213 patients, with 25% undergoing subsequent RRT and 75% not requiring such intervention. The median time to RRT initiation was 2 (1-3) days. Fifty-nine patients (20%) met the primary outcome: 14 patients (4.7%) died during their hospital stay or within 1 year after discharge, and 45 (15.3%) developed WRF. In the main analysis, the adjusted odds ratio (OR) for primary outcome occurrence was of 2.12 (95% confidence interval: 1.03 to 4.37, p = 0.04; reference group: “delayed” strategy). In secondary analyses, there was a significantly higher probability of WRF at one year in patients assigned to the “early strategy” (OR = 2.79 [1.28-6.11], p = 0.010). Mortality at one year according to RRT timing strategy did not differ (p = 0.6). Conclusion Early initiation of RRT was not found to improve the combined outcome of death and development of WRF in patients with leptospirosis and stage 3 AKI. A strategy based on a delayed initiation of RRT should be formally recommended for patients with severe AKI-L as in the general population of critically ill patients.

#59 Fibroblast Growth factor 23 for prediction of outcome in community-acquired acute kidney injury: a prospective observational study

Abstract Background and Aims Serum FGF23 level has been found to be elevated in AKI through multiple previous studies. Moreover, higher FGF23 level was associated with mortality and adverse outcomes in critically ill patients and patients undergoing cardiac surgery who developed AKI suggesting that FGF23 can have a prognostic value in AKI. The current study aims to investigate the association of serum FGF23 levels with morbidity and mortality in patients with community-acquired AKI. Method A longitudinal prospective study was carried out that included 64 patients who presented with community-acquired AKI to Mansoura Nephrology and Dialysis Unit (MNDU) at Mansoura University Hospital. Serum intact FGF23 level was measured after admission. patients were followed up during hospital stay and then at 90-day after admission. The primary outcomes were hospital and 90-day mortality and renal recovery. Results In this study, nine of the studied 64 AKI patients (14.1%) died during hospital admission and 18 out of 62 AKI patients (29%) died during the 90-day follow up period including those who died during hospital admission. Hospital mortality was significantly higher in patients with shock, sepsis, hypoalbuminemia, or high FGF23 in univariate analysis (p &amp;lt; 0.05). However, when binary logistic regression was carried out, shock was the only significant independent predictor for mortality [p value = 0.015; adjusted odds ratio = 12.465], while serum FGF23 did not independently predict in-hospital [p value = 0.589; adjusted odds ratio = 1.001] or 90-day mortality [p value = 0.055; adjusted odds ratio = 1.003] when entered in the regression equation together with the other confounders. Serum FGF23 in this study was not also associated with renal recovery, need for renal replacement, or the duration from peak serum creatinine to renal recovery (p &amp;gt; 0.05). Conclusion This research showed that serum iFGF23 could not predict mortality, renal recovery, or need for renal replacement therapy in patients with community-acquired AKI.

#2401 Association of clinical characteristics with relative changes in plasma lyso-Gb3 levels by enzyme replacement therapy in patients with Fabry disease

Abstract Background and Aims Enzyme replacement therapy (ERT) is an established treatment for Fabry disease (FD), notably for its efficacy in reducing plasma globotriaosylceramide (Gb3) levels. FD is characterized by the accumulation of Gb3 in lysosomes, resulting in the formation of globotriaosylsphingosine (lyso-Gb3), a degradation product associated with disease severity. While numerous studies have demonstrated that ERT can reduce plasma lyso-Gb3 levels, these reductions are usually quantified in absolute terms. This study is the first to explore the relative changes in plasma lyso-Gb3 levels due to ERT and to investigate the association of these changes with patients' clinical characteristics through a retrospective analysis. Method This study included patients with FD who attended the division of Nephrology at our hospital. We excluded patients lacking sufficient clinical data or without plasma lyso-Gb3 measurements both before and after initiating ERT. We considered pre-ERT plasma lyso-Gb3 concentrations as the baseline and evaluated the relative changes at one year post-ERT and at the lowest recorded lyso-Gb3 level after ERT. These variations were compared across various clinical backgrounds to assess their correlation with clinical factors including age, gender, type of ERT medication (agalsidase α or β), and disease severity, as determined by the FASTEX score. Results This study included twelve patients with FD (5 males, 7 females). We observed significantly greater relative declining changes in plasma lyso-Gb3 concentration among males compared to females (one year: −71.9 ± 4.5% in males vs. −20.6 ± 13.7% in females, p &amp;lt; 0.01; minimum levels: −78.5 ± 8.9% in males vs. −48.4 ± 19.6% in females, p = 0.01). No significant difference emerged in lyso-Gb3 reduction between agalsidase α and β treatments over all. One year after treatment, the relative changes in the levels were −45.1 ± 32.6% for agalsidase α and −37.7 ± 24.2% for agalsidase β (p = 0.68). The minimum levels followed suit, with −59.4 ± 26.8% for agalsidase α and −63.2 ± 15.2% for agalsidase β (p = 0.78). Lastly, the relative changes in lyso-Gb3 one year after initiating ERT were significantly associated with age (r = −0.873, p &amp;lt; 0.001), FASTEX score (r = 0.814, p = 0.001), baseline α-galactosidase A activity (r = −0.833, p = 0.001), and baseline lyso-Gb3 levels (r = 0.877, p &amp;lt; 0.001). Additionally, at the lowest levels, the relative changes in lyso-Gb3 showed a significant association with baseline α-galactosidase A activity (r = −0.887, p &amp;lt; 0.001), and baseline lyso-Gb3 levels (r = 0.702, p = 0.011). Conclusion The findings of our study suggest that relative changes in lyso-Gb3 are more pronounced in male patients, those with lower baseline α-galactosidase A activity, and those with higher baseline plasma lyso-Gb3 levels. Patients exhibiting severe α-galactosidase A deficiency and more severe clinical symptoms may experience greater relative declines in plasma lyso-Gb3 concentration following ERT.

Testosterone replacement therapy in patients with cachexia: a contemporary review of the literature.

Patients with long-term chronic illnesses frequently present with hypogonadism, which is primarily managed through exogenous testosterone. These same patients also experience a high degree of cachexia, a loss of skeletal muscle and adipose tissue. To perform a contemporary review of the literature to assess the effectiveness of testosterone replacement therapy (TRT) for managing chronic disease-associated cachexia. We performed a PubMed literature search using MeSH terms to identify studies from 2000 to 2022 on TRT and the following cachexia-related chronic medical diseases: cancer, COPD, HIV/AIDS, and liver cirrhosis. From the literature, 11 primary studies and 1 meta-analysis were selected. Among these studies, 3 evaluated TRT on cancer-associated cachexia, 3 on chronic obstructive pulmonary disease, 4 on HIV and AIDS, and 2 on liver cirrhosis. TRT showed mixed results favoring clinical improvement on each disease. Cachexia is commonly observed in chronic disease states. Its occurrence with hypogonadism, alongside the shared symptoms of these 2 conditions, points toward the management of cachexia through the administration of exogenous testosterone. Robust data in the literature support the use of testosterone in increasing lean body mass, improving energy levels, and enhancing the quality of life for patients with chronic disease. However, the data are variable, and further studies are warranted on the long-term efficacy of TRT in patients with cachexia.

THE POTENTIAL OF HUMAN PLASMA AND HUMAN BLOOD PRODUCTS FOR IMMUNE PROTECTION

Intravenous Immunoglobulin (IVIg) were used for the first time at the late seventies for treatment of patient with primary and secondary immunodeficiencies. Тhe first observations opened a wide field for basic and clinical research leading to a rapidly expanding use of IVIg for the treatment of patients with multiple diseases. The immunoglobulin preparations contain large amount of intact form of IgG molecules with comparable values of subclasses as in native plasma. Тhose properties account for the normal half-life of injected immunoglobulin of three weeks, and its ability to react normal with the complement components and with the specific Fcγ-receptors on the surface of phagocytes and lymphocytes. IVIg is produced from plasma pules from several thousand donors, which leads to wide spectre of variable regions of IgG molecules in the product. Some of the antibodies in the product can recognize bacterial, virus and fungal antigens are essential in replacement therapy in patients with antibody deficiency. А good understanding of the molecular and cellular basis of the immunoregulatory actions of intravenous immunoglobulin preparations is important for optimizing their use in inflammatory diseases for conducting new clinical observations.