Rev Assoc Med Bras (1992). 2023 Feb 17;69(1):147-152. doi: 10.1590/1806-9282.20221035. eCollection 2023.

ABSTRACT

OBJECTIVE: Postvaccine side effects were evaluated in patients presenting to our emergency department with complaints of vaccine side effects after taking COVID-19 vaccine, and new unknown side effects ranging from mild complaints to life-threatening risks, and frequency of all side effects were investigated. This study aimed to establish a scientific resource to identify the potential side effects of the vaccine.

METHODS: Patients' demographic information, clinical characteristics, epicrisis reports, COVID-19 disease and vaccination histories, vital values, and blood values were examined. The SPSS 20.0 package program was used for statistical evaluation. p<0.05 was considered statistically significant.

RESULTS: Notably, 13.1% of patients presenting to the emergency department started to have complaints after taking Sinovac vaccine, whereas 86.9% of them had complaints after taking BioNTech vaccine. Also, 36.9% of patients stated that they had COVID-19. All patients had a Glasgow coma scale score of 15 during admission. No patient was hospitalized, ventilator was not needed, and all patients were discharged. While the most common presenting complaint to the emergency department after vaccination was fatigue in 29.7%, the most common diagnoses after examination in the emergency department were myalgia in 32.1% and upper respiratory tract infection in 28.6%.

CONCLUSION: Results and conclusions of our study will guide healthcare workers and patients on the side effects of COVID-19 vaccine.

PMID:36820721 | PMC:PMC9937590 | DOI:10.1590/1806-9282.20221035

Rev Soc Bras Med Trop. 2023 Feb 20;56:e0253. doi: 10.1590/0037-8682-0253-2022. eCollection 2023.

ABSTRACT

BACKGROUND: This cross-sectional study compared the general impact of cutaneous leishmaniasis (CL) and patient satisfaction with treatment and health services as perceived by those undergoing different therapeutic regimens in an endemic region in South-Eastern Brazil. We also investigated the factors associated with both outcomes (general impact and satisfaction).

METHODS: We included 84 patients with CL treated between 2018 and 2019 with intravenous meglumine antimoniate, liposomal amphotericin B, or intralesional meglumine antimoniate therapy. Data were collected through interviews that assessed sociodemographic characteristics, comorbidity status, access and use of health services for CL diagnosis and treatment, and the items of the Cutaneous Leishmaniasis Impact Questionnaire (CLIQ). The CLIQ is a psychometric questionnaire previously validated to assess the general impact of CL on patient satisfaction with treatment and health services. Multivariate logistic regression analysis was used to identify the factors associated with high CL impact and low patient satisfaction.

RESULTS: The general impact of CL and patient satisfaction with treatment and health services were not significantly associated with the therapeutic regimen. High CL impact was associated with low family income (odds ratio [OR]:3.3; 95% confidence interval [CI]:1.0-10.3), occurrence of complications/adverse effects during treatment (OR:7.7; 95%CI:2.4-25.6), and additional costs during diagnosis and/or treatment (OR:12.1; 95% CI:2.8-52.4). Low satisfaction was associated with high disease impact (OR: 9.5; 95% CI:2.7-33.9), occurrence of complications/adverse effects (OR:4.2; 95% CI:1.3-13.0), and high family income (OR:7.1; 95%CI:1.7-28.2).

CONCLUSIONS: Our data support public health policies aimed at reducing the impact of CL and its treatment as well as the use of therapy with fewer adverse effects.

PMID:36820654 | PMC:PMC9957146 | DOI:10.1590/0037-8682-0253-2022

Rev Soc Bras Med Trop. 2023 Feb 20;56:e0209. doi: 10.1590/0037-8682-0209-2022. eCollection 2023.

ABSTRACT

BACKGROUND: This study aimed to assess the immunoglobulin G (IgG) antibody response rate in emergency department (ED) healthcare workers (HCWs) and potential adverse effects after CoronaVac vaccination.

METHODS: All included HCWs were grouped based on the previous history of coronavirus disease 2019 (COVID-19) and the number of vaccinations. Furthermore, the IgG antibody response was evaluated based on the sex and smoking status of HCWs. Those with a cut-off index of ≥1.00 after vaccination with CoronaVac were considered to have had COVID-19 and had an adequate humoral response.

RESULTS: Among 224 ED HCWs, 18% experienced the adverse effects of CoronaVac vaccine, the most prevalent being pain in the injection site. The IgG antibody response rate was 20% after the first dose of vaccine, while the response rate increased to 90% after the second dose. Female HCWs had higher IgG response rates compared with male HCWs (53.8 [15.9-147.0] vs 31.2 [4.5-124.0]). Non-smokers had higher IgG response rate compared with smokers (49.0 [11.5-160.5] vs 23.1 [7.4-98.5]).

CONCLUSION: A single dose of CoronaVac does not produce a sufficient antibody response; hence, two doses are recommended. Men have a lower IgG response compared with women. Smokers had a lower IgG response rate compared with non-smokers. Therefore, it may be necessary to carefully assess the humoral responses of men and smokers when implementing a community vaccination program.

PMID:36820653 | PMC:PMC9957120 | DOI:10.1590/0037-8682-0209-2022

Medicina (B Aires). 2023 Mar;83 Suppl 2:27-31.

ABSTRACT

INTRODUCTION: ADHD treatment is safe and effective, but often, adherence to t reatment is suboptimal. We studied factors associated to adherence to treatment in ADHD described in the literature.

METHODS: We conducted a non-systematic bibliographic search on recent articles on medication adherence in children and adolescents with ADHD.

RESULTS: There are factors associated with the disorder itself, the patient, the health system, socio-economic factors, the medication and the environment that can reduce/improve medication adherence. Some factors that improve adherence include: knowledge about ADHD, medication and its benefits and potential adverse effects. Simple, once-daily, extended-release medication regimens. Age of the patient less than 12 years and female sex. Fewer barriers to access the health system. Positive and proactive attitude of parents regarding ADHD and the use of medication.

DISCUSSION: Adherence could be optimized and therefore the long-term prognosis of ADHD improved by reinforcingfactors that increase adherence, and reducing biases and ignorance about ADHD.

PMID:36820479

Br J Gen Pract. 2023 Feb 23;73(728):e211-e219. doi: 10.3399/BJGP.2022.0181. Print 2023 Mar.

ABSTRACT

BACKGROUND: To date, research on adverse drug reactions (ADRs) has focused on secondary care, and there is a paucity of studies that have prospectively examined ADRs affecting older adults in general practice.

AIM: To examine the cumulative incidence and severity of ADRs and associated patient characteristics in a sample of community-dwelling older adults.

DESIGN AND SETTING: Prospective cohort study of older adults (aged ≥70 years, N = 592) recruited from 15 general practices in the Republic of Ireland.

METHOD: Manual review of the participant's general practice electronic medical record, linked to the national dispensed prescription medicine database, and a detailed, self-reported patient postal questionnaire. The primary outcomes were ADR occurrence and severity over a 6-year period (2010-2016). Unadjusted and adjusted logistic regression models examined potential associations between patient characteristics and ADR occurrence.

RESULTS: A total of 211 ADRs were recorded for 159 participants, resulting in a cumulative incidence of 26.9% over 6 years. The majority of ADRs detected were mild (89.1%), with the remainder classified as moderate (10.9%). Eight moderate ADRs, representing 34.8% of moderate ADRs and 3.8% of all ADRs, required an emergency hospital admission. ADRs were independently associated with female sex (adjusted odds ratio [OR] 1.83, 95% confidence interval [CI] = 1.17 to 2.85; P = 0.008), polypharmacy (5-9 drug classes) (adjusted OR 1.81, 95% CI = 1.17 to 2.82; P = 0.008), and major polypharmacy (≥10 drug classes) (adjusted OR = 3.33, 95% CI = 1.62 to 6.85; P = 0.001).

CONCLUSION: This prospective cohort study of ADRs in general practice shows that over one-quarter of older adults experienced an ADR over a 6-year period. Polypharmacy is independently associated with ADR risk in general practice and older adults on ≥10 drug classes should be prioritised for regular medication review.

PMID:36823047 | DOI:10.3399/BJGP.2022.0181

Cancer Treat Rev. 2023 Feb 14;115:102525. doi: 10.1016/j.ctrv.2023.102525. Online ahead of print.

ABSTRACT

Non-metastatic castration resistant prostate cancer (nmCRPC) is a clinical setting defined as confirmed rising levels of PSA in patients treated with ADT but without detectable metastases on conventional imaging with computerized tomography (CT) and technetium-99 m scintigraphy. Men with nmCRPC and a PSA doubling time (PSADT) ≤ 10 months are considered at high risk of rapidly developing metastases with a consequent possible impact on survival. Three recent phase III trials have demonstrated, in this setting, the efficacy of adding a next-generation androgen receptor targeted agent (ARTA) to ADT in respect to ADT only, in delaying the development of metastases (metastasis-free survival, MFS) and prolong overall survival. The magnitude of clinical benefit of these agents was even more meaningful if considering the low incidence of drug related adverse events. Our review described the latest advances in the management of nmCRPC, deriving from the pivotal clinical trials, SPARTAN, PROSPER and ARAMIS, in order to support clinicians to optimally manage these patients. Of note, the emergence of novel, more accurate, next-generation imaging techniques (including Ga PSMA-PET/CT), as well as eventual future tumor biomarkers, is modifying the entity and definition of the nmCRPC setting, with a consequent impact on patient's diagnosis and management.

PMID:36822009 | DOI:10.1016/j.ctrv.2023.102525

Medicine (Baltimore). 2023 Feb 10;102(6):e32844. doi: 10.1097/MD.0000000000032844.

ABSTRACT

RATIONALE: Eczematous eruption is an increasingly recognized form of drug-related eruption, typically reported in association with interleukin 17 (IL-17)A inhibitors. However, severe paradoxical eczematous eruption due to IL-17A inhibitors has been rarely reported. Herein, we reported a case of a man with severe psoriasis with erythematous scaly plaques on the scalp, trunk, and arms and legs after the administration of secukinumab was initiated.

PATIENT CONCERNS: We reported a case of a 20-year-old man with severe psoriasis with erythematous scaly plaques on the scalp, trunk, and arms and legs after the administration of secukinumab was initiated. A skin biopsy was performed. It revealed spongiotic dermatitis consistent with eczematous reaction. Direct and indirect immunofluorescence assays were negative.

DIAGNOSES: He was diagnosed with eczematous eruption.

INTERVENTIONS: Discontinuation of secukinumab and administration of cyclosporine and prednisone were considered.

OUTCOMES: Significant improvement was observed, with no adverse events.

CONCLUSION: Our case shows that eczematous eruption can paradoxically occur in patients on IL-17A inhibitors and this report is expected to increase awareness of the rising number of cutaneous eruptions related to biological agents.

PMID:36820537 | DOI:10.1097/MD.0000000000032844

Cureus. 2023 Jan 16;15(1):e33849. doi: 10.7759/cureus.33849. eCollection 2023 Jan.

ABSTRACT

β-Lactams, particularly penicillins, may cause several allergic reactions. We described symmetric drug-related intertriginous and flexural exanthema (SDRIFE) illness in this case, a rare instance of systemic contact dermatitis caused by amoxicillin/clavulanic acid that needs to be considered in the differential diagnosis. A 65-year-old male patient was admitted to our Allergy Outpatient Clinic because of increased blue-purple pigmentation on the flexural surfaces of the hip, forearm, axilla, and posterior face of the neck. The patient was receiving a combination of angiotensin receptor blocker (ARB) and hydrochlorothiazide diuretic medication for hypertension. The patient used an antibiotic containing amoxicillin three months ago; As a result, there was localized redness, itching, and black spotting without any systemic symptoms. Similarly, the patient reported that when he used amoxicillin for an upper respiratory tract infection eight months ago, he experienced similar side effects within 20 days and recovered when he applied corticosteroid ointment. Due to the symmetrical site involvement following the consumption of penicillin group antibiotics with a five-month gap and subsequent comparable reactions in our patient, SDRIFE was taken into consideration. The results of the skin punch biopsy identified Baboon Syndrome (SDRIFE). Treatment with topical corticosteroids and antihistamines began. Clinically speaking, SDRIFE is distinguished by significant erythema of the gluteal/perianal area and/or V-shaped erythema of the inguinal/perigenital area, symmetric involvement of at least one other intertriginous or flexural area, and the absence of systemic signs or symptoms. The possibility that the medication may have contributed to the patient's erythematous eruption in the flexural regions should be taken into account, and the patient should be advised to stop taking the medication and not use it again.

PMID:36819357 | PMC:PMC9932368 | DOI:10.7759/cureus.33849

World J Clin Cases. 2023 Feb 6;11(4):866-873. doi: 10.12998/wjcc.v11.i4.866.

ABSTRACT

BACKGROUND: The advent of molecular targeted agents and immune checkpoint inhibitors has greatly improved the treatment of advanced renal cell carcinoma (RCC), thus significantly improving patient survival. The incidence of rare drug-related adverse events has gained increased attention.

CASE SUMMARY: We report a patient with advanced RCC treated with multiple lines of molecular targeted agents and immune checkpoint inhibitors, who developed a pulmonary infection after treatment with everolimus in combination with lenvatinib. Determining the pathogenic organism was difficult, but it was eventually identified as Pneumocystis jirovecii by next-generation sequencing (NGS) of bronchoscopic alveolar lavage fluid (BALF) and successfully treated with trimethoprim-sulfamethoxazole.

CONCLUSION: Rare pulmonary infections caused by molecular targeted agents are not uncommon in clinical practice, but their diagnosis is difficult. Evaluating BALF with NGS is a good method for rapid diagnosis of such infections.

PMID:36818625 | PMC:PMC9928697 | DOI:10.12998/wjcc.v11.i4.866

Wideochir Inne Tech Maloinwazyjne. 2022 Dec;17(4):634-640. doi: 10.5114/wiitm.2022.120913. Epub 2022 Nov 8.

ABSTRACT

INTRODUCTION: Ropivacaine is widely used as a local analgesic, but it has toxicity that is related to the concentration, and highly concentrated ropivacaine can induce motor nerve blockage.

AIM: To investigate the safety of low-concentration pre-incisional ropivacaine injection for postoperative pain control and compare postoperative adverse events between a low-concentration ropivacaine injection group and a high-concentration ropivacaine injection group.

MATERIAL AND METHODS: Patients who underwent thyroidectomy via the bilateral axillo-breast approach (BABA) between June 2017 and October 2021 performed by a single surgeon at Samsung Medical Center were retrospectively identified. These outcomes were compared between the two groups after 1 : 1 propensity score matching.

RESULTS: From a total of 633 patients, 620 patients were selected. There were 527 in the low-concentration ropivacaine group and 93 in the high-concentration ropivacaine group. After propensity score matching, two comparable groups with 93 patients in each were obtained. The incidence of ropivacaine-related adverse events was similar between the two groups (p = 0.186) but the occurrence of postoperative bradycardia (p = 0.048) was lower in the low-concentration ropivacaine group than in the high-concentration ropivacaine group. Other outcomes such as postoperative pain scores (p = 0.363), postoperative nausea and vomiting (p > 0.999), and postoperative opioid consumption (p = 0.699) were similar between the two groups.

CONCLUSIONS: Pre-incisional low-concentration ropivacaine injection was effective for postoperative pain control and can be safely used in BABA thyroidectomy.

PMID:36818509 | PMC:PMC9909757 | DOI:10.5114/wiitm.2022.120913

Drug Ther Bull. 2023 Mar;61(3):44-47. doi: 10.1136/dtb.2022.243191rep. Epub 2022 Aug 31.

NO ABSTRACT

PMID:36813280 | DOI:10.1136/dtb.2022.243191rep

BMC Health Serv Res. 2023 Feb 21;23(1):176. doi: 10.1186/s12913-023-09164-6.

ABSTRACT

BACKGROUD: With the reform of medical system in China, Beijing municipal hospitals explored a new pharmaceutical care model and set up medication therapy management services (MTMs) in ambulatory care since 2019. We were one of the first hospitals to set up this service in China. At the present, there were relatively few reports about the effect of MTMs in China. In this study, we summarized the implementation of MTMs in our hospital, explore the feasibility of pharmacist-led MTMs in ambulatory care and the impact of MTMs on patients' medical costs.

METHODS: A retrospective study was conducted in a university-affiliated, tertiary comprehensive hospital in Beijing, China. The patients who received at least one MTMs and with complete medical records and pharmaceutical documents from May 2019 to February 2020 were included. Pharmacists provided pharmaceutical care for patients according to the MTMs standards issued by the American Pharmacists Association, identified the numbers and classification of the patients' perceived medication-related demands, identified medication-related problems (MRPs), and developed the medication-related action plans (MAPs). All MRPs found by pharmacists, pharmaceutical interventions, and resolving recommendations were documented, and calculate the cost of treatment drugs that patients can reduce.

RESULTS: A total of 112 patients received MTMs in ambulatory care, among them 81 cases with the completed record were included in this study. 67.9% of patients had five or more diseases, 83% of them co-took over 5 drugs. While performing MTMs, 128 patients' perceived medication-related demands were recorded in all, monitoring and judgment of adverse drug reaction (ADR) (17.19%) was the most common demand. 181 MRPs were found, with an average of 2.55 MPRs per patient. Nonadherence (38%), excessive drug treatment (20%), and adverse drug events (17.12%) were the top three MRPs. Pharmaceutical care (29.77%), adjustment of drug treatment plan (29.10%) and referral to the clinical department (23.41%) were the top three MAPs. Whereby the MTMs provided by pharmacists, the cost-saving of each patient was about $ 43.2 monthly.

CONCLUSION: By participating in the MTMs of outpatients, the pharmacists could identify more MRPs and develop personalized MAPs timely for patients, thereby promoting rational drug use and reducing medical expenses.

PMID:36810022 | DOI:10.1186/s12913-023-09164-6

J Clin Neuromuscul Dis. 2023 Mar 1;24(3):162-163. doi: 10.1097/CND.0000000000000434.

NO ABSTRACT

PMID:36809204 | DOI:10.1097/CND.0000000000000434

Dermatol Online J. 2022 Oct 15;28(5). doi: 10.5070/D328559261.

NO ABSTRACT

PMID:36809142 | DOI:10.5070/D328559261

Dermatol Online J. 2022 Oct 15;28(5). doi: 10.5070/D328559239.

ABSTRACT

BACKGROUND: Changes to the iPLEDGE platform on December 13, 2021 made isotretinoin virtually inaccessible for many patients. Prior to the FDA approval of isotretinoin, a derivative of vitamin A, in 1982, vitamin A was used for severe acne.

OBJECTIVE: To review the efficacy, safety, affordability, and practicality of vitamin A as a substitute for isotretinoin when the latter is inaccessible.

METHODS: A literature review of PubMed was conducted using the key words: oral vitamin A, retinol, isotretinoin, Accutane, acne, iPLEDGE, hypervitaminosis A, and side effects.

RESULTS: We identified 9 studies (8 clinical trials and one case report); acne improved in 8 studies. Dosages ranged from 36,000IU daily to 500,000IU with 100,000IU as the most common. Mean duration until clinical improvement was 7 weeks to four months after initiation of therapy. Mucocutaneous side effects were most common, along with headaches, which resolved with either continued treatment or cessation.

CONCLUSION: Oral vitamin A is efficacious for the treatment of acne vulgaris, although the available studies have limited controls and outcomes. Side effects are qualitatively similar to those of isotretinoin and avoiding pregnancy for at least three months after stopping treatment is critical; like isotretinoin, vitamin A is a teratogen.

PMID:36809126 | DOI:10.5070/D328559239

Bioinformatics. 2023 Feb 3;39(2):btad085. doi: 10.1093/bioinformatics/btad085.

ABSTRACT

MOTIVATION: Predicting molecule-disease indications and side effects is important for drug development and pharmacovigilance. Comprehensively mining molecule-molecule, molecule-disease and disease-disease semantic dependencies can potentially improve prediction performance.

METHODS: We introduce a Multi-Modal REpresentation Mapping Approach to Predicting molecular-disease relations (M2REMAP) by incorporating clinical semantics learned from electronic health records (EHR) of 12.6 million patients. Specifically, M2REMAP first learns a multimodal molecule representation that synthesizes chemical property and clinical semantic information by mapping molecule chemicals via a deep neural network onto the clinical semantic embedding space shared by drugs, diseases and other common clinical concepts. To infer molecule-disease relations, M2REMAP combines multimodal molecule representation and disease semantic embedding to jointly infer indications and side effects.

RESULTS: We extensively evaluate M2REMAP on molecule indications, side effects and interactions. Results show that incorporating EHR embeddings improves performance significantly, for example, attaining an improvement over the baseline models by 23.6% in PRC-AUC on indications and 23.9% on side effects. Further, M2REMAP overcomes the limitation of existing methods and effectively predicts drugs for novel diseases and emerging pathogens.

AVAILABILITY AND IMPLEMENTATION: The code is available at https://github.com/celehs/M2REMAP, and prediction results are provided at https://shiny.parse-health.org/drugs-diseases-dev/.

SUPPLEMENTARY INFORMATION: Supplementary data are available at Bioinformatics online.

PMID:36805623 | PMC:PMC9940625 | DOI:10.1093/bioinformatics/btad085

Drug Ther Bull. 2023 Mar;61(3):38. doi: 10.1136/dtb.2023.000008.

ABSTRACT

Overview of: Kim BK, Hong SJ, Lee YJ, et al Long-term efficacy and safety of moderate-intensity statin with ezetimibe combination therapy versus high-intensity statin monotherapy in patients with atherosclerotic cardiovascular disease (RACING): a randomised, open-label, non-inferiority trial. Lancet 2022;400:380-390.

PMID:36813278 | DOI:10.1136/dtb.2023.000008

Drug Ther Bull. 2023 Feb 22:dtb-2023-000010. doi: 10.1136/dtb.2023.000010. Online ahead of print.

ABSTRACT

Overview of: Medicines and Healthcare products Regulatory Agency. Xaqua (metolazone) 5mg tablets: exercise caution when switching patients between metolazone preparations. Drug Safety Update 2023;16:1.

PMID:36813274 | DOI:10.1136/dtb.2023.000010

Haemophilia. 2023 Feb 22. doi: 10.1111/hae.14762. Online ahead of print.

ABSTRACT

INTRODUCTION: The UK National Haemophilia Database (NHD) collects data from all UK persons with haemophilia A with inhibitors (PwHA-I). It is well-placed to investigate patient selection, clinical outcomes, drug safety and other issues not addressed in clinical trials of emicizumab.

AIMS: To determine safety, bleeding outcomes and early effects on joint health of emicizumab prophylaxis in a large, unselected cohort using national registry and patient reported Haemtrack (HT) data between 01 January 2018 and 30 September 2021.

METHODS: Prospectively collected bleeding outcomes were analysed in people with ≥6 months emicizumab HT data and compared with previous treatment if available. Change in paired Haemophilia Joint Health Scores (HJHS) were analysed in a subgroup. Adverse events (AEs) reports were collected and adjudicated centrally.

RESULTS: This analysis includes 117 PwHA-I. Mean annualised bleeding rate (ABR) was .32 (95% CI, .18; .39) over a median 42 months treatment with emicizumab. Within-person comparison (n = 74) demonstrated an 89% reduction in ABR after switching to emicizumab and an increase in zero treated bleed rate from 45 to 88% (p < .01). In a subgroup of 37 people, total HJHS improved in 36%, remained stable in 46% and deteriorated in 18%, with a median (IQR) within-person change of -2.0 (-9, 1.5) (p = .04). Three arterial thrombotic events were reported, two possibly drug related. Other AEs were generally non-severe and usually limited to early treatment, included cutaneous reactions (3.6%), headaches (1.4%), nausea (2.8%) and arthralgia (1.4%).

CONCLUSIONS: Emicizumab prophylaxis is associated with sustained low bleeding rates and was generally well-tolerated in people with haemophilia A and inhibitors.

PMID:36811304 | DOI:10.1111/hae.14762

J Xenobiot. 2023 Jan 28;13(1):29-41. doi: 10.3390/jox13010005.

ABSTRACT

Immune thrombocytopenic purpura (ITP) is an acquired antibody or cell-mediated platelet damage or decreased platelet production. Steroids, IV immunoglobulins (IVIG), and Rho-anti-D antibodies are the commonly used initial treatments for ITP. However, many ITP patients either do not respond or do not maintain a response to initial therapy. Splenectomy, rituximab, and thrombomimetics are the commonly used second-line treatment. More treatment options include tyrosine kinases inhibitors (TKI), including spleen tyrosine kinase (Syk) and Bruton's tyrosine kinase (BTK) inhibitors. This review aims to assess the safety and efficacy of TKIs. Methods: Literature was searched on PubMed, Embase, WOS, and clinicaltrials.gov using keywords, "tyrosine kinase" and "idiopathic thrombocytopenic purpura". PRISMA guidelines were followed. Results: In total, 4 clinical trials were included with 255 adult patients with relapsed/refractory ITP. In all, 101 (39.6%) patients were treated with fostamatinib, 60 (23%) patients with rilzabrutinib, and 34 (13%) with HMPL-523. Patients treated with fostamatinib achieved a stable response (SR) and overall response (OR) in 18/101 (17.8%) and 43/101 (42.5%) of the patients, respectively, while SR and OR were achieved in 1/49 (2%) and 7/49 (14%) of the patients, respectively, in the placebo group. Patients treated with HMPL-523 (300 mg dose expansion) achieved an SR and OR in 5/20 (25%) and 11/20 (55%) of the patients, respectively, while SR and OR were achieved in 1/11 (9%) of the patients treated with the placebo. Patients treated with rilzabrutinib achieved an SR in 17/60 (28%) patients. Dizziness (1%), hypertension (2%), diarrhea (1%), and neutropenia (1%) were serious adverse events in fostamatinib patients. Rilzabrutinib or HMPL-523 patients did not require a dose reduction due to drug-related adverse effects. Conclusions: Rilzabrutinib, fostamatinib, and HMPL-523 were safe and effective in the treatment of relapsed/refractory ITP.

PMID:36810430 | DOI:10.3390/jox13010005