Ben Holmes, DVM

Intended Audience: Pediatricians, orthopedists/orthopedic surgeons, endocrinologists, including metabolic bone specialists, geneticists, and their teams of NPs, PAs, pharmacists, and nurses Despite being considered a rare disorder, achondroplasia (ACH) is the most common skeletal dysplasia, accounting for ~90% of cases of disproportionate stature. With recent updates to the International Consensus Statement on the diagnosis and lifelong care of individuals with ACH, as well as an evolving therap

Approximately how many adult patients with spinal muscular atrophy (SMA) have you encountered in your clinical practice? Approximately how many adult patients with spinal muscular atrophy (SMA) have you encountered in your clinical practice?

What is the greatest challenge facing your clinical team in the treatment and management of adult patients with SMA? What is the greatest challenge facing your clinical team in the treatment and management of adult patients with SMA?

Please rate your level

Associate Professor, Department of Neurology at NYU Grossman School of Medicine Director, Division of Child Neurology Director, Neurofibromatosis Program

Neurofibromatosis type 1 (NF1) is a debilitating and disfiguring condition characterized by benign plexiform neurofibromas (PN) that develop along nerve sheaths throughout the body. In this 1-hour video activity, two renowned NF1 PN experts discuss strategies to improve early identification of NF1 PN, best screening and diagnostic practices, t

Immune-mediated inflammatory diseases (IMIDs), including rheumatoid arthritis (RA) and spondyloarthritides, are common, clinically diverse and chronic. While significant variations in population distribution, tissue localisation, clinical phenotypes and therapeutic responses are apparent between different IMIDs, they share many similar pathophysiological mechanisms.1–3 Small-molecule Janus kinase (JAK) inhibitors are a therapeutic class for the management of IMIDs.4 5 Although certain adverse ev

Chemotherapy patients often experience nausea and vomiting that not only has a negative impact on the patient’s quality of life but can also result in unplanned hospitalizations with high associated costs. Numerous medications and specific guidelines are available to prevent nausea and vomiting in patients with cancer. Specifically, the combination of two classes of medications (serotonin inhibitors + neurokinin type 1 inhibitors) has been shown to provide the greatest benefit. However, hospital

For patients with fibrodysplasia ossificans progressiva (FOP), prompt diagnosis and effective management are crucial to preventing disease exacerbation and improving quality of life. In this convenient three-part micro-learning video library, expert faculty will review the pathophysiology of FOP, latest diagnostic strategies, clinical evidence related to emerging therapeutics that are poised to revolutionize the FOP treatment paradigm, and strategies for personalized, patient-centered FOP care.

Background
Upadacitinib is a selective reversible Janus kinase (JAK) inhibitor with established efficacy in moderate-to-severe atopic dermatitis (AD).

Objective
We evaluated the safety of upadacitinib in patients with moderate-to-severe AD.

Methods
Integrated safety data from the 16-week placebo-controlled periods of 1 phase 2b and 3 ongoing phase 3 studies (16 weeks) and longer-term safety data from patients receiving upadacitinib during the blinded extension periods of the three phase 3 studies were analyzed (all upadacitinib exposure). Treatment-emergent adverse events (TEAEs) were presented as exposure-adjusted rates per 100 patient-years (PY).

Results
Safety results were similar between the 16-week and all upadacitinib exposure groups. The latter group included 2485 patients (333 adolescents), receiving upadacitinib 15 mg (n = 1239) or 30 mg (n = 1246) for a mean duration of approximately 1 year. Upadacitinib was well tolerated by both adults and adolescents. TEAEs and discontinuation due to AEs were reported more frequently in patients receiving 30 mg upadacitinib (respectively, 311.9 and 5.7 events per 100 PY) versus 15 mg (respectively, 274.6 and 4.4 events per 100 PY). Serious adverse event rates (15/30 mg, 7.1/7.7 events per 100 PY) were similar in both groups. Acne was the most frequently reported adverse event (15/30 mg, 13.3/20.2 events per 100 PY). Serious infection rates were similar across treatment groups. Adjudicated major adverse cardiovascular event and venous thromboembolic event rates were ≤0.1 events per 100 PY. Rates of malignant neoplasms were within the expected range for the general population.

Conclusions
Upadacitinib was well tolerated, and no new important safety risks were observed among adults and adolescents with moderate-to-severe AD treated for approximately 1 year compared to the known safety profile of upadacitinib.

Background/Purpose: Patients with untreated immune-mediated inflammatory diseases, such as RA, PsA, and AS, are at increased risk for major adverse cardiovascular events (MACE) and venous thromboembolic events (VTE) compared with the general population.

The purpose of this analysis was to describe the events and risk factors for MACE and VTE in the RA, PsA, and AS clinical trial programs of the Janus kinase (JAK) inhibitor, upadacitinib (UPA).

Methods: Treatment-emergent adverse events (TEAEs)

As with any infection, immunosuppressed/immunocompromised (ISC) patients are at great risk for COVID-19, with solid organ transplant (SOT) recipients experiencing an approximately 15-fold increased incidence of COVID-19 compared to the general immunocompetent population.1

Even though COVID vaccines have benefitted the general population, including the elderly and those with comorbidities, the value of vaccination in SOT patients remains in question. While adequate response to natural SARS-CoV-2

Research is ongoing to explore all potential uses for circulating tumor DNA to help reduce the global burden of colorectal cancer.

Circulating tumor (ctDNA) technology has the potential to improve diagnosis, treatment, and monitoring of patients with colorectal cancer (CRC).1 Research is ongoing to explore all potential uses for ctDNA to help reduce the global burden of CRC.1-4 Although CRC is preventable and curable when diagnosed early, most patients receive diagnoses at advanced stages.2 Ear

Out-of-hospital cardiac arrest (OHCA) is a leading cause of mortality in adults worldwide, with approximately 10% of patients with OHCA surviving to hospital discharge and only 8.2% surviving with good functional status.1,2 Coordinated, timely intervention is paramount, requiring accurate recognition, cardiopulmonary resuscitation (CPR), and defibrillation to maximize the chances of a positive outcome.2

While improving initial survival is a primary focus, there has been increased interest in lo

As the shift toward precision medicine drives a push for more individualized treatments for patients with cancer, uterine sarcoma therapy is an area ripe for greater personalization. With expanding knowledge of the molecular characteristics of uterine sarcomas, investigators are working to develop treatments specific to each subtype.

Uterine sarcomas are a group of rare, aggressive mesenchymal tumors, accounting for approximately 3% to 4% of all malignant uterine neoplasms.1-3 The most common h

Elagolix is an oral GnRH antagonist approved for the treatment of moderate to severe endometriosis-associated pain (EAP) in the US, Canada, and Israel. Hormonal add-back therapy (1 mg estradiol/0.5 mg norethindrone acetate QD) may alleviate the hypoestrogenic effects (including bone mineral density [BMD] loss) associated with elagolix. The long-term safety of elagolix 200mg BID+add-back are currently under investigation in an ongoing phase 3, 48-month (M) study for EAP; this report focuses on op

Antiandrogens Progress In New Settings, Combinations in Prostate Cancer

Data presented at the 2022 American Society of Clinical Oncology Genitourinary Cancers Symposium highlight the inhibitors’ use in combination regimens, other settings, and next-generation agents.

Second-generation androgen receptor (AR) signaling inhibitors have become the standard of care for treating patients with castration-resistant prostate cancer (CRPC). Data presented at the 2022 American Society of Clinical Oncolog

The Potential of STING Agonists Is Explored in Cancer

Numerous classes of STING agonists are being evaluated for use, including novel cyclic dinucleotides, next-generation noncyclic dinucleotides, bacterial vectors, and ENPP1 inhibitors.

Stimulator of interferon genes (STING) is an endoplasmic protein that induces the production of proinflammatory cytokines and has become a target of interest to obtain or boost antitumor immune responses.1 First-generation STING agonists are structurally unsta

Although numerous therapeutics are available for the management of metastatic renal cell carcinoma in the first-line setting, there are few options and little guidance on the approach to adjuvant therapy.

Most patients renal cell carcinoma (RCC) initially present with localized disease that is curable with surgical intervention (complete nephrectomy).1,2 Unfortunately, 20% to 40% of those with localized primary tumors will develop metastatic disease, and approximately 1 in 4 patients with RCC h

Optimal management of MIBC should include multidisciplinary care involving tumor board discussions and/or direct consultations with a medical oncologist, radiation oncologist, and urologist [IV, B]. RC with pelvic lymph node dissection remains the standard of care for MIBC cT2-T4aN0M0 [I, A]. 9

Due to the nature of the studies and lack of approvals for both pembrolizumab and nadofaragene firadenovec, further robust data are required before stronger recommendations can be made for their use. 3

Seeking Clarity for Treatment Sequencing With CAR T in Follicular Lymphoma

Considering later lines of treatment such as chimeric antigen receptor T-cell therapy, make it difficult for chimeric antigen receptor T-cell therapy to find its optimal place in the armamentarium.

Numerous therapeutic options are available for the treatment of follicular lymphoma (FL) but identifying the ideal sequence of therapies, especially when considering later lines of treatment such as chimeric antigen receptor

Early evidence indicates that iterative detection, profiling, and targeting of minimal residual disease could improve outcomes and even lead to a cure.

Cancer treatment is undergoing a reformation because of novel therapeutics that provide dramatic responses, including complete radiographic or pathologic remission. However, minimal residual disease (MRD) is often retained, resulting in relapse. Early evidence indicates that iterative detection, profiling, and targeting of MRD could improve outc

In conjunction with the 10-year anniversary of Targeted Oncology™, we are highlighting the most impactful advancements concerning hepatocellular carcinoma (HCC) over the previous decade.

Tanios S. Bekaii-Saab, MD, FACP, medical oncologist, medical director, Cancer Clinical Research Office, vice chair and section chief, Medical Oncology, Department of Internal Medicine, Mayo Clinic; Richard S. Finn, MD, professor of medicine, Division of Hematology/Oncology, director, Signal Transduction and The

Novel ER Approaches for HR+/HER2– Breast Cancer Are Explored During SABCS

Research is ongoing to combat key resistance mechanisms, with new data recently presented by Sara A. Hurvitz, MD, FACP, and other oncologists and investigators during the 2021 San Antonio Breast Cancer Symposium.

Many patients with hormone receptor-positive/ HER2-negative metastatic breast cancer (mBC) develop resistance during endocrine therapy (ET) or relapse afterward because of various mechanisms, including mutations

Recent trials explore drugs like bemcentinib, everolimus, talazoparib, and others for potential approaches to targeting STK11 mutations.

STK11 mutation recently have become a biomarker and potential target of interest, especially for patients with lung cancer. Recent trials explore drugs like bemcentinib, everolimus, talazoparib, and others for potential approaches to targeting these mutations. The serine/threonine kinase 11 (STK11) gene encodes the liver kinase B1 (LKB1) protein, an intracellu

(CAR) T-cell therapy has revolutionized the treatment of hematologic malignancies since the initial approval of antiCD19 CAR T for acute lymphoblastic leukemia (ALL) in 2017.1 2021 proved an exceptionally busy year for approvals in CAR T therapeutics, with several ongoing trials showing that additional indications are not far behind.

Brexucabtagene autoleucel (Tecartus, brexu-cel), a CD19-directed CAR T product, was approved on October 1, 2021, by the FDA for the treatment of adults with relaps

ATR’s integral role in DNA damage repair has made it an appealing target for cancer treatment, and several ATR inhibitors are being evaluated for efficacy and safety across solid tumors.

Dysreguation of dysfunction of the DNA damage response (DDR) system can result in genomic instability, a well-known hallmark of many cancers.1,2 DDR deficiency can lead to greater potential for cancer development and malignant transformation (FIGURE), but DDR can also be a mechanism of resistance to chemotherap