Rare Disease Advisor

RareDiseaseAdvisor.com stands out as the leading online platform for news, insights, and resources aimed at healthcare providers treating rare diseases. This site focuses on filling the gaps in information for medical professionals who are looking for up-to-date knowledge on the rare conditions they encounter, offering original, reliable, and engaging content.

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SACQ IgG4-RD Status Has Potential to Manage Risk for Disease Relapse

1 day ago | rarediseaseadvisor.com | Sheila Jacobs

In patients with immunoglobulin G4-related disease (IgG4-RD), monitoring the re-elevation of serum IgG4 levels has the potential to predict relapses with respect to long-term management of serologically active clinically quiescent (SACQ) disease. A single-center study was conducted at Peking Union Medical College Hospital, Beijing, China, based on a prospective cohort study (NCT01670695) of individuals with IgG4-RD. The findings were published recently in Clinical Rheumatology.
Mayo Alliance Prognostic Score Better at Predicting Survival in SM

1 day ago | rarediseaseadvisor.com | Sheila Jacobs

The Mayo Alliance Prognostic Score (MAPS) for survival prediction in patients with systemic mastocytosis (SM) has demonstrated superior performance to the 5th edition of the World Health Organization (WHO5), with little additional contribution from mutations. In addition, subclassification of SM by the International Consensus Classification (ICC) has been shown to be prognostically independent of MAPS and more valuable than classification according to the WHO5.
Air Pollution Increases Risk of Mortality and Lung Transplantation in PAH

2 days ago | rarediseaseadvisor.com | Ryner Lai

Long-term ambient air pollution is associated with increased pulmonary arterial hypertension (PAH) baseline disease severity and a higher risk of death and lung transplantation among patients with this condition, according to a study published in the Journal of the American Heart Association. Air pollution continues to be a significant environmental contributor to the rise in noncommunicable diseases.
New Study Suggests Serum IgG4 Levels Not Perfect Tool to Diagnose IgG4-RD

2 days ago | rarediseaseadvisor.com | Sheila Jacobs

In patients with immunoglobulin G4-related disease (IgG4-RD), serum IgG4 levels alone are not an adequate screening tool for diagnosis of the disorder because of their low positive predictive value (PPV), according to findings from a single-center, retrospective study conducted in Spain and published recently in Reumatología Clínica.
Novel Noninvasive Biomarker Identified for PBC

2 days ago | rarediseaseadvisor.com | Ozge Ozkaya

Anti-RPL30 seems to be a promising biomarker for the diagnosis of primary biliary cholangitis (PBC) especially in patients who are negative for antimitochondrial antibody (AMA), antimitochondrial E2 subunit antibody (AMA-M2), antispeckled protein 100 (antiSp100) antibody, and antiglycoprotein 210 (antigp210) antibody, according to a new study published in the World Journal of Gastroenterology.
Cholesterol-Modified Prognostic Nutritional Index May Be Better Than Other Prognostic Indices for PBC

2 days ago | rarediseaseadvisor.com | Ozge Ozkaya

Cholesterol-modified Prognostic Nutritional Index (CPNI) is better than other nutritional scores for the prognosis of primary biliary cholangitis (PBC), according to a study published in the scientific journal BMC Gastroenterology. In other words, CPNI can better predict overall survival in patients with the disease.
Clinical Management Process Can Identify Undiagnosed Cases of PBC

2 days ago | rarediseaseadvisor.com | Ozge Ozkaya

Patients with undiagnosed primary biliary cholangitis (PBC) can be identified using hospital databases, according to a new study published in the scientific journal Annals of Hepatology. The authors of the study reported that they were able to diagnose and offer treatment to more than half (16 out of 28) of the patients they detected in this way. Read more about the diagnosis of PBCThe team analyzed immunology and biochemistry data from patients’ medical records from January 2019 to December 2021.
Safety Profile of Acalabrutinib Superior to Chemoimmunotherapy in CLL

3 days ago | rarediseaseadvisor.com | Sheila Jacobs

The cardiovascular safety profile of acalabrutinib is superior to that of chemoimmunotherapy in treating patients with chronic lymphocytic leukemia (CLL), according to findings from a retrospective cohort analysis presented at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago, Illinois. Of the study participants, 847 were treated with acalabrutinib and 3172 received chemoimmunotherapy.
Romiplostim Is Possible Alternative to Multiple Platelet Transfusions in NICU

3 days ago | rarediseaseadvisor.com | Sheila Jacobs

The use of romiplostim as an alternative therapeutic option in the neonatal intensive care unit (NICU) in an effort to avoid multiple platelet transfusions, has proved successful in patients with disorders such as fetal and neonatal alloimmune thrombocytopenia (FNAIT). Findings from 3 case studies of romiplostim-treated neonates with thrombocytopenia were reported recently in Pediatric Blood & Cancer.
Machine Learning May Lead to Personalized Medicine in MF

3 days ago | rarediseaseadvisor.com | Ozge Ozkaya

Machine learning can improve the risk stratification of patients with myelofibrosis (MF) who are being treated with allo-hematopoietic stem cell transplantation, according to a new study published in the scientific journal Blood.