January 23rd 2024
Momelotinib was given category 2A and 2B status for patients with high- and low-risk myelofibrosis (MF) and MF with anemia. However, ruxolitinib retains a higher category of recommendation as a treatment for patients with MF.
December 26th 2023
Research Offers Clues Into T Cell Differentiation in Worsening of Acute Gut GVHD
August 1st 2020Previous research has established acute graft-versus-host disease (GVHD) in the gastrointestinal tract as the primary cause of both GVHD severity and mortality, and as a result, emphasis is placed on preventing acute gut GVHD while still preserving the graft-versus-leukemia effect of allogeneic hematopoietic stem cell transplantation.
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Supplemental Process Can Aid in Assessing Value of Rare Disease Treatments
July 25th 2020A study looking at the ways in which different countries handle appraisal and reimbursement processes for rare disease treatments (RDTs) revealed that implementing supplemental processes can aid in patient and physician decision making.
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Ruxolitinib Improves ORR Over Best Available Therapy in Patients With Chronic GVHD
July 24th 2020A phase 3 study of ruxolitinib (Jakafi) has met its primary end point of superior overall response rate (ORR) compared with the best available therapy in patients with moderate or severe steroid-refractory or steroid dependent chronic graft-versus-host disease (GVHD).
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Therapy Provides Readily Available Stem Cell Source to Treat Rare Genetic Diseases
July 22nd 2020Umbilical cord blood provides a readily available source of stem cells to provide better access to hematopoietic stem cell transplantation to safely and effectively treat various noncancerous genetic disorders in children.
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Review Calls For Clear Guidance to Ensure Patient Centricity in Orphan Drug Trials
July 12th 2020Researchers called for new guidance to address the underreporting of patient engagement in rare disease clinical trials after finding a dismal amount of patient-reported outcome measures listed in orphan drug labeling claims.
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FDA Approves At-Home Therapy to Treat Myelodysplastic Syndromes
July 11th 2020A new oral therapy approved by the FDA will provide patients with myelodysplastic syndromes, who typically visit a health care facility for intravenous treatment, with an oral at-home option that can be beneficial during the coronavirus pandemic.
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Authors Call for More Research on Rare Blood Cancers to Fill Knowledge Gaps
July 4th 2020Authors hope that identifying knowledge gaps surrounding rare blood cancers, such as essential thrombocythemia and polycythemia vera, will inspire the science community to invest in new comprehensive research on how to properly diagnose and treat patients.
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Ruxolitinib Significantly Improves ORR in Acute GVHD Compared With Best Available Therapy
June 30th 2020Patients with steroid refractory acute graft-versus-host-disease (GVHD) have a better overall response rate (ORR) when treated with ruxolitinib compared with the best available therapy, according to results from a phase 3 trial presented at the European Hematology Association’s annual meeting.
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How Is Machine Learning Being Used to Improve Treatment of Rare Diseases?
June 26th 2020There are thousands of rare diseases, but they each affect so few patients that diagnosis and treatment can be delayed by years. Researchers examined how machine learning has been studied in rare diseases and identified areas of opportunity where future studies can improve care and treatment of rare diseases.
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Combination Therapy More Effective Than Standard Treatment for Rare Blood Disease
June 16th 2020A study conducted by Mayo Clinic researchers revealed that a combined treatment featuring daratumumab in conjunction with the standard-of-care for patients with amyloid light-chain (AL) amyloidosis was more effective in improving the condition than the standard treatment alone.
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Orphan Drug Reimbursements Create Tough Situation for South Korean Public Budget
June 12th 2020A data analysis of real-world data found that the rise of orphan drugs (ODs) in the Republic of Korea has not solved all patient accessibility issues as budget concerns can make it difficult to justify reimbursements.
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Test Determines Whether Children Will Respond to Rare Disease Treatment
June 4th 2020Researchers created a test to determine which children with Carbamoyl-Phosphate Synthetase 2, Aspartate Transcarbamylase, And Dihydroorotase (CAD) gene deficiency will benefit from receiving a nutritional supplement previously deemed effective in children with the condition, according to a study published in Genetics in Medicine.
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