Peers and Perspectives
Rare Disease Report
Rare Disease Report
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60th ASH Annual Meeting & Exposition
Mohamad Mohty, MD, PhD, Discusses Cytotoxic T Lymphocytes as Potential EBVPTLD Therapy
Mohamad Mohty, MD, PhD, discusses the investigational therapy cytotoxic T lymphocytes (CTLs) therapy for the treatment of Epstein-Barr virus associated post-transplant lymphoproliferative disorder (EBVPTLD).
Peter Turecek, PhD, Explains How Medical Devices May Benefit Patients with Hemophilia
An early prophylaxis in combination with an assistant device like myPKFit can improve the outcomes of treatment.
Ruxolitinib Therapy Benefits Patients with Chronic Neutrophilic Leukemia
This rare leukemia form may be treated with ruxolitinib, which appears to be well-tolerated by patients.
Hydroxyurea Therapy Found Effective for Myeloproliferative Neoplasms
Patients with myeloproliferative neoplasms (MPN) with essential thrombocythemia (ET) or polycythemia vera (PV) were found to benefit from hydroxyurea therapy.
Pemigatinib Shows Promising Efficacy as Myeloid/Lymphoid Neoplasms Treatment
Patients with myeloid/lymphoid neoplasms enrolled in the phase 2 clinical trial demonstrated pemigatinib was generally well-tolerated.
Nizar Bahlis, MD, Recaps Follow-Up Data on Combo Therapy for R/R Multiple Myeloma
Nizar Bahlis, MD, highlights the phase 3 POLLUX trial evaluating daratumumab plus lenalidomide and dexamethasone for the treatment of patients with relapsed/refractory multiple myeloma.
Potential Paroxysmal Nocturnal Hemoglobinuria Treatment, APL-2, Shows Positive Trial Update
Among the highlights, significant and sustained increases in hemoglobin without transfusions stood out.
John Leonard, MD, Reviews Investigational R/R Follicular Lymphoma Treatment
Lead investigator, John Leonard, MD, reviews the phase 3 AUGMENT trial investigating lenalidomide plus rituximab for the treatment of relapsed/refractory follicular lymphoma.
Patients with Sickle Cell Disease Benefit Most from Interventions That Reduce Hemolytic Anemia
Models show that for every gram of hemoglobin increased per deciliter, the risks associated with hemolytic anemia decrease.
Jorge Cortes, MD, Reviews Positive Phase 3 Quizartinib Data for AML Treatment
Jorge Cortes, MD, highlights the phase 3 QuANTUM-R trial results and explains their clinical implications for patients with
-ITD-mutated relapsed/refractory acute myeloid leukemia (AML).
Addressing Underreported and Untreated Bleeds in Hemophilia A Patients
Patients’ daily bleed diaries helped investigators understand why some bleeds are treated, and some are not, as well as their causes.
Jasmine Zain, MD, Highlights Current & Upcoming T-Cell Lymphoma Treatments
Jasmine Zain, MD, recaps the current and upcoming treatments in T-cell lymphoma and highlights the key clinical trials playing a role in the rare disease.
Michael Jordan, MD, Differentiates Emapalumab Treatment in Pediatric Patients Versus Adults with HLH
The pediatrician discusses key differences between treating pediatric and adult patients with hemophagocytic lymphohistiocytosis (HLH) with the recently FDA-approved emapalumab.
Novel Gene Therapy Promising for Sickle Cell Disease
The therapy attempts to restore fetal hemoglobin production.
Ravulizumab Noninferior to Eculizumab for Paroxysmal Nocturnal Hemoglobinuria
Ravulizumab dosed every 8 weeks was noninferior to eculizumab given every 2 weeks, but the treatment did not achieve superiority.
Investigational Multiple Myeloma Therapy, Ixazomib, Yields Positive Results in Phase 3 Trial
Among the highlights observed in the data, the single-agent oral demonstrated significant improvement in progression-free survival (PFS).
CLL Therapy, Venetoclax/Rituximab, Shows Reduced Risk of Disease Progression
Full results from the phase 3 MURANO trial showed a reduced risk in disease progression and 87.9% overall survival in relapsed/refractory chronic lymphocytic leukemia patients treated with venetoclax/rituximab.
Anemia Patients with MDS Benefit from Luspatercept in Transfusion Burden
Currently available anemia therapies only provide benefit for about half of patients with a lower-risk myelodysplastic syndromes (MDS)-related condition—and about one-quarter of such patients dependent on red blood cell transfusions.
Germ Cell Tumors Have the Potential to Become Leukemia
Justin Taylor, MD, explains new and unique data that show cases in which a solid tumor can become a hematologic malignancy, or leukemia.
Males with MPN Have Inferior Survival Compared to Females
Males with myeloproliferative neoplasms (MPN) were found to have inferior survival compared to females in a well-defined, prospective, observational cohort.
Luspatercept Relieves Transfusion Burden in Beta-Thalassemia Patients
The odds ratio of patients on the therapy reaching ≥33% RBC transfusion burden reduction was 5 times greater than those on placebo.
Familial Haploidentical Stem Cell Transplantation Improves Quality of Life & Neurocognition in Sickl
Long-term data shows familial haploidentical stem cell transplantation (HISCT) improves quality of life and neurocognition in sickle cell disease patients.
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