Publications by authors named "M EFRAIM"

Immune thrombocytopenia (ITP) is an autoimmune disease associated with autoantibody-mediated platelet destruction and impaired platelet production, resulting in thrombocytopenia and a predisposition to bleeding. The ongoing, global phase 1/2 study showed that rilzabrutinib, a Bruton tyrosine kinase inhibitor specifically developed to treat autoimmune disorders, could be an efficacious and well-tolerated treatment for ITP. Clinical activity, durability of response, and safety were evaluated in 16 responding patients who continued rilzabrutinib 400 mg twice daily in the long-term extension (LTE) study.

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Article Synopsis
  • Autoimmune phenomena commonly complicate chronic lymphocytic leukemia (CLL), affecting 10% to 25% of patients, with rare cases like myasthenia gravis (MG) reported alongside CLL.
  • A case study is presented of a 63-year-old woman suffering from severe MG that is likely linked to her CLL relapse, and she was treated with a combination of targeted and immunotherapy.
  • The interaction of targeted therapies and monoclonal antibodies with the immune system is complex, highlighting the need to explore their effectiveness in treating autoimmune complications associated with CLL.
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Background: Rilzabrutinib, an oral, reversible covalent inhibitor of Bruton's tyrosine kinase, may increase platelet counts in patients with immune thrombocytopenia by means of dual mechanisms of action: decreased macrophage (Fcγ receptor)-mediated platelet destruction and reduced production of pathogenic autoantibodies.

Methods: In an international, adaptive, open-label, dose-finding, phase 1-2 clinical trial, we evaluated rilzabrutinib therapy in previously treated patients with immune thrombocytopenia. We used intrapatient dose escalation of oral rilzabrutinib over a period of 24 weeks; the lowest starting dose was 200 mg once daily, with higher starting doses of 400 mg once daily, 300 mg twice daily, and 400 mg twice daily.

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Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph + ALL) is an aggressive disease with poor outcomes. Despite the incorporation of tyrosine kinase inhibitors (TKIs) in the therapeutic strategies, patients who relapse after chemotherapy plus TKI have poor overall survival (OS) and less chance to proceed to hematopoietic stem cell transplantation (HSCT) which remains the only curative approach. Therefore, new drugs, such as antibody-targeted therapies alone or in combination with TKIs, offer new therapeutic options for those patients.

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The purpose of this study was to examine the effects of after-school sedentary screen time on children's brain activation in reward and cognitive control regions in response to pictures of high- and low-calorie foods. Thirty-two children participated in a randomized crossover study with counterbalanced treatment conditions. Conditions took place on separate days after school and included three hours of active or sedentary play.

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