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Accreditation/Credit Designation

Physicians' Education Resource®, LLC, is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.

Physicians' Education Resource®, LLC, designates this enduring material for a maximum of 1.0 AMA PRA Category 1 Credit™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Acknowledgment of Commercial Support

This activity is supported by an educational grant from Agios Pharmaceuticals.

Hematology Briefings™: Advancing Care and Improving Outcomes for Patients With Pyruvate Kinase Deficiency

Release Date: October 31, 2017
Expiration Date: October 31, 2018
Media: Internet - based

 

Activity Overview

The Hematology Briefings™ is an online interactive monograph that will include a review of recent data regarding treatments in patients with pyruvate kinase (PK) deficiency, designed to aid physicians in assessing and interpreting emerging data and novel therapeutic strategies, and to apply those findings to their practices. Commentary by a thought leader, key take-home points, and clinical pearls for practice will place the content into perspective. Sidebars and tables will provide supporting evidence.

Acknowledgment of Commercial Support

This activity is supported by an educational grant from Agios Pharmaceuticals.

CME Activity Table of Contents

  • Background and Disease Biology
  • Diagnosis of PK Deficiency
  • Supportive Treatment Options for PK Deficiency
  • Emerging Treatment Options for PK Deficiency

Instructions for This Activity and Receiving Credit

  • You will need to log in to participate in the activity.
  • Each presentation may contain an interactive question(s). You may move forward through the presentation; however, you may not go back to change answers or review audio files/content until you finish the presentation.
  • At the end of the activity, "Educational Content/Audio Files" will be available for your reference.
  • In order to receive a CME certificate, participants must complete the activity.
  • Complete the Posttest and pass with a score of 70% or higher, complete the Evaluation, and then click on “Request for Credit.” Participants may immediately download a CME certificate upon completion of these steps.

Target Audience

This educational initiative is directed toward hematologists who treat patients with nonmalignant hematologic disorders and patients with anemia. Nurse practitioners, nurses, physician assistants, pharmacists, researchers, fellows, and other healthcare professionals interested in the treatment of anemias also are invited to participate.

Learning Objectives

Upon completion of this activity, you should be better able to:

  • Describe the epidemiology and pathophysiology of PKD
  • Discuss signs and symptoms of PKD and diagnostic strategies for its management
  • Explain emerging clinical trial evidence concerning novel treatment strategies for PKD
  • Describe how to place potentially practice-changing evidence in the context of evolving treatment paradigms in the management of PKD

Faculty, Staff, and Planners' Disclosures

Faculty

Rachael Grace, MD
Director, Hematology Clinic
Assistant Professor of Pediatrics
Harvard Medical School
Boston, MA
 

Disclosure: Grant/Research Support: Agios Pharmaceuticals; Consultant: Agios Pharmaceuticals

The staff of Physicians' Education Resource®, LLC have no relevant financial relationships with commercial interests to disclose.

Disclosure Policy and Resolution of Conflicts of Interest (COI)

As a sponsor accredited by the ACCME, it is the policy of PER® to ensure fair balance, independence, objectivity, and scientific rigor in all of its CME activities. In compliance with ACCME guidelines, PER® requires everyone who is in a position to control the content of a CME activity to disclose all relevant financial relationships with commercial interests. The ACCME defines “relevant financial relationships” as financial relationships in any amount occurring within the past 12 months that creates a COI.

Additionally, PER® is required by ACCME to resolve all COI. PER® has identified and resolved all COI prior to the start of this activity by using a multistep process.

Off-Label Disclosure and Disclaimer

This CME activity may or may not discuss investigational, unapproved, or off-label use of drugs. Participants are advised to consult prescribing information for any products discussed. The information provided in this CME activity is for continuing medical and nursing education purposes only, and is not meant to substitute for the independent clinical judgment of a physician relative to diagnostic or treatment options for a specific patient’s medical condition. The opinions expressed in the content are solely those of the individual faculty members and do not reflect those of PER®.




PER Pulse™ Recap (1 of 3)
Understanding the Underlying Factors of Pyruvate Kinase Deficiency

The online Hematology Briefings CME activity, Advancing Care and Improving Outcomes for Patients With Pyruvate Kinase Deficiency, provides hematologists and other health care providers with engaging instruction on how to identify and diagnose pyruvate kinase (PK) deficiency in their patients and the current and evolving supportive treatment standards for this rare blood disorder. Leading expert, Rachael Grace, MD, director of the hematology clinic and assistant professor of pediatrics at Harvard Medical School, answers key questions, supported by the presentation of clinical science, about PK deficiency. This first of 3 PER Pulse™ Recaps from this program focuses on the distinguishing features of PK deficiency and methods of diagnostics.

Dr. Grace overviewed the pathophysiology of PK deficiency, including its molecular origins in glycolysis. Also discussed were the signs and symptoms commonly presented in patients with PK deficiency and the differences between enzyme- and DNA-based testing:

  • During one of the final steps in glycolysis, PK catalyzes the transphosphorylation of phosphoenolpyruvate to adenosine diphosphate, generating pyruvate and adenosine triphosphate (ATP). This transphosphorylation is the last ATP-generating reaction in glycolysis. PK is responsible for the generation of 50% of a red blood cells’ (RBCs’) total ATP.
  • Mature RBCs, which lack mitochondria, are completely dependent on glycolysis for the synthesis of ATP, as such PK deficiency readily impairs RBCs’ lifespan. PK deficiency is the most common glycolytic defect causing chronic no-spherocytic hemolytic anemia, estimated to be prevalent in approximately 1 in 20,000 individuals.
  • PK deficiency symptoms vary among individuals, and even within an individual, by their age, from the neonatal period to adulthood. The clinical manifestations are heterogeneous, and some of the more common symptoms include anemia, jaundice, gallstones, and iron overload. Other complications include extramedullary hematopoiesis, pulmonary hypertension, and osteopenia. All complications can occur at any age.
  • In patients with hemolytic anemia, PK deficiency should be considered after more common causes of hemolysis are ruled out. PK deficiency can be diagnosed by quantitative RBC enzyme activity testing. Gene testing for PKLR mutations have become more widely commercially available and can be used as confirmatory testing.

For additional information and commentary on this topic, as well as audio and supporting text, visit gotoper.com/online-cme-activities/oncology-briefing/hematology-briefing-advancing-care-and-improving-outcomes-for-patients-with-pyruvate-kinase-deficiency.

For information on other topics, visit gotoper.com.




PER Pulse™ Recap (2 of 3)
Supportive Care Options and Considerations for Patients With Pyruvate Kinase Deficiency

The online Hematology Briefings CME activity, Advancing Care and Improving Outcomes for Patients With Pyruvate Kinase Deficiency, provides hematologists and other health care providers with engaging instruction on how to identify and diagnose pyruvate kinase (PK) deficiency in their patients and the current and evolving supportive treatment standards in this rare blood disorder. Leading expert, Rachael Grace, MD, director of the hematology clinic and assistant professor of pediatrics at Harvard Medical School, answers key questions, supported by the presentation of clinical science, about PK deficiency. This second of 3 PER Pulse™ Recaps from this program focuses on the supportive care options for patients with PK deficiency.

Dr. Grace discussed the various treatment options for PK deficiency, which remain supportive rather than curative. Current supportive care includes blood transfusions, splenectomy, and chelation therapy to address iron overload. Special considerations should be made for women with PK deficiency who become pregnant:

  • Red blood cell (RBC) transfusions may be required in patients with symptomatic anemia, particularly in the first years of life. Iron overload is a predictable complication of chronic transfusion therapy in PK deficiency and is treated with iron chelation therapy. However, transfusion-independent iron loading is also common in PK deficiency and iron status should be monitored at least annually over the age continuum.
  • In patients with severe anemia, splenectomy is indicated for managing PK deficiency–associated anemia. As the spleen is a site of RBC removal, splenectomy is an effective means of increasing hemoglobin levels and decreasing transfusion needs in severe cases of PK deficiency.
  • Gallstones are a frequent complication in patients of all ages with PK deficiency due to ongoing hemolysis. Gallstones may present with abdominal pain or increased jaundice. Patients may have a cholecystectomy at the time of splenectomy or at the onset of symptoms.
  • Close monitoring and follow-up are needed for patients with PK deficiency while pregnant. Indications and guidelines for transfusions during pregnancy do not exist. If delivery is by cesarean section, this may increase the likelihood of patients with PK deficiency needing transfusions. Pregnant women with PK deficiency should be followed by a high-risk obstetrician who can partner with the patient’s hematologist.

For additional information and commentary on this topic, as well as audio and supporting text, visit gotoper.com/online-cme-activities/oncology-briefing/hematology-briefing-advancing-care-and-improving-outcomes-for-patients-with-pyruvate-kinase-deficiency.

For information on other topics, visit gotoper.com.




PER Pulse™ Recap (3 of 3)
Emerging Treatment Options for Pyruvate Kinase Deficiency

The online Hematology Briefings CME activity, Advancing Care and Improving Outcomes for Patients With Pyruvate Kinase Deficiency, provides hematologists and other health care providers with engaging instruction on how to identify and diagnose pyruvate kinase (PK) deficiency in your patients and the current and evolving supportive treatment standards in this rare blood disorder. Leading expert, Rachael Grace, MD, director of the hematology clinic and assistant professor of pediatrics at Harvard Medical School, answers key questions, supported by the presentation of clinical science, about PK deficiency. This third and final PER Pulse™ Recap from this program focuses on the emerging treatment options for PK deficiency and results from the DRIVE PK trial.

Dr. Grace summarized reported results of the ongoing phase II DRIVE PK trial investigating AG-348:

  • The DRIVE PK trial is in an open-label, multicenter, dose-ranging phase II trial in transfusion-independent adults with PK deficiency. Patients are randomized to receive AG-348 at a dose of 50 or 300 mg orally twice daily for 6 months. After the first 6 months, based on safety and efficacy, patients are given the choice to extend treatment for 2 years.
  • AG-348 is a novel small molecule activator of wild-type and mutant PK isoform PK-R. In an ex-vivo investigation into AG-348, incubation with the novel activator greatly increased PK activity and adenosine triphosphate levels.
  • As of January 2017, the trial had reached its goal enrolment of 52 patients. Of the 52 patients evaluated at the most recent update, 25 (48%) had achieved a maximal increase in hemoglobin >1 g/dL. The median time to a hemoglobin increase was 10 days.
  • The most commonly reported adverse events included headaches (44%), nausea (37%) and insomnia (39%).
  • Updated data from the DRIVE PK trial were presented at the 59th ASH Annual Meeting and Exposition

For additional information and commentary on this topic, as well as audio and supporting text, visit gotoper.com/online-cme-activities/oncology-briefing/hematology-briefing-advancing-care-and-improving-outcomes-for-patients-with-pyruvate-kinase-deficiency.

For information on other topics, visit gotoper.com.








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