Happy spring! March marks the final installment of JADPRO’s Benign Hematology Updates program. In addition to coverage of several presentations from the 63rd American Society of Hematology (ASH) annual meeting, this month we offer important research updates and online educational tools not only in our News & Literature section but also in the Resources section.

The updates for the Autoimmune Cytopenias pillar include reports on 2 presentations from ASH: the TARGET 020 trial, which evaluated the benefit of adding tacrolimus to high-dose dexamethasone in first-line treatment of adult immune thrombocytopenia (ITP); and a study in which addition of all-trans retinoic acid to low-dose rituximab significantly increased overall and sustained responses in corticosteroid-resistant or relapsed adult patients with ITP.

In the Bone Marrow Failure Disorders section is a report on a multicenter phase 3 trial published in the New England Journal of Medicine, in which investigators found eltrombopag added to standard immunosuppressive therapy in previously untreated patients with severe aplastic anemia significantly improved the hematologic response. In a report relevant to the care of patients with paroxysmal nocturnal hemoglobinuria (PNH), researchers developed and evaluated a novel composite endpoint to assess the impact of complement inhibition treatment in future clinical trials.

The Hemoglobinopathies pillar includes highlights from a report published in the New England Journal of Medicine on key results from a multicenter, international phase 3 study of betibeglogene autotemcel gene therapy, or beti-cell; this treatment achieved high rates of transfusion independence in both pediatric and adult patients with non–β⁰/β⁰ genotype β-thalassemia, with no observed cases of cancer.

The Hereditary/Acquired Bleeding Disorders pillar features reports on M'HEMORRH-AGE, a prospective, observational, multicenter study that evaluated exposure of older community-dwelling patients with hemophilia or von Willebrand Disease (VWD) to potentially inappropriate medications and treatments with anticholinergic and sedative properties; a systematic literature review by the SYMPHONY Consortium of patient-reported outcomes in patients with hemophilia and VWD; and comprehensive online patient brochures on VWD and mild hemophilia.

As always, I would be delighted to hear your thoughts about the latest content and/or any comments you may have regarding the overall structure and scope of Benign Hematology Updates. I also want to thank the program faculty for so generously sharing their time and clinical expertise in development of the many videos and case studies that truly enriched this special JADPRO program.

Be well!

Greetings! This month, JADPRO’s Benign Hematology Updates program continues to highlight relevant research from the December meeting of the American Society of Hematology (ASH). In addition, we have new additions to the Resources section.

Highlighted reports from ASH in the Autoimmune Cytopenias pillar include a phase 1b study of the potent Syk inhibitor HMPL-523 in adults with relapsed/refractory immune thrombocytopenia (ITP), which yielded promising response rates and will be evaluated further; and a machine-learning model that utilizes gut microbiota and function signatures to predict treatment response and relapse in ITP.

New ASH reports in the Bone Marrow Failure Disorders section focus on uncovering genetic and molecular factors possibly impacting treatment response and outcomes in aplastic anemia (AA) and paroxysmal nocturnal hemoglobinuria (PNH). Using genotyping, a large international study uncovered multiple modes of molecular immune escape in patients with idiopathic AA or PNH. A retrospective cohort study of over 650 patients with severe AA evaluated mutations associated with higher risk of clonal evolution post treatment.  

The Hemoglobinopathies pillar includes an ASH report on the randomized phase 2 BEYOND trial in non–transfusion-dependent beta thalassemia, showing the recombinant fusion protein luspatercept improved quality of life and reduced the red blood cell transfusion burden. In conjunction with the meeting, ASH issued a news release in support of the Sickle Cell Disease Comprehensive Care Act (H.R. 6216), a bill introduced in December 2021 with assistance from ASH.

Hereditary/Acquired Bleeding Disorders updates from ASH feature a retrospective observational study evaluating major bleeding in patients with von Willebrand disease (VWD) receiving anticoagulation or antiplatelet therapy for atrial fibrillation. In hemophilia news from ASH, investigators of a large observational study using the National Inpatient Sample database concluded that chronic infection with hepatitis C virus "remains a significant risk factor of [hepatocellular carcinoma] in hemophilia despite availability of [direct-acting antiviral] therapy."

As we look ahead to the longer and brighter days of spring, I hope you will find our latest research updates and resources enlightening. I welcome your thoughts and feedback.

I hope the start of the new year finds everyone well. This month, new content for JADPRO’s Benign Hematology Updates program highlights coverage of research presented at the recent annual meeting of the American Society of Hematology (ASH) on immune thrombocytopenia, paroxysmal nocturnal hemoglobinuria, sickle cell disease, beta-thalassemia, hemophilia, and von Willebrand disease.

Also featured in the January updates are intriguing new case studies for each of the four program pillars.

For the Autoimmune Cytopenias pillar, David Hughes and Frances Blevins discuss a clinical conundrum of balancing therapeutic anticoagulation with management of chronic immune thrombocytopenia (ITP) and highlight the importance of closely monitoring these patients.

Natasha Johnson's case study for Bone Marrow Failure Disorders illuminates the differential diagnosis and management of a 77-year-old man presumptively diagnosed with ITP who was ultimately found to have aplastic anemia.

The case study for Hemoglobinopathies, by Susan Carson, focuses on the hidden dangers of adrenal insufficiency and iron overload when managing infections in patients with thalassemia.

Jennifer Donkin presents a double case study for the Hereditary/Acquired Bleeding Disorders pillar, discussing management of postpartum bleeding in a 22-year-old hemophilia A carrier with low factor VIII levels, and of intracranial hemorrhage in her newborn son, who was diagnosed with severe hemophilia A.

I encourage you to take a look at the case studies and the research summaries from ASH offered in the current updates. Additional reports from ASH will be included in the February program updates as well, so please stay tuned!

As 2021 draws to a close and we look to the year ahead, many of our December program updates offer the promise of new understanding of the features, pathophysiology, and treatment of a range of benign hematologic disorders.

The Autoimmune Cytopenias pillar highlights a retrospective study of thromboembolic complications in autoimmune hemolytic anemia (AIHA) to discern whether thromboembolic events may be related to complement activation. A separate review in the New England Journal of Medicine discusses immunosuppression and newer treatments for AIHA. In a large meta-analysis of randomized controlled trials, researchers found targeted therapies for immune thrombocytopenia (ITP) were as safe as placebo but conferred lower risks of both major and minor bleeding plus a trend towards lower mortality rates. A timely literature review by researchers from Montefiore Medical Center/Albert Einstein College of Medicine in New York City highlights current literature on the clinical course of autoimmune cytopenias in patients with COVID-19.

Bone Marrow Failure Disorders includes updates from the ECU-PNH-III study, a randomized multicenter phase 3 clinical trial comparing eculizumab with an eculizumab biosimilar; a review of novel treatment strategies for paroxysmal nocturnal hemoglobinuria (PNH); a short video that explains hemolytic processes occurring in PNH and highlights PNH management with C5 inhibitors; and "clinical snapshots" from Memorial Sloan Kettering Cancer Center that provide insight into Fanconi anemia and several other inherited bone marrow failure syndromes.

The latest updates for the Hemoglobinopathies pillar include a retrospective cohort study from Amsterdam evaluating delayed hemolytic transfusion reaction in sickle cell disease (SCD); a study from Mumbai investigating genetic modifiers associated with higher fetal hemoglobin (HbF) production in β-thalassemia and SCD; a comprehensive review of SCD research by investigators from the Children's National Medical Center and the Sickle Cell Branch of the National Heart, Lung and Blood Institute (NHLBI), including genetic therapies emerging from improved understanding of mechanisms underlying the transition from HbF to adult Hb; and a 1-hour webinar from NHLBI describing ongoing bone marrow transplant clinical trials in SCD.

Hereditary/Acquired Bleeding Disorders updates present a retrospective analysis that breaks down healthcare utilization and costs for patients with von Willebrand disease (VWD) undergoing surgery; a report of subdural hemorrhage due to acquired von Willebrand syndrome in a female patient with polycythemia vera; and 2 guidelines from the National Hemophilia Foundation's Medical and Scientific Advisory Council.

In January, please look for coverage of important studies presented at the American Society of Hematology annual meeting, in addition to useful new resources and updates from the medical literature. Until then, I hope this month's content piques your interest.

Best wishes to all for a happy, healthy 2022!

Our November program updates include recent interesting reviews, case studies, and multimedia resources.

The Autoimmune Cytopenias pillar features a discussion of novel therapies for immune thrombocytopenia (ITP), a case study highlighting an emerging association of ITP with celiac disease, a webinar on ITP management from the Foundation for Women & Girls With Blood Disorders, and oral healthcare recommendations for patients with ITP.

Bone Marrow Failure Disorders updates include a review of inhibitors of complement C3, C5, factor B, and factor D, as well as several novel agents for patients with paroxysmal nocturnal hemoglobinuria (PNH); a new PNH Symptom Questionnaire (PNH-SQ) that may be useful in clinical trials; and short, informative animated videos on PNH and pyruvate kinase deficiency.

Updates for Hemoglobinopathies include a discussion of the role of red blood cell (RBC) oxidative stress in sickle cell disease (SCD) and its pathologic implications; analysis of BCL11A missense variants that may uncover new therapies for fetal hemoglobin silencing to treat SCD and beta-thalassemia; pearls for management of a variety of SCD complications; and a 1-hour webinar from the National Heart, Lung, and Blood Institute on genetic therapies in SCD.

Hereditary/Acquired Bleeding Disorders includes findings from PERCEPT 3, a trial of eptacog beta (human recombinant factor VIIa) in perioperative management of patients with hemophilia A or B with inhibitors; a review of perioperative management in hemophilia A and B; and current strategies for diagnosis and management of acquired hemophilia and acquired von Willebrand disease. In addition, a European study indicates development of thrombosis in COVID-19 is associated with a high ratio of von Willebrand factor:ADAMTS13, and discusses the therapeutic implications.

In November, many of us look forward to the 63^rd annual meeting of the American Society of Hematology (ASH), to be held in person in Atlanta and virtually from December 11 to 14. In addition to the many abstracts relevant to benign hematologic disorders are selected educational program presentations.

These include newer approaches to SCD care, such as transfusion management and gene therapies; novel treatments for alpha-thalassemia major and beta-thalassemia; use of factor concentrates and novel therapeutics for hemophilia A and B; case-based discussions of inherited RBC disorders besides hemoglobinopathies; evidence-based treatment approaches for common clotting and bleeding conundrums; management of inherited bleeding disorders in pregnant and elderly patients; and clinical and laboratory evaluation of patients with suspected bone marrow failure disorders.

We anticipate that future Updates will cover some of the exciting developments from ASH 2021 in the area of nonmalignant hematology. In the meantime, do check out our latest content.

Be well, and happy Thanksgiving!

I hope all are well and enjoying the cooler days of autumn. For some, bright red and yellow foliage makes the new season obvious; for others, October brings more subtle changes.

Many benign hematologic disorders can be medically complex and challenging, with diagnosis made through multistep testing and a careful process of elimination. Investigating clinical findings, often subtle, and patient-reported symptoms to detect rare disorders presents a unique challenge to the AP in hematology and oncology.

This month, JADPRO’s Benign Hematology Updates program is complemented by JADPRO Live Virtual 2021. The theme is "New Paths to Patient Care," and important insights are shared both within and outside of benign hematology. In late October, the Lymphoma, Leukemia & Myeloma Congress, also virtual, offers a preconference workshop on nonmalignant hematology that covers current treatments for thrombocytopenia, the latest updates on hemophilia and von Willebrand disease, gene therapy for inherited anemias, and management considerations for sickle cell disease and thalassemia.

The Updates program this month features case studies highlighting approaches to diagnosis and treatment pearls for hematologic disorders within each of the four program pillars.

For the Autoimmune Cytopenias pillar, David Hughes and Connor Flynn illustrate best practices for selecting thrombopoietin agonists in chronic immune thrombocytopenia; the patient, a 40-year-old male, has a past medical history significant for type II diabetes mellitus, hyperlipidemia, and untreated HIV.

Natasha Johnson, in her case study for Bone Marrow Failure Disorders, highlights identification and management of pyruvate kinase deficiency, emphasizing the importance of chelation therapy to protect against the detrimental effects of iron overload.

The case study for Hemoglobinopathies, by Susan Carson, presents a 21-year-old man with sickle cell disease (SCD) and right upper quadrant pain. As she emphasizes, one should be mindful that SCD-associated pain can have multiple underlying causes beyond vasoocclusive crisis.

Jennifer Donkin, in the Hereditary/Acquired Bleeding Disorders pillar, presents a case study of thrombosis management in a 26-year-old woman with antiphospholipid antibody syndrome.

There is much to consider this month. I encourage you to peruse the latest content, and I hope you find the updates engaging!

As many return to school this fall, we are reminded that as health care providers, we are challenged to engage in life-long learning to assimilate the constant advances in science and the resulting changes in recommendations for the diagnosis and management of illnesses relevant to our clinical practice, as well as disorders that may provide valuable insights into patient issues outside our scope of care.

JADPRO’s benign hematology program this month features compelling short videos in which faculty pair with a colleague or invited guest to highlight key concepts, current treatment, supportive care decisions, and patient education in the management of selected blood disorders.

For the Autoimmune Cytopenias pillar, David Hughes and Frances Blevins discuss in a video the current initial treatment and long-term management of chronic immune thrombocytopenia (ITP), along with dosing considerations and adverse drug events related to specific agents. Don’t miss David’s presentation on ITP at JADPRO Live Virtual, starting this October 7!

Natasha Johnson and Sara Tinsley describe the case of a 22-year-old male patient in their Bone Marrow Failure Disorders video, in their review of diagnosis and management of benign paroxysmal nocturnal hemoglobinuria (PNH). As Dr. Tinsley explains, PNH should be considered in younger patients with Coombs-negative hemolytic anemia and/or an unprovoked thrombosis.

The Hemoglobinopathies pillar video by Susan Carson and Anne Nord reviews the pathophysiology of sickle cell disease and its four subtypes, highlighting ways to address its acute and chronic complications and smooth the transition into adult care.

Jennifer Donkin, in the Hereditary/Acquired Bleeding Disorders video, interviews Michelle Witkop, Head of Research at the National Hemophilia Foundation (NHF). Dr. Witkop provides an overview of the NHF’s work; the Foundation’s resources for patients and advanced practitioners; and important trends in hemophilia research.

I hope you continue to find areas where these updates complement your ongoing education, and I welcome any comments you may have on the latest videos, news reports, and resources.

This month, we are featuring exclusive video discussions in which faculty from each of the Class Categories partner with a colleague to highlight challenges and clinical pearls in the management of a specific blood disorder.

David Hughes and Frances Blevins review essential features of chronic immune thrombocytopenia (ITP), including common presenting symptoms, etiologies, and options for treatment in a video for the Autoimmune Cytopenias pillar.

In the Bone Marrow Failure Disorders video, Natasha Johnson and Sara Tinsley discuss the management of a 33-year-old male patient with no significant past medical history who presented with dyspnea, fatigue, easy bruising; he was found to be pancytopenic and was diagnosed with aplastic anemia.

The video for the Hemoglobinopathies pillar delves into beta-thalassemia in adult patients, with Susan Carson and Anne Nord providing important patient care tips, guidelines for referral, and strategies for management of iron overload.

Jennifer Donkin interviews her colleague Jacquelyn Keegan about essential tools and strategies for the care of women with bleeding disorders in a video for the Hereditary/Acquired Bleeding Disorders pillar. Ms. Keegan describes patient evaluation, education, and treatment in a specialty clinic for patients presenting with heavy menstrual bleeding, common in women with von Willebrand disease.

Besides the videos, we have of course posted more resources, updates from the medical literature, and news—including coverage of the International Society for Thrombosis and Haemostasis 2021 Congress, hosted from Philadelphia and held virtually from July 17 to 21. 

Enjoy the rest of your summer!

We are pleased to offer more real-world case studies this month—one for each of the four Class Categories, to bring the content to life.

The Autoimmune Cytopenias case study highlights clinical pearls in the use of steroids and rituximab for warm autoimmune hemolytic anemia, plus tips for optimizing supportive care.

The Bone Marrow Failure Disorders case study describes development of a second bone marrow disorder in an older patient originally treated with anti-thymocyte globulin (ATG), cyclosporine (CSA), and eltrombopag for aplastic anemia (AA). Prior to receiving ATG/CSA, the patient had been passing dark urine in the morning and was experiencing severe joint and muscle pain. He was ultimately diagnosed with paroxysmal nocturnal hemoglobinuria (PNH). The pearl here is that PNH can occur in conjunction with other bone marrow disorders. While the PNH clone in AA may be near normal when first tested (due to the aplastic marrow), once normal hematopoiesis is restored following successful treatment of AA the clone can expand to cause some of the complications outlined in this case.

The case study for the Hemoglobinopathies pillar offers excellent tips for management of menorrhagia and secondary acute anemia in young women with sickle cell disease.

Finally, in Hereditary/Acquired Bleeding Disorders, the importance of multidisciplinary management is underscored in a discussion of compartment syndrome in a young man with severe hemophilia A. This case highlights the importance of comprehensive specialty care for patients with rare bleeding disorders.   

As always, please share this resource with your colleagues, and feel free to contact me with comments or suggestions. 

I hope everyone is enjoying the start of summer and perhaps even taking a break from busy work schedules. I hope you will take a few minutes to look at the newest content on Benign Hematology Updates.

Our Benign Hematology Basics 101 videos provide an overview of common and rare disorders.

The Autoimmune Cytopenias pillar updates this month include a report describing I-WISh, a large international survey of treatment perceptions related to immune thrombocytopenia (ITP), and a comprehensive review of novel emerging therapies for ITP and CLL-associated autoimmune hemolytic anemia.

The Bone Marrow Failure Disorders pillar offers information about a mechanism underlying the pathogenesis of paroxysmal nocturnal hemoglobinuria (PNH); Marrowforums, a discussion and resource center for BMF patients and their healthcare providers; and the recent approval of the complement C3 inhibitor pegcetacoplan for treatment of PNH.

The Hemoglobinopathies pillar includes guidance on testing and follow-up to support early detection of hemoglobin disorders; and a study using a lifetime Markov model to assess the cost-effectiveness of a hypothetical cell or gene-therapy cure for sickle cell disease.

In Hereditary/Acquired Bleeding Disorders, recommendations to optimize testing for VWD during times of acute heavy menstrual bleeding and development of a risk score to predict high bleeding risk in low VWF patients are highlighted.

It’s an exciting time for patients with benign hematologic disorders, as new information about potential pathways and targets for treatment continues to emerge. Of course, connections to COVID-19 and hematologic malignancies still thread through the conversation. Please dive into the Benign Hematology Updates website. We will be adding new case studies and videos soon, so stay tuned!

Greetings! I hope you’ve had an opportunity to explore JADPRO’s Benign Hematology Updates website, which launched in April. The site offers clinical pearls and learning opportunities in several formats, including important news and studies in the medical literature; useful resources and guidelines; and case studies and Fast Facts sheets that provide clinical snapshots of an array of benign hematologic disorders, with tips for management.

This month, we’re pleased to add video interviews that I conducted with the expert faculty editors for each of the four Class Categories: Jennifer Donkin offers “day in the life” insights into her work and resources for management of hemophilia and von Willebrand disease from childhood through adulthood; David Hughes and Frances Blevins discuss their collaboration in managing immune thrombocytopenia; Natasha Johnson highlights best practices in the management of paroxysmal nocturnal hemoglobinuria, or PNH; and Susan Carson focuses on what the advanced practitioner should keep front-of-mind when caring for patients with beta-thalassemia. A common theme in these videos is multidisciplinary management and coordination of care for patients with these often-chronic but rare disorders. 

As I noted in my welcome video, many of the disorders covered in this program are rare and less likely to be encountered in your clinical practice. However, I believe that information centered on the expanding and evolving management of benign hematologic disorders provides the advanced practitioner with myriad benefits—perhaps most notably a deepened understanding of how these disorders intersect with patient education and supportive care; malignant transformation; and, as we are learning, COVID-19.

Selected content is refreshed monthly, so please visit the Benign Hematology Updates website often and share the site with your colleagues. Take a moment to subscribe to the Journal of the Advanced Practitioner in Oncology (JADPRO) and join the Advanced Practitioner Society for Hematology and Oncology (APSHO) to get access to even more resources created expressly for advanced practitioners in oncology.

Greetings, and welcome to JADPRO Benign Hematology Updates. I hope you will explore and engage with the full array of resources that we’ve developed for our advanced practitioner (AP) audience on management approaches to a broad range of disease states in the evolving field of benign blood disorders. APs play a critical role in the multidisciplinary care of these conditions—which can be inherited or acquired; chronic and mild or acute and life-threatening; are often rare; and may be challenging to diagnose. Comprehensive differential diagnosis is critical since the patients’ symptoms may be incorrectly attributed to inconsequential causes. These often-complex disorders may occur in conjunction with or as a result of cancer, and in some cases may transform into malignancies.

Complicating today’s management of benign hematologic disorders, of course, is the challenge of COVID-19, declared as a pandemic by the World Health Organization just a year ago, in March 2020. As we have learned, COVID-19 can manifest with acute vascular effects such as micro-occlusions, and “long haul” COVID survivors may experience key symptoms (such as chronic fatigue) that often manifest in our patients with blood disorders. Ongoing research and our own experience have shown that patients with inherited or acquired benign hematologic disorders are at increased risk of severe COVID-19. The impact of this emerging clinical challenge, which will likely continue in the near future even with ongoing rollout and uptake of the SARS-CoV-2 vaccine, will be explored in the current JADPRO Benign Hematology Updates program, which is planned to run through March 2022, with content refreshed regularly.

This comprehensive program is organized into four broad topic areas, each guided by a dedicated specialist or specialist team: Hereditary/Acquired Bleeding Disorders, led by Jennifer Donkin, PNP, DNP, from the Children’s Center for Cancer and Blood Diseases, Children’s Hospital Los Angeles; Autoimmune Cytopenias, led by David Hughes, PharmD, BCOP, and Frances Blevins, PA-C, from Boston Medical Center; Hemoglobinopathies, led by Susan Carson, RN, MSN, CPNP, from the Children’s Center for Cancer and Blood Diseases, Children’s Hospital Los Angeles; and Bone Marrow Failure Disorders, led by Natasha Johnson, MSN, APRN, AOCNP, from Moffitt Cancer Center.

This microsite offers a selection of important clinical studies and best-practice resources; my comprehensive Benign Hematology Basics presentation; Fast Facts, with essential information on current diagnosis, treatment, supportive care, and prognosis of selected benign blood disorders; case studies from practitioners working in a variety of clinical settings; and video discussions in which expert APs share their tips and perspectives on challenging aspects of care. The program faculty and I look forward to sharing our years of experience and practice pearls with you.

Learn more about our program launch, as well as other planned content for this special series, in my welcome video.

I look forward to sharing new content with you on a monthly basis, as we develop this expanding resource further. Please feel free to contact me via editor@advancedpractitioner.com with your comments and questions.