Hematology Reports

7 pages, 422 KiB

Open AccessEditor’s ChoiceBrief Report

Risk Factors for Death or Cardiovascular Events after Acute Coronary Syndrome in Patients with Myeloproliferative Neoplasms

by Orly Leiva, Andrew Jenkins, Rachel P. Rosovsky, Rebecca K. Leaf, Katayoon Goodarzi and Gabriela Hobbs

Hematol. Rep. 2023, 15(2), 398-404; https://doi.org/10.3390/hematolrep15020040 - 7 Jun 2023

Cited by 6 | Viewed by 3695

Abstract

Patients with myeloproliferative neoplasms (MPNs) are at increased risk of cardiovascular disease (CVD), including acute coronary syndrome (ACS). However, data on long-term outcomes of patients with MPN who have had ACS and risk factors for all-cause death or CV events post-ACS hospitalization are [...] Read more.

Patients with myeloproliferative neoplasms (MPNs) are at increased risk of cardiovascular disease (CVD), including acute coronary syndrome (ACS). However, data on long-term outcomes of patients with MPN who have had ACS and risk factors for all-cause death or CV events post-ACS hospitalization are lacking. We conducted a single-center study of 41 consecutive patients with MPN with ACS hospitalization after MPN diagnosis. After a median follow-up of 80 months after ACS hospitalization, 31 (76%) experienced death or a CV event (myocardial infarction, ischemic stroke, or heart failure hospitalization). After multivariable Cox proportional hazards regression, index ACS within 12 months of MPN diagnosis (HR 3.84, 95% CI 1.44–10.19), WBC ≥ 20 K/µL (HR 9.10, 95% CI 2.71–30.52), JAK2 mutation (HR 3.71, 95% CI 1.22–11.22), and prior CVD (HR 2.60, 95% CI 1.12–6.08) were associated with increased death or CV events. Further studies are warranted to improve cardiovascular outcomes in this patient population. Full article

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14 pages, 333 KiB

Open AccessConference Report

Managing Relevant Clinical Conditions of Hemophilia A/B Patients

by Massimo Morfini, Jacopo Agnelli Giacchiello, Erminia Baldacci, Christian Carulli, Giancarlo Castaman, Anna Chiara Giuffrida, Giuseppe Malcangi, Angiola Rocino, Sergio Siragusa and Ezio Zanon

Hematol. Rep. 2023, 15(2), 384-397; https://doi.org/10.3390/hematolrep15020039 - 7 Jun 2023

Cited by 2 | Viewed by 2903

Abstract

The Medical Directors of nine Italian Hemophilia Centers reviewed and discussed the key issues concerning the replacement therapy of hemophilia patients during a one-day consensus conference held in Rome one year ago. Particular attention was paid to the replacement therapy needed for surgery [...] Read more.

The Medical Directors of nine Italian Hemophilia Centers reviewed and discussed the key issues concerning the replacement therapy of hemophilia patients during a one-day consensus conference held in Rome one year ago. Particular attention was paid to the replacement therapy needed for surgery using continuous infusion (CI) versus bolus injection (BI) of standard and extended half-life Factor VIII (FVIII) concentrates in severe hemophilia A patients. Among the side effects, the risk of development of neutralizing antibodies (inhibitors) and thromboembolic complications was addressed. The specific needs of mild hemophilia A patients were described, as well as the usage of bypassing agents to treat patients with high-responding inhibitors. Young hemophilia A patients may take significant advantages from primary prophylaxis three times or twice weekly, even with standard half-life (SHL) rFVIII concentrates. Patients affected by severe hemophilia B probably have a less severe clinical phenotype than severe hemophilia A patients, and in about 30% of cases may undergo weekly prophylaxis with an rFIX SHL concentrate. The prevalence of missense mutations in 55% of severe hemophilia B patients allows the synthesis of a partially changed FIX molecule that can play some hemostatic role at the level of endothelial cells or the subendothelial matrix. The flow back of infused rFIX from the extravascular to the plasma compartment allows a very long half-life of about 30 h in some hemophilia B patients. Once weekly, prophylaxis can assure a superior quality of life in a large severe or moderate hemophilia B population. According to the Italian registry of surgery, hemophilia B patients undergo joint replacement by arthroplasty less frequently than hemophilia A patients. Finally, the relationships between FVIII/IX genotypes and the pharmacokinetics of clotting factor concentrates have been investigated. Full article

(This article belongs to the Special Issue Personalized Therapy and Clinical Outcomes for Congenital and Acquired Haemorrhagic Disorders, Thromboembolic Disease and Platelet Disorders)

14 pages, 6746 KiB

Open AccessCase Report

Splenic Rupture Secondary to Amyloidosis: A Case Report and Review of the Literature

by Hisham F. Bahmad, Samantha Gogola, Lorena Burton, Ferial Alloush, Mike Cusnir, Michael Schwartz, Lydia Howard and Vathany Sriganeshan

Hematol. Rep. 2023, 15(2), 370-383; https://doi.org/10.3390/hematolrep15020038 - 6 Jun 2023

Cited by 1 | Viewed by 3390

Abstract

Amyloidosis is a term describing the extracellular deposit of fibrils composed of subunits of several different normal serum proteins in various tissues. Amyloid light chain (AL) amyloidosis contains fibrils that are composed of fragments of monoclonal light chains. Many different disorders and conditions [...] Read more.

Amyloidosis is a term describing the extracellular deposit of fibrils composed of subunits of several different normal serum proteins in various tissues. Amyloid light chain (AL) amyloidosis contains fibrils that are composed of fragments of monoclonal light chains. Many different disorders and conditions can lead to spontaneous splenic rupture, including AL amyloidosis. We present a case of a 64-year-old woman with spontaneous splenic rupture and hemorrhage. A final diagnosis of systemic amyloidosis secondary to plasma cell myeloma was made with infiltrative cardiomyopathy and possible diastolic congestive heart failure exacerbation. We also provide a narrative review of all documented cases of splenic rupture associated with amyloidosis from the year 2000 until January 2023, along with the main clinical findings and management strategies. Full article

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12 pages, 923 KiB

Open AccessEditor’s ChoiceReview

Role of Therapeutic Anticoagulation in COVID-19: The Current Situation

by Mandeep Singh Rahi, Jay Parekh, Prachi Pednekar, Mayuri Mudgal, Vishal Jindal and Kulothungan Gunasekaran

Hematol. Rep. 2023, 15(2), 358-369; https://doi.org/10.3390/hematolrep15020037 - 5 Jun 2023

Cited by 5 | Viewed by 2966

Abstract

Thrombotic complications from COVID-19 are now well known and contribute to significant morbidity and mortality. Different variants confer varying risks of thrombotic complications. Heparin has anti-inflammatory and antiviral effects. Due to its non-anticoagulant effects, escalated-dose anticoagulation, especially therapeutic-dose heparin, has been studied for [...] Read more.

Thrombotic complications from COVID-19 are now well known and contribute to significant morbidity and mortality. Different variants confer varying risks of thrombotic complications. Heparin has anti-inflammatory and antiviral effects. Due to its non-anticoagulant effects, escalated-dose anticoagulation, especially therapeutic-dose heparin, has been studied for thromboprophylaxis in hospitalized patients with COVID-19. Few randomized, controlled trials have examined the role of therapeutic anticoagulation in moderately to severely ill patients with COVID-19. Most of these patients had elevated D-dimers and low bleeding risks. Some trials used an innovative adaptive multiplatform with Bayesian analysis to answer this critical question promptly. All the trials were open-label and had several limitations. Most trials showed improvements in the meaningful clinical outcomes of organ-support-free days and reductions in thrombotic events, mainly in non-critically-ill COVID-19 patients. However, the mortality benefit needed to be more consistent. A recent meta-analysis confirmed the results. Multiple centers initially adopted intermediate-dose thromboprophylaxis, but the studies failed to show meaningful benefits. Given the new evidence, significant societies have suggested therapeutic anticoagulation in carefully selected patients who are moderately ill and do not require an intensive-care-unit level of care. There are multiple ongoing trials globally to further our understanding of therapeutic-dose thromboprophylaxis in hospitalized patients with COVID-19. In this review, we aim to summarize the current evidence regarding the use of anticoagulation in patients with COVID-19 infection. Full article

(This article belongs to the Special Issue Personalized Therapy and Clinical Outcomes for Congenital and Acquired Haemorrhagic Disorders, Thromboembolic Disease and Platelet Disorders)

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11 pages, 277 KiB

Open AccessEditor’s ChoiceArticle

Etiology of Anemia and Risk Factors of Mortality among Hospitalized Patients: A Real-Life Retrospective Study in a Tertiary Center in Greece

by Petros Ioannou, Andria Papazachariou, Maria Tsafaridou, Ioannis E. Koutroubakis and Diamantis P. Kofteridis

Hematol. Rep. 2023, 15(2), 347-357; https://doi.org/10.3390/hematolrep15020036 - 2 Jun 2023

Cited by 1 | Viewed by 2965

Abstract

Anemia is a prominent global health issue with a wide variety of causes and can be associated with decreased quality of life, increased hospitalization, and higher mortality, especially in older individuals. Therefore, studies further shedding light on the causes and the risk factors [...] Read more.

Anemia is a prominent global health issue with a wide variety of causes and can be associated with decreased quality of life, increased hospitalization, and higher mortality, especially in older individuals. Therefore, studies further shedding light on the causes and the risk factors of this condition should be performed. The aim of the present study was to examine the causes of anemia in hospitalized patients in a tertiary hospital in Greece and identify risk factors related to higher mortality. In total, 846 adult patients with a diagnosis of anemia were admitted during the study period. The median age was 81 years, and 44.8% were male. The majority of patients had microcytic anemia, with the median mean corpuscular volume (MCV) being 76.3 fL and the median hemoglobin being 7.1 g/dL. Antiplatelets were used by 28.6% of patients, while 28.4% were using anticoagulants at the time of diagnosis. At least one unit of packed red blood cells (PRBCs) was transfused in 84.6% of patients, and a median of two PRBCs was used per patient. A gastroscopy was performed in 55%, and a colonoscopy was performed in 39.8% of patients in the present cohort. Anemia was considered to be multifactorial in almost half the cases, while the most commonly identified cause was iron deficiency anemia, more commonly with positive endoscopic findings. Mortality was relatively low, at 4.1%. Multivariate logistic regression analysis identified higher B12 levels and longer duration of hospital stay to be independently positively associated with mortality. Full article

16 pages, 1443 KiB

Open AccessEditor’s ChoiceReview

Combination Therapies with Kinase Inhibitors for Acute Myeloid Leukemia Treatment

by Shinichiro Takahashi

Hematol. Rep. 2023, 15(2), 331-346; https://doi.org/10.3390/hematolrep15020035 - 24 May 2023

Cited by 9 | Viewed by 3145

Abstract

Targeting kinase activity is considered to be an attractive therapeutic strategy to overcome acute myeloid leukemia (AML) since aberrant activation of the kinase pathway plays a pivotal role in leukemogenesis through abnormal cell proliferation and differentiation block. Although clinical trials for kinase modulators [...] Read more.

Targeting kinase activity is considered to be an attractive therapeutic strategy to overcome acute myeloid leukemia (AML) since aberrant activation of the kinase pathway plays a pivotal role in leukemogenesis through abnormal cell proliferation and differentiation block. Although clinical trials for kinase modulators as single agents remain scarce, combination therapies are an area of therapeutic interest. In this review, the author summarizes attractive kinase pathways for therapeutic targets and the combination strategies for these pathways. Specifically, the review focuses on combination therapies targeting the FLT3 pathways, as well as PI3K/AKT/mTOR, CDK and CHK1 pathways. From a literature review, combination therapies with the kinase inhibitors appear more promising than monotherapies with individual agents. Therefore, the development of efficient combination therapies with kinase inhibitors may result in effective therapeutic strategies for AML. Full article

(This article belongs to the Topic Myeloma and Leukemia-Challenges and Current Treatment Options)

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6 pages, 345 KiB

Open AccessCase Report

Phenazopyridine-Induced Methemoglobinemia in a Jehovah’s Witness Treated with High-Dose Ascorbic Acid Due to Methylene Blue Contradictions: A Case Report and Review of the Literature

by Sasmith R. Menakuru, Vijaypal S. Dhillon, Mona Atta, Keeret Mann and Ahmed Salih

Hematol. Rep. 2023, 15(2), 325-330; https://doi.org/10.3390/hematolrep15020034 - 24 May 2023

Cited by 2 | Viewed by 2738

Abstract

Methemoglobinemia is an acute medical emergency that requires prompt correction. Physicians should have a high degree of suspicion of methemoglobinemia in cases that present with hypoxemia that does not resolve with supplemental oxygenation, and they should confirm this suspicion with a positive methemoglobin [...] Read more.

Methemoglobinemia is an acute medical emergency that requires prompt correction. Physicians should have a high degree of suspicion of methemoglobinemia in cases that present with hypoxemia that does not resolve with supplemental oxygenation, and they should confirm this suspicion with a positive methemoglobin concentration on arterial blood gas. There are multiple medications that can induce methemoglobinemia, such as local anesthetics, antimalarials, and dapsone. Phenazopyridine is an azo dye used over-the-counter as a urinary analgesic for women with urinary tract infections, and it has also been implicated in causing methemoglobinemia. The preferred treatment of methemoglobinemia is methylene blue, but its use is contraindicated for patients with glucose-6-phosphatase deficiency or those who take serotonergic drugs. Alternative treatments include high-dose ascorbic acid, exchange transfusion therapy, and hyperbaric oxygenation. The authors report a case of a 39-year-old female who took phenazopyridine for 2 weeks to treat dysuria from a urinary tract infection and subsequently developed methemoglobinemia. The patient had contraindications for the use of methylene blue and was therefore treated with high-dose ascorbic acid. The authors hope that this interesting case promotes further research into the utilization of high-dose ascorbic acid for managing methemoglobinemia in patients who are unable to receive methylene blue. Full article

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8 pages, 714 KiB

Open AccessCase Report

Additional Genetic Alterations and Clonal Evolution of MPNs with Double Mutations on the MPL Gene: Two Case Reports

by Maria Stella Pennisi, Sandra Di Gregorio, Elena Tirrò, Chiara Romano, Andrea Duminuco, Bruno Garibaldi, Gaetano Giuffrida, Livia Manzella, Paolo Vigneri and Giuseppe A. Palumbo

Hematol. Rep. 2023, 15(2), 317-324; https://doi.org/10.3390/hematolrep15020033 - 23 May 2023

Cited by 1 | Viewed by 1981

Abstract

Essential thrombocythemia (ET) and primary myelofibrosis (PMF) are two of the main BCR-ABL1-negative chronic myeloproliferative neoplasms (MPNs) characterized by abnormal megakaryocytic proliferation. Janus kinase 2 (JAK2) mutations are detected in 50–60% of ET and PMF, while myeloproliferative leukemia (MPL [...] Read more.

Essential thrombocythemia (ET) and primary myelofibrosis (PMF) are two of the main BCR-ABL1-negative chronic myeloproliferative neoplasms (MPNs) characterized by abnormal megakaryocytic proliferation. Janus kinase 2 (JAK2) mutations are detected in 50–60% of ET and PMF, while myeloproliferative leukemia (MPL) virus oncogene mutations are present in 3–5% of cases. While Sanger sequencing is a valuable diagnostic tool to discriminate the most common MPN mutations, next-generation sequencing (NGS) is a more sensitive technology that also identifies concurrent genetic alterations. In this report, we describe two MPN patients with simultaneous double MPL mutations: a woman with ET presenting both MPL^V501A-W515R and JAK2^V617F mutations and a man with PMF displaying an uncommon double MPL^V501A-W515L. Using colony-forming assays and NGS analyses, we define the origin and mutational landscape of these two unusual malignancies and uncover further gene alterations that may contribute to the pathogenesis of ET and PMF. Full article

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5 pages, 209 KiB

Open AccessCase Report

Acquired Factor X Deficiency without Amyloidosis Presenting with Massive Hematuria: A Case Report and Review of the Literature

by Sasmith R. Menakuru, Vijaypal S. Dhillon, Ahmed Salih and Amir F. Beirat

Hematol. Rep. 2023, 15(2), 312-316; https://doi.org/10.3390/hematolrep15020032 - 15 May 2023

Cited by 2 | Viewed by 2263

Abstract

Acquired factor X deficiency is a rare diagnosis, especially without the association of other co-existing conditions such as amyloidosis. The authors report the case of a 34-year-old male with severe frank hematuria found to have markedly prolonged prothrombin time and activated partial thromboplastin [...] Read more.

Acquired factor X deficiency is a rare diagnosis, especially without the association of other co-existing conditions such as amyloidosis. The authors report the case of a 34-year-old male with severe frank hematuria found to have markedly prolonged prothrombin time and activated partial thromboplastin time. A mixing study showed correction utilizing normal plasma and a coagulation panel testing revealed decreased factor X activity. The patient was treated with multiple blood transfusions, fresh frozen plasma, high-dose pulse steroids, and rituximab. The patient’s condition improved during his 21-day hospital stay and was followed up every 2 weeks for 3 months. The patient’s factor X level recovered after two weeks of discharge with no other hemorrhagic episodes. Full article

7 pages, 661 KiB

Open AccessEditor’s ChoiceCase Report

A Report of a Symptomatic Progressive Myeloma during Pregnancy and Postpartum Period from Asymptomatic State

by Gehad Elgabry, Lydia Spencer, Hisam Siddiqi, Soumya Ojha and Farooq Wandroo

Hematol. Rep. 2023, 15(2), 305-311; https://doi.org/10.3390/hematolrep15020031 - 5 May 2023

Cited by 2 | Viewed by 2578

Abstract

Multiple myeloma is a plasma cell malignancy that is most commonly observed in males in the sixth and seventh decade of life. The clinical scenario of multiple myeloma with concurrent pregnancy is considered to be very rare. We detail here the case [...] Read more.

Multiple myeloma is a plasma cell malignancy that is most commonly observed in males in the sixth and seventh decade of life. The clinical scenario of multiple myeloma with concurrent pregnancy is considered to be very rare. We detail here the case of a young female with known IgG kappa multiple myeloma who was found to have a steady elevation of her IgG kappa paraprotein during pregnancy and symptomatic progression in the postpartum period. She delivered a healthy baby at 40 weeks gestation. We present a review of all reported cases of known multiple myeloma progressing during pregnancy and in the postpartum period, the treatments given, and their outcomes. The report also provides suggestions for diagnosis and management of myeloma during pregnancy in order to have an outcome of successful uncomplicated pregnancy with healthy offspring. Full article

(This article belongs to the Special Issue Opportunities and Challenges in Multiple Myeloma Treatment and Management)

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7 pages, 471 KiB

Open AccessArticle

Comparison of Two Methods of Capillary Sampling in Blood Pre-Donation Anemia Screening in Brazil

by Cristina Rabelo Flor, André de Oliveira Baldoni, Sheila de Oliveira Garcia Mateos, Ester Cerdeira Sabino and Cláudia Di Lorenzo Oliveira

Hematol. Rep. 2023, 15(2), 298-304; https://doi.org/10.3390/hematolrep15020030 - 26 Apr 2023

Cited by 1 | Viewed by 2011

Abstract

Background: The laboratory tests most used by blood banks to diagnose anemia are the hemoglobin (Hb) and microhematocrit (Hct) tests, measured from capillary samples. Objective: To analyze the two capillary screening methods for pre-donation anemia by comparing their agreement in diagnosing anemia. Method: [...] Read more.

Background: The laboratory tests most used by blood banks to diagnose anemia are the hemoglobin (Hb) and microhematocrit (Hct) tests, measured from capillary samples. Objective: To analyze the two capillary screening methods for pre-donation anemia by comparing their agreement in diagnosing anemia. Method: A cross-sectional study in a population of 15,521 blood donation candidates for whom information was available on Hb and Hct, performed from capillary blood samples. Hb was determined using the HemoCue^® test and Hct by the centrifugation method. The Kappa coefficient was calculated to assess the agreement between the methods. Pearson’s correlation tests and gender-adjusted linear regression were used to assess the change in the response variable (Hb) as a function of the explanatory variable (Hct). Results: The majority of the study population were men (70.4%), aged between 18 and 44 years (72.1%), who declared themselves white or mixed skin color (85.6%), and had undergone at least 11 years of complete education (72.4%). The Kappa coefficient found was 92.7 and 99.2 for women and men, respectively. Pearson’s correlation showed a correlation coefficient of 0.98 and the linear regression graph showed an adequate relationship between the tests with R² = 0.97. Conclusions: Comparing the Hb and Hct capillary tests, it was found that Hct can be safely used to screen for anemia in pre-blood donation. Full article

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8 pages, 2063 KiB

Open AccessEditor’s ChoiceCase Report

Testosterone Usage Leading to Pulmonary Embolisms and Deep Vein Thrombosis: A Case Report and Review of the Literature

by Sasmith R. Menakuru, Mona Atta, Vijaypal S. Dhillon and Ahmed Salih

Hematol. Rep. 2023, 15(2), 290-297; https://doi.org/10.3390/hematolrep15020029 - 26 Apr 2023

Cited by 5 | Viewed by 4450

Abstract

Androgen usage has widely increased in recent times via prescribed and unprescribed means. Testosterone is a popular androgen taken by both athletes and the general population. While there is some evidence of androgens being thrombogenic, we report on a 19-year-old male who presented [...] Read more.

Androgen usage has widely increased in recent times via prescribed and unprescribed means. Testosterone is a popular androgen taken by both athletes and the general population. While there is some evidence of androgens being thrombogenic, we report on a 19-year-old male who presented to the hospital after the usage of testosterone for one month, leading to the development of multiple pulmonary emboli and deep vein thrombosis. The authors hope to elucidate the relationship between testosterone usage and thrombosis formation. Full article

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7 pages, 577 KiB

Open AccessEditor’s ChoiceCase Report

A Case of Vancomycin-Induced Severe Immune Thrombocytopenia

by Shivani Shah, Ryan Sweeney, Maitreyee Rai and Deep Shah

Hematol. Rep. 2023, 15(2), 283-289; https://doi.org/10.3390/hematolrep15020028 - 24 Apr 2023

Cited by 3 | Viewed by 3858

Abstract

A male in his 60s presented with left lower extremity fractures following a vehicle accident. Hemoglobin, initially, was 12.4 mmol/L, and platelet count was 235 k/mcl. On day 11 of admission, his platelet count initially dropped to 99 k/mcl, and after recovery it [...] Read more.

A male in his 60s presented with left lower extremity fractures following a vehicle accident. Hemoglobin, initially, was 12.4 mmol/L, and platelet count was 235 k/mcl. On day 11 of admission, his platelet count initially dropped to 99 k/mcl, and after recovery it rapidly decreased to 11 k/mcl on day 16 when the INR was 1.3 and aPTT was 32 s, and he continued to have a stable anemia throughout admission. There was no response in platelet count post-transfusion of four units of platelets. Hematology initially evaluated the patient for disseminated intravascular coagulation, heparin-induced thrombocytopenia (anti-PF4 antibody was 0.19), and thrombotic thrombocytopenic purpura (PLASMIC score of 4). Vancomycin was administered on days 1–7 for broad spectrum antimicrobial coverage and day 10, again, for concerns of sepsis. Given the temporal association of thrombocytopenia and vancomycin administration, a diagnosis of vancomycin-induced immune thrombocytopenia was established. Vancomycin was discontinued, and 2 doses of 1000 mg/kg of intravenous immunoglobulin 24 h apart were administered with the subsequent resolution of thrombocytopenia. Full article

(This article belongs to the Special Issue Personalized Therapy and Clinical Outcomes for Congenital and Acquired Haemorrhagic Disorders, Thromboembolic Disease and Platelet Disorders)

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17 pages, 1550 KiB

Open AccessEditor’s ChoiceArticle

Changes in Hematologic Lab Measures Observed in Patients with Paroxysmal Nocturnal Hemoglobinuria Treated with C5 Inhibitors, Ravulizumab and Eculizumab: Real-World Evidence from a US Based EMR Network

by Jesse Fishman, Seth Kuranz, Michael M. Yeh, Kaylen Brzozowski and Herman Chen

Hematol. Rep. 2023, 15(2), 266-282; https://doi.org/10.3390/hematolrep15020027 - 21 Apr 2023

Cited by 8 | Viewed by 3892

Abstract

Paroxysmal nocturnal hemoglobinuria (PNH), a rare acquired hematologic disorder, can be treated with C5 inhibitors (C5i) such as eculizumab or ravulizumab. This retrospective study is the first to describe real-world treatment patterns and changes in hematologic PNH-monitoring laboratory tests among C5i-treated US patients. [...] Read more.

Paroxysmal nocturnal hemoglobinuria (PNH), a rare acquired hematologic disorder, can be treated with C5 inhibitors (C5i) such as eculizumab or ravulizumab. This retrospective study is the first to describe real-world treatment patterns and changes in hematologic PNH-monitoring laboratory tests among C5i-treated US patients. Data were extracted from TriNetX Dataworks Network and included patients with a PNH diagnosis between 1 January 2010, and 20 August 2021. Patients were stratified into three cohorts based on their C5i usage: eculizumab, ravulizumab (prior eculizumab), and ravulizumab (eculizumab naïve). Hematological markers (hemoglobin [Hb], lactate dehydrogenase [LDH], and absolute reticulocyte count [ARC]) and relevant clinical events (e.g., breakthrough hemolysis [BTH], complement-amplifying conditions [CAC], thrombosis, infection, and all-cause mortality) were captured any time within 12 months post-index treatment. Of the 143 (eculizumab), 43 (ravulizumab, prior eculizumab), and 33 (ravulizumab, eculizumab naïve) patients, mean age across cohorts was 42–51 years, 55–61% were female, 63–73% were White, and 33–40% had aplastic anemia. Among all cohorts 12 months post-C5i treatment, 50–82% remained anemic, 8–32% required ≥1 transfusion, and 13–59% had BTH, of which 33%-54% had CACs. Additionally, thrombosis was seen in 7–15% of patients, infection in 20–25%, and mortality in 1–7%. These findings suggest many C5i-treated patients experience suboptimal disease control. Full article

(This article belongs to the Special Issue Personalized Therapy and Clinical Outcomes for Congenital and Acquired Haemorrhagic Disorders, Thromboembolic Disease and Platelet Disorders)

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10 pages, 273 KiB

Open AccessEditor’s ChoiceArticle

Utilization of Genomic Tumor Profiling in Pediatric Liquid Tumors: A Clinical Series

by Ishna Sharma, Min Ji Son, Shoaleh Motamedi, Alice Hoeft, Christa Teller, Tyler Hamby and Anish Ray

Hematol. Rep. 2023, 15(2), 256-265; https://doi.org/10.3390/hematolrep15020026 - 19 Apr 2023

Cited by 2 | Viewed by 1838

Abstract

Hematologic tumors are mostly treated with chemotherapies that have poor toxicity profiles. While molecular tumor profiling can expand therapeutic options, our understanding of potential targetable drivers comes from studies of adult liquid tumors, which does not necessarily translate to efficacious treatment in pediatric [...] Read more.

Hematologic tumors are mostly treated with chemotherapies that have poor toxicity profiles. While molecular tumor profiling can expand therapeutic options, our understanding of potential targetable drivers comes from studies of adult liquid tumors, which does not necessarily translate to efficacious treatment in pediatric liquid tumors. There is also no consensus on when profiling should be performed and its use in guiding therapies. We describe a single institution’s experience in integrating profiling for liquid tumors. Pediatric patients diagnosed with leukemia or lymphoma and who underwent tumor profiling were retrospectively reviewed. Ten (83.3%) patients had relapsed disease prior to tumor profiling. Eleven (91.7%) patients had targetable alterations identified on profiling, and three (25%) received targeted therapy based on these variants. Of the three patients that received targeted therapy, two (66.7%) were living, and one (33.3%) decreased. For a portion of our relapsing and/or treatment-refractory patients, genetic profiling was feasible and useful in tailoring therapy to obtain stable or remission states. Practitioners may hesitate to deviate from the ‘standard of therapy’, resulting in the underutilization of profiling results. Prospective studies should identify actionable genetic variants found more frequently in pediatric liquid tumors and explore the benefits of proactive tumor profiling prior to the first relapse. Full article

12 pages, 1595 KiB

Open AccessEditor’s ChoiceArticle

Molecular Tumor Boards: The Next Step towards Precision Therapy in Cancer Care

by Angela Liu, Paige Vicenzi, Ishna Sharma, Kaci Orr, Christa Teller, Micha Koentz, Heidi Trinkman, Kelly Vallance and Anish Ray

Hematol. Rep. 2023, 15(2), 244-255; https://doi.org/10.3390/hematolrep15020025 - 4 Apr 2023

Cited by 8 | Viewed by 4144

Abstract

The application of molecular tumor profiles in clinical decision making remains a challenge. To aid in the interpretation of complex biomarkers, molecular tumor boards (MTBs) have been established worldwide. In the present study, we show that a multidisciplinary approach is essential to the [...] Read more.

The application of molecular tumor profiles in clinical decision making remains a challenge. To aid in the interpretation of complex biomarkers, molecular tumor boards (MTBs) have been established worldwide. In the present study, we show that a multidisciplinary approach is essential to the success of MTBs. Our MTB, consisting of pediatric oncologists, pathologists, and pharmacists, evaluated 115 cases diagnosed between March 2016 and September 2021. If targetable mutations were identified, pharmacists aided in the evaluation of treatment options based on drug accessibility. Treatable genetic alterations detected through molecular testing most frequently involved the cell cycle. For 85% of the cases evaluated, our MTB provided treatment recommendations based on the patient’s history and results of molecular tumor testing. Only three patients, however, received MTB-recommended targeted therapy, and only one of these patients demonstrated an improved clinical outcome. For the remaining patients, MTB-recommended treatment often was not administered because molecular tumor profiling was not performed until late in the disease course. For the three patients who did receive MTB-recommended therapy, such treatment was not administered until months after diagnosis due to physician preference. Thus, the education of healthcare providers regarding the benefits of targeted therapy may increase acceptance of these novel agents and subsequently improve patient survival. Full article

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19 pages, 1863 KiB

Open AccessReview

Risk of Thrombosis during and after a SARS-CoV-2 Infection: Pathogenesis, Diagnostic Approach, and Management

by Henry Sutanto and Gatot Soegiarto

Hematol. Rep. 2023, 15(2), 225-243; https://doi.org/10.3390/hematolrep15020024 - 3 Apr 2023

Cited by 30 | Viewed by 4854

Abstract

Coronavirus disease 2019 (COVID-19) increases the risk of thromboembolic events, especially in patients with severe infections requiring intensive care and cardiorespiratory support. COVID-19 patients with thromboembolic complications have a higher risk of death, and if they survive, these complications are expected to negatively [...] Read more.

Coronavirus disease 2019 (COVID-19) increases the risk of thromboembolic events, especially in patients with severe infections requiring intensive care and cardiorespiratory support. COVID-19 patients with thromboembolic complications have a higher risk of death, and if they survive, these complications are expected to negatively affect these patients’ quality of life. Moreover, recent data reported that the risk of thromboembolism remains high months after a COVID-19 infection. Therefore, understanding the pathogenesis of thrombosis in the setting of COVID-19 may facilitate the early prevention and treatment of COVID-19-associated thromboembolism to reduce concomitant morbidity, mortality, and disability. This review will first discuss the clinical characteristics of COVID-19 infections, particularly with regard to the underlying pathophysiology. Then, the pathogenesis of COVID-19-associated thrombosis at the molecular and cellular levels will be comprehensively reviewed. Next, the clinical manifestations of venous and arterial thromboembolism in COVID-19 as well as the potential benefits of several laboratory markers of thrombosis will be further discussed. Lastly, the preventive and therapeutic management of thromboembolism during and after COVID-19 will also be explained. Full article

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Hematol. Rep., Volume 15, Issue 2 (June 2023) – 17 articles

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