Hematology/oncology And Stem Cell Therapy[JOURNAL]
Sun
200 papers
Application of EBMT, MASCC, and qSOFA Scores to Predict Complicated Febrile Neutropenia and Mortality in Autologous Stem Cell Transplant Recipients.
Barros TA, Schaffel R, de Azevedo Neto GS … +5 more , de Lucena Gaio B, Batista AT, Valentim MR, Maiolino A, Garnica M
Hematol Oncol Stem Cell Ther · 2023 May · PMID 37399007 · Publisher ↗
BACKGROUND AND OBJECTIVES: Three different scores were addressed as predictors of outcomes in autologous stem cell transplant (Auto SCT): one was calculated by pretransplant characteristics (European Society for Blood an...
BACKGROUND AND OBJECTIVES: Three different scores were addressed as predictors of outcomes in autologous stem cell transplant (Auto SCT): one was calculated by pretransplant characteristics (European Society for Blood and Marrow Transplantation [EBMT] risk score), and two were calculated at the onset of febrile neutropenia (Multinational Association for Supportive Care in Cancer [MASCC] and Quick Sequential Organ Failure Assessment [qSOFA]). We considered bloodstream infection (BSI), carbapenem prescription, admission to the intensive care unit (ICU), and mortality as outcomes. PATIENTS: A total of 309 patients with a median age of 54 years were enrolled. RESULTS: Patients with EBMT score ≥4 (EBMT 4+) had higher ICU rates (14% vs. 4%; p < 0.01) and more carbapenem prescriptions (61% vs. 38%; p < 0.001) than those with EBMT score <4. MASCC <21 points (MASCC HR) was associated with carbapenem prescription (59% vs. 44%; p = 0.013), ICU (19% vs. 3%; p < 0.01), and death (4% vs. 0; p = 0.014). Patients with at least two points by qSOFA (qSOFA 2+) had more frequent BSI (55% vs. 22%; p = 0.03), ICU admissions (73% vs. 7; p < 0.01), and death (18% vs. 0.7, p = 0.02). EBMT 4+ and MASCC HR achieved the best sensitivities for ICU. For death, the best sensitivity was obtained with MASCC. CONCLUSION: In conclusion, risk scores for Auto SCT showed an association with outcomes and had different performances when combined or used alone. Therefore, risk scores for Auto SCT are useful in supportive care and clinical surveillance in stem cell transplant recipients.
Hematologic Manifestations of Parvovirus B19 Infection.
Algwaiz G, Alharbi A, Alsehaim K … +3 more , Alahmari A, El Fakih R, Aljurf M
Hematol Oncol Stem Cell Ther · 2023 May · PMID 37363985 · Publisher ↗
Parvovirus B19 virus infection is widespread among humans because of its highly infectious and obstinate nature, with up to 80% of the population testing positive for IgG antibodies against the virus. Pronormoblasts obse...
Parvovirus B19 virus infection is widespread among humans because of its highly infectious and obstinate nature, with up to 80% of the population testing positive for IgG antibodies against the virus. Pronormoblasts observed in biopsy are the hallmarks of PVB19 infection. In addition, PVB19 affects the skin, heart, brain, joints, and liver and can be diagnosed through antibody detection or DNA detection via PCR. Due to its capsid proteins' high affinity for bone marrow receptors, its main presentation is the suppression of bone marrow functions. It has been shown to affect patients with hemolytic anemia and patients with hematological malignancies, presenting with pure red cell aplasia. The main available effective treatment option is IV immunoglobulins; however, the risk of recurrence remains high after treatment.
Cytogenetically Normal Acute Myeloid Leukaemia at a Single Centre in South Africa.
Jenkins N, Blanshard LA, Stone M … +3 more , Verburgh E, Oosthuizen J, Shires K
Hematol Oncol Stem Cell Ther · 2023 May · PMID 37363984 · Publisher ↗
BACKGROUND AND OBJECTIVES: The heterogeneous molecular landscape of cytogenetically normal acute myeloid leukemia (CN-AML) renders it an ongoing therapeutic challenge. The European LeukemiaNet (ELN) 2017 guidelines attem...
BACKGROUND AND OBJECTIVES: The heterogeneous molecular landscape of cytogenetically normal acute myeloid leukemia (CN-AML) renders it an ongoing therapeutic challenge. The European LeukemiaNet (ELN) 2017 guidelines attempted to address this by guiding post-remission therapy according to six prognostically informative mutations. However, its applicability in a South African setting remains unclear due to limited local data. This retrospective study aimed to describe a South African CN-AML cohort according to clinicopathological and molecular features as well as treatment outcomes and, consequently, to investigate the local applicability of a triple-mutation testing approach for risk stratification in accordance with the ELN 2017 guidelines, using nucleophosmin 1 (NPM1), fms-related receptor tyrosine kinase 3 internal tandem duplication (FLT3-ITD), and CCAAT enhancer-binding protein alpha (CEBPA) mutation status. MATERIALS AND METHODS: A review of cytogenetic results for adult de novo AML cases diagnosed at Groote Schuur Hospital between 2005 and 2018 was performed. CN-AML cases were further characterized via a review of clinical and laboratory data and additional molecular testing on stored DNA samples to allow for mutation-based risk stratification and outcome analysis. RESULTS: In total, 218 patients with AML were identified, of which 33% were cytogenetically normal. NPM1, FLT3-ITD, and CEBPA mutations were found in 39%, 34%, and 9% of CN-AML cases, respectively. Retrospective risk stratification according to mutations in these three genes accurately identified both patients at a high risk of induction-resistant disease and those who required an allogeneic stem cell transplant in their first complete remission. CONCLUSION: Local rates of CN-AML and associated NPM1 and FLT3-ITD mutations were comparable to those of European cohorts. Limited mutation analysis in the form of triple-mutation testing proved to be an economical and therapeutically informative prognostication approach for CN-AML in a resource-limited setting.
Post-transplantation Cyclophosphamide-based Graft-versus-host-disease Prophylaxis Compared to Methotrexate-cyclosporine a in Matched-related Allogeneic Hematopoietic Stem Cell Transplantation.
Shouman MT, Mansour OM, El Gammal MM … +5 more , Abdel-Fattah RM, Samra MA, Elhaddad AM, Maher MA, Mahmoud HK
Hematol Oncol Stem Cell Ther · 2023 May · PMID 37363983 · Publisher ↗
BACKGROUND AND OBJECTIVES: Post-transplant cyclophosphamide (PTCy) has shown promising results with low rates of severe graft-versus-host-disease (GVHD), either alone or combined with conventional immunosuppression (CIS)...
BACKGROUND AND OBJECTIVES: Post-transplant cyclophosphamide (PTCy) has shown promising results with low rates of severe graft-versus-host-disease (GVHD), either alone or combined with conventional immunosuppression (CIS). However, studies comparing PTCy with CIS as a GVHD prophylaxis are scarce. The study aimed to determine the rates of GVHD and survival outcomes for patients undergoing peripheral blood stem cell transplant (PBSCT) from HLA-matched related donors (MRD) receiving PTCy-based GVHD prophylaxis and compare these outcomes with those of patients receiving methotrexate (MTX) and cyclosporine-A (CsA) as a GVHD prophylaxis. PATIENTS AND METHODS: Seventy-five patients with advanced hematologic malignancies who underwent MRD allogeneic hematopoietic cell transplantation (allo-HCT) were analyzed prospectively. These patients received PTCy and CSA as a GVHD prophylaxis (therapeutic group) and their outcomes were compared with those of 75 retrospectively collected patients who received methotrexate and CsA as a GVHD prophylaxis (historical group) from the same two transplant centers. RESULTS: The median recipient age was significantly lower in the MTX/CsA group at 28 years compared to 34 years in the PTCy/CSA group. Peripheral blood was the only graft source used. All patients had a complete MRD, with two patients having a one-antigen mismatched related donor within the PTCy/CsA group. The 1-year cumulative incidence (CI) of chronic GVHD was 13.4% with PTCy/CsA and 38.6% with MTX/CsA (P = .001). Acute GVHD CI across all grades did not differ between the groups, with 10.7% for PTCy/CsA and 14.7% for MTX/CsA (P = .46). At two years, the overall survival (OS) (54.4% vs 67.2%, P = 0.282), disease-free survival (DFS) (54.1% vs 66.1%, P = 0.358), relapse rates (27.4% vs 20.1%, P = 0.245), and non-relapse mortality (NRM) (29.3% vs 25%, P = 0.904) did not differ between PTCy/CsA and MTX/CsA, respectively. CONCLUSION: PTCy-based GVHD prophylaxis in MRD transplant is feasible and leads to lower chronic GVHD rates without causing a significantly different risk of relapse or survival than MTX/CsA. More extensive studies are needed to confirm our results.
Donor Age Influences Graft-Versus-Host Disease Relapse-Free Survival after Allogeneic Stem Cell Transplant in Elderly Patients in Two Countries from Latin America.
Berro M, Hamerschlak N, Milovic V … +19 more , Castro B, García AP, Ferini G, Real JJ, Vitriu A, Conca AG, Bendek G, Yantorno S, Rolon JM, Saslavsky M, Jarchum S, Cerutti A, da Silva CC, Rodrigues M, Riera L, Arbelbide J, Kusminsky G, Basquiera AL, GATMO-TC: Grupo Argentino de Trasplante de Medula Osea y Terapia Celular
Hematol Oncol Stem Cell Ther · 2023 May · PMID 37363982 · Publisher ↗
BACKGROUND AND OBJECTIVES: Allogeneic stem cell transplantation (Allo-SCT) in elderly patients is a growing practice. We aimed to determine the graft-versus-host disease (GVHD) relapse-free survival (GRFS) in patients ≥6...
BACKGROUND AND OBJECTIVES: Allogeneic stem cell transplantation (Allo-SCT) in elderly patients is a growing practice. We aimed to determine the graft-versus-host disease (GVHD) relapse-free survival (GRFS) in patients ≥65 years who underwent Allo-SCT in two countries from Latin America. PATIENTS AND METHODS: We performed a retrospective analysis of patients ≥65 years who underwent Allo-SCT in Argentina and Brazil from 2007 to 2019. RESULTS: Ninety-eight patients were evaluated, with primary diagnoses of acute myeloid leukemia and myelodysplastic syndrome; 30% of patients had a hematopoietic cell transplant-comorbidity index (HCT-CI) score ≥3 and 49% were in complete remission. Donor types included matched sibling (n = 41), matched unrelated (n = 31), and haploidentical (HID; n = 26) donors. The conditioning regimen was myeloablative in 28 patients (14 busulfan pharmacokinetically [PK]-guided) and reduced-intensity in 70 patients. The two-year non-relapse mortality (NRM) was 29%, with a higher NRM in melphalan-based compared to other conditionings (51% vs. 33%, p = 0.02). The two-year relapse rate was 24%, with a reduction in PK-guided busulfan (0% vs. 28%, p = 0.03). The two-year overall survival (OS) and GRFS was 52% and 38%, respectively, with a significant reduction in GRFS in HCT-CI ≥3 (27% vs. others 42%, p = 0.02) and donors ≥40 years (29% vs. <40 years 55%, p = 0.02). These variables remained significantly associated with GRFS after multivariate analysis. CONCLUSION: In this cohort of elderly patients from Argentina and Brazil undergoing Allo-SCT, donor age and comorbidities significantly influenced GRFS. The role of the conditioning regimen in this population deserves further investigation.
Post-Autologous Hematopoietic Cell Transplant Care in the "Home Sweet Home" Setting: A Treatment Paradigm Shift.
Kharfan-Dabaja MA, Roy V, Murthy H … +14 more , Fischer D, Mohty R, Greathouse A, Brown A, Moreno K, Godsey E, Higginbotham JM, Bartholomew A, Jackson A, Torres-Guzman RA, Forte AJ, Ailawadhi S, Dronca R, Maniaci M
Hematol Oncol Stem Cell Ther · 2023 May · PMID 37363981 · Publisher ↗
BACKGROUND: Multiple myeloma (MM) is the second most common hematologic malignancy, with 34,470 estimated new cases in 2022. High-dose therapy followed by autologous hematopoietic cell transplantation (auto-HCT) remains...
BACKGROUND: Multiple myeloma (MM) is the second most common hematologic malignancy, with 34,470 estimated new cases in 2022. High-dose therapy followed by autologous hematopoietic cell transplantation (auto-HCT) remains a standard treatment for MM even in the era of novel therapies. This is usually performed in hospital-based settings, either in the inpatient or outpatient units. Advanced Care at Home (ACH) represents a virtual hybrid hospital-at-home program that combines a virtual provider-staffed command center with a vendor-mediated supply chain capable of delivering high-acuity care in the comfort of the patients' own homes. In our program, we used the existing ACH platform to deliver post-HCT care for recipients of auto-HCT. PATIENTS AND METHODS: Four patients (female = 2, 50%) with MM, with a median age of 60 (range, 40-74) years, were admitted to the inpatient Blood and Marrow Transplant (BMT) unit. The conditioning regimen consisted of melphalan 200 mg/m, administered on day -2. All patients received stem cell infusion (day 0) in the inpatient setting, with a median dose of 3.64 (range, 2.92-8.22) × 10/kg CD34 cells. RESULTS: Patients were discharged to their homes after completing the infusion on day 0 or day +1 at the latest. Post-infusion care was provided by the ACH team in coordination with the BMT team. The median time intervals to absolute neutrophil count and platelet engraftment were 12 (range, 11-13) and 11 (range, 9-16) days, respectively. All patients were successfully discharged from the ACH program at a median of day +14 (range, day +14 to day +15). CONCLUSIONS: Our results highlight the feasibility of delivering post-HCT care for auto-HCT recipients in the home setting and confirm the generalizability of this approach.
Physical Activity Compliance to American Cancer Society Recommendations Amongst Hematopoietic Stem Cell Transplant Survivors.
Mead LE, Kelly DL, Dahl WJ … +5 more , Colee JC, Merchant A, Weaver MT, Wingard JR, Farhadfar N
Hematol Oncol Stem Cell Ther · 2023 May · PMID 37363980 · Publisher ↗
BACKGROUND AND OBJECTIVES: The aims of this study were to determine the extent to which hematopoietic cell transplantation (HCT) survivors adhere to the American Cancer Society recommendations for weekly physical activit...
BACKGROUND AND OBJECTIVES: The aims of this study were to determine the extent to which hematopoietic cell transplantation (HCT) survivors adhere to the American Cancer Society recommendations for weekly physical activity and identify potential demographic and transplant characteristics associated with the lack of compliance. METHODS: This cross-sectional study included adults who had undergone HCT and were at least 1 year post transplantation. Physical activity was assessed using the screening tool of the Block 2014. The type of activity, frequency, and intensity were converted into the metabolic equivalent of task (MET) scores (0-499.0 MET min/week, inadequate activity; 500-1000 MET min/week, adequate activity; >1000 MET min/week, highly vigorous activity). RESULTS: Participants (n = 81) reported a median MET score of 153 min/week, and 83% failed to reach the physical activity guideline of >500 MET min/week. Only 17.3% met the ACS recommendations, with three reporting above 1000 MET min/week. Median daily moderate and vigorous physical activity minute totals were 18.0 and 5.9 min/d, with 85.2% and 60.5% of participants involved, respectively. The median total physical activity energy expenditure was 744 kcal/d. Only race was associated with MET score, with Whites reporting higher MET scores. CONCLUSION: Most HCT survivors assessed in this study did not meet the ACS physical activity recommendations. These findings reinforce the need to incorporate screening for physical activity into HCT survivorship care, offer counseling to those who do not meet the recommended levels, and encourage a physically active lifestyle among HCT survivors.
OncotypeDX Testing Does Not Benefit Patients with Estrogen and Progesterone Receptor Positive Grade 1 Breast Cancers: A TAILORx Validated Study.
Sibia US, Mylander C, Martin T … +5 more , Rosman M, Sanders TJ, Lee Y, Tafra L, Jackson RS
Hematol Oncol Stem Cell Ther · 2023 May · PMID 37363979 · Publisher ↗
BACKGROUND & OBJECTIVES: We previously described a predictive AAMC model that identifies patients (grade 1, hormonepositive) who would not benefit from OncotypeDX testing. The purpose of this study was to validate the AA...
BACKGROUND & OBJECTIVES: We previously described a predictive AAMC model that identifies patients (grade 1, hormonepositive) who would not benefit from OncotypeDX testing. The purpose of this study was to validate the AAMC model by assessing distant recurrence-free interval (DRFI) and invasive disease-free survival (IDFS) using TAILORx clinical trial data. MATERIALS & METHODS: We retrospectively analyzed TAILORx trial data and categorized patients based on the AAMC model. AAMC low-risk patients are those with grade 1 and hormone-positive tumors. Kaplan-Meier curves examined DRFI and IDFS. RESULTS: Of the 9195 cases, 2246 (24.4%) were identified by AAMC as low-risk. Among these AAMC low-risk patients, 55.2% had Recurrence Score (RS) 0-15, 42.3% had RS 15-25, and 2.4% had RS > 25. The 10-year DRFI did not differ for those who received adjuvant chemotherapy versus those who did not (98% vs. 96%, log-rank p = 0.46). Similarly, IDFS was comparable between those who received adjuvant chemotherapy and those that did not (86% vs. 86%, log-rank p = 0.66). Only 2.4% of AAMC low-risk patients were categorized as high-risk (RS > 25). A sensitivity analysis of this discordant group, wherein those with RS > 25 were re-classified into the no-chemotherapy group and assumed to have experienced recurrences at the rate expected without chemotherapy, did not find any difference in DRFI between those who received adjuvant chemotherapy and those who did not (log-rank p = 0.16). CONCLUSION: OncotypeDX testing does not benefit AAMC low-risk patients with hormone-positive grade 1 tumors. Based on these data, 1 in 4 TAILORx participants would not need OncotypeDX testing.
Prevalence, Patterns, and Predictors of Venous Thromboembolic Events in Patients Undergoing Salvage Chemotherapy and Autologous Stem Cell Transplantation for Relapsed Lymphomas.
Ma'koseh M, Alrwashdeh M, Abdel-Razeq N … +6 more , Alfar R, Edaily S, Bater R, Zmaily M, Almomani M, Abdel-Razeq H
Hematol Oncol Stem Cell Ther · 2023 May · PMID 37363978 · Publisher ↗
BACKGROUND AND OBJECTIVES: Almost 25% of patients with lymphoma may have relapse or develop refractory disease, and a majority of such patients undergo salvage chemotherapy and autologous stem cell transplantation (ASCT)...
BACKGROUND AND OBJECTIVES: Almost 25% of patients with lymphoma may have relapse or develop refractory disease, and a majority of such patients undergo salvage chemotherapy and autologous stem cell transplantation (ASCT). Data on venous thromboembolism (VTE) in this setting are scarce. This study aimed to investigate the prevalence and factors that may increase the risk of VTE in such patients. PATIENTS AND METHODS: Adult patients who were diagnosed with lymphoma and received salvage chemotherapy and ASCT were included in the study, and the subgroup with radiologically confirmed VTE were identified. Correlations between different clinical and laboratory variables and VTE were evaluated. RESULTS: A total of 216 patients (median age, 31 [range, 19-60] years) were enrolled in the study. Most patients (n = 140, 64.8%) had Hodgkin's lymphoma, while 54 (25.0%) had diffuse large B-cell lymphoma. A total of 36 (16.7%) patients had VTE, mostly as upper extremity deep vein thrombosis (n = 28, 77.8%); 18 (50%) of the cases were related to central venous catheter insertion. Thrombosis rates were higher among patients with high lactate dehydrogenase (LDH) level (29.2% vs. 5.9%, p < 0.001), those with mediastinal involvement (25.9% vs. 11.5%, p = 0.025). and those with longer hospital stay (22.3% vs.9.5%, p = 0.036). In the multivariate analysis, high LDH level (odds ratio (OR), 6.53; p < 0.001), mediastinal involvement (OR, 2.70; p = 0.005) and hospital stay ≥24 days (OR, 2.71; p = 0.007) were all associated with significantly higher VTE rates. CONCLUSION: Patients with relapsed lymphoma undergoing salvage chemotherapy and ASCT are at higher risk for VTE, especially in those with high LDH level, mediastinal involvement, and prolonged hospital stay. If no contraindications exist, thromboprophylaxis might be considered in these settings.
Venetoclax-based Treatment as Frontline Therapy for Chronic Lymphocytic Leukemia.
Guru Murthy GS, Atallah E
Hematol Oncol Stem Cell Ther · 2023 May · PMID 37363977 · Publisher ↗
The availability of novel targeted agents has revolutionized the management of chronic lymphocytic leukemia (CLL). Both B-cell lymphoma 2 (BCL2) and Bruton tyrosine kinase (BTK) inhibitors are highly effective agents for...
The availability of novel targeted agents has revolutionized the management of chronic lymphocytic leukemia (CLL). Both B-cell lymphoma 2 (BCL2) and Bruton tyrosine kinase (BTK) inhibitors are highly effective agents for CLL treatment. Several clinical trials have demonstrated the efficacy and safety of these agents in the management of newly diagnosed and relapsed/refractory CLL. This has led to two broad approaches in the frontline management of CLL, namely venetoclax-based time-limited therapy versus BTK inhibitor-based continuous therapy. In this review, we discussed why we consider venetoclax-based therapy as a suitable frontline option for patients with CLL.
Prevalence of Sleep Aid Medication Use in Patients Receiving a Hematopoietic Cell Transplant on an Inpatient Unit.
Cusatis R, Ibrahim A, Knight JM … +2 more , D'Souza A, Shaw BE
Hematol Oncol Stem Cell Ther · 2023 May · PMID 37363976 · Full text
BACKGROUND AND OBJECTIVES: There is a lack of research regarding the use of sleep aids after hematopoietic stem cell transplantation (HCT). We describe the prevalence of sleep aid administration in the HCT unit and ident...
BACKGROUND AND OBJECTIVES: There is a lack of research regarding the use of sleep aids after hematopoietic stem cell transplantation (HCT). We describe the prevalence of sleep aid administration in the HCT unit and identify associations with patient or clinical characteristics. PATIENTS AND METHODS: In this retrospective analysis of sequential inpatient HCTs from July 1 to December 31, 2016 we describe whether and when patients were prescribed sleep aid medications. Chi-square tests determined significant differences between patient characteristics, sleep aid prescription, and time of prescription. RESULTS: Of the 225 patients identified, 193 (86%) were prescribed sleep aids. Significantly more women received prescriptions for sleep aids (90.4%) than men (81%; P = .047). One hundred patients (44%) received prescriptions exclusively while in the hospital. CONCLUSION: Findings show a high prevalence of sleep medication use in patients undergoing inpatient HCT, primarily during hospitalization. Future efforts toward standardized recommendations to optimize peri-transplant sleep would help clinicians and patients.
Platelet Inventory and Using Out-of-group Platelet Suspension: A Cost-Effective Strategy for a Blood Transfusion Service.
Yıldız A, Doğan M, Yalvaç M … +1 more , Zihar B
Hematol Oncol Stem Cell Ther · 2023 May · PMID 37363973 · Publisher ↗
BACKGROUND: Platelet (PLT) transfusions are essential for advanced hospitals, especially those with onco-hematology departments. However, platelet concentrates (PCs) have supply limitations and a shorter shelf life, whic...
BACKGROUND: Platelet (PLT) transfusions are essential for advanced hospitals, especially those with onco-hematology departments. However, platelet concentrates (PCs) have supply limitations and a shorter shelf life, which create difficulties for blood transfusion services (TSs). MATERIALS AND METHODS: This retrospective study was conducted over a 4-year period between January 2017 and January 2021 in a tertiary referral hospital. From the beginning of 2020, as a new strategy of our TS, a PLT inventory was produced and ABO-identical transfusions were prioritized when the inventory allowed; when this was not possible, ABO and Rh incompatible transfusion was employed. The numbers of transfused and discarded PCs were compared for each year. RESULTS: In 2017, a total of 799 PPCs were used and 70 PPCs were discarded with the expiration ratio (ER) of 8.0%. In 2018, 1124 PPCs were used and 99 PPCs were discarded with the ER of 7.4%. In 2019, 726 PPCs were used and 91 PPCs were discarded with the ER of 11.1%. In 2020, 1100 PPCs were used for 569 patients, of which 251 PPCs were ABO and Rh incompatible without any severe transfusion reaction. A total of 56 PPCs were discarded with the ER of 4.8%. CONCLUSION: The results of the current study suggested that with the determination of the platelet stock level and the use of out-of-group PCs, the rate of discarded PLT could be reduced. Nevertheless, based on current literature and experience, each TSs should make their own strategies and policies to provide an adequate supply of PCs.
Updates on the Treatment of Tenosynovial Giant Cell Tumor.
Chan AS, Katiyar V, Dy P … +1 more , Singh V
Hematol Oncol Stem Cell Ther · 2023 May · PMID 37363972 · Publisher ↗
Tenosynovial giant cell tumor (TGCT) is a rare inflammatory disorder affecting the joint synovium, bursae, and tendon sheaths that causes non-specific and often insidious joint discomfort. The application of systemic che...
Tenosynovial giant cell tumor (TGCT) is a rare inflammatory disorder affecting the joint synovium, bursae, and tendon sheaths that causes non-specific and often insidious joint discomfort. The application of systemic chemotherapy has been limited due to poor and unsustained disease responses. Surgery with or without adjuvant radiation is the primary treatment modality for TGCT. With its locally destructive nature and increased recurrence, multiple surgical interventions become necessary throughout the course of the disease, leading to disfigurement, decreased quality of life, and increased mortality. However, owing to recent evidence demonstrating the overexpression of colony-stimulating factor 1 (CSF-1) in TGCT, selective tyrosine kinase inhibitors targeting CSF-1 receptors are being developed. Pexidartinib is the first CSF-1 receptor inhibitor approved for the treatment of TGCT. Here, we discuss various available treatment strategies and ongoing investigations and trials targeting diffuse TGCT, which include nilotinib, lacnotuzumab, cabiralizumab, vimseltinib, and emactuzumab.
Analysis of Macrophage Migration Inhibitory Factor Genotype in Hemophilia a Patients.
Hadi MA, Ibrahim WN, Hassan MK
Hematol Oncol Stem Cell Ther · 2023 May · PMID 37363971 · Publisher ↗
BACKGROUND: Hemophilia A, an X-linked bleeding disorder, is caused by a complete or partial deficiency in factor VIII. Multiple factors engage in the development and progression of bleeding episodes in hemophilia patient...
BACKGROUND: Hemophilia A, an X-linked bleeding disorder, is caused by a complete or partial deficiency in factor VIII. Multiple factors engage in the development and progression of bleeding episodes in hemophilia patients, especially arthropathy. OBJECTIVES: Detection of macrophage migration inhibitory factor (MIF)-173 G/C polymorphism in people with hemophilia A (PWH) and the possible associations between the type of MIF gene polymorphism and selected disease-related variables. PATIENTS AND METHODS: This case-control study included 95 male patients with hemophilia A and 95 non-hemophiliac subjects, all aged from 2 months to 63 years. An allele-specific polymerase chain reaction (AS-PCR) with a multiplex technique was used to detect MIF polymorphisms. RESULTS: A significantly higher frequency of GG polymorphism was reported in the control group (81, 85.3%) compared to PWH (64, 67.4%), while a significantly higher frequency of GC polymorphism was found in PWH (21, 22.1%) than that in healthy subjects (10, 10.5%), P < 0.05. The G allele polymorphism was detected in 90.0% of the control group compared to 78.4% of PWH (149 subjects), while the C allele frequency was higher in PWH (41, 21.6%) compared to that in healthy individuals (18, 10.0%), P < 0.05. The frequencies of varied MIF-173 polymorphisms did not show significant differences among patients with different clinical presentations or in relation to presence of inhibitors, P > 0.05. CONCLUSIONS: MIF-173 GC polymorphism is seen in PWH more than that in healthy individuals. Further studies are required to detect additional SNPs through sequencing of the MIF gene and to detect MIF serum levels during bleeding episodes.
Cross Fire: BTK Inhibitors Alone or in Combination are the Best Frontline Therapy for CLL.
Sottero T, Awan FT
Hematol Oncol Stem Cell Ther · 2023 May · PMID 37363970 · Publisher ↗
BTK (Bruton's tyrosine kinase) inhibitors are highly effective front-line therapy for CLL (chronic lymphocytic leukemia) due to high response rates and prolonged progression-free survival, even in patients with high-risk...
BTK (Bruton's tyrosine kinase) inhibitors are highly effective front-line therapy for CLL (chronic lymphocytic leukemia) due to high response rates and prolonged progression-free survival, even in patients with high-risk disease features. They are also generally well tolerated, with the newer BTK inhibitors demonstrating better tolerability than ibrutinib while maintaining efficacy. Adverse effects such as bleeding or infections are usually manageable with supportive care or dose adjustments. Orally administered BTK inhibitors do not require intensive or inpatient monitoring and improve quality-of-life outcomes. Moreover, the established activity of venetoclax in the setting of BTK inhibitor failure is also reassuring as a salvage option. Nevertheless, the advantage of venetoclax as a time-limited treatment option is substantial, despite its inferior progression-free survival, since these patients can get another challenge with a reasonable chance of success. BTK inhibitors after venetoclax may be effective, but long-term data is limited. Given these reasons, BTK inhibitors remain the preferred treatment option as initial therapy for patients with CLL, especially those with del17p or TP53 mutations.
Effectiveness, Safety, and Cost Implications of Outpatient Autologous Hematopoietic Stem Cell Transplant for Multiple Myeloma.
Marini J, Maldonado A, Weeda E … +3 more , Neppalli A, Hashmi H, Edwards K
Hematol Oncol Stem Cell Ther · 2023 May · PMID 37363969 · Publisher ↗
BACKGROUND AND OBJECTIVE: Autologous hematopoietic stem cell transplant (aHCT) has become standard care for patients with multiple myeloma (MM). Outpatient aHCT with high-dose melphalan conditioning has reduced costs and...
BACKGROUND AND OBJECTIVE: Autologous hematopoietic stem cell transplant (aHCT) has become standard care for patients with multiple myeloma (MM). Outpatient aHCT with high-dose melphalan conditioning has reduced costs and length of hospital stay. This study aimed to highlight the effectiveness, safety, and cost implications of outpatient vs inpatient aHCT at a tertiary academic medical center, as well as the utility of growth factor use in these patients. PATIENTS AND METHODS: Using an institutional HCT database, a total of 100 patients undergoing aHCT for MM were identified; 50 patients who underwent aHCT in the outpatient setting (chemotherapy and stem cell infusion followed by inpatient admission if needed) were compared with 50 patients in the inpatient setting (chemotherapy and stem cell infusion followed by discharge to outpatient setting). Patients were excluded if the melphalan dose was less than 200 mg/m. Outcomes assessed through retrospective chart review included time to engraftment, incidence of infection, febrile neutropenia, growth factor use, and total length of inpatient stay through day +100. RESULTS: Time to neutrophil and platelet engraftment was shorter in the outpatient group than in the inpatient group (14 vs 16 days and 19 vs 21 days, respectively; P < 0.001). Median length of hospital stay was also shorter in the outpatient group (8.5 vs 15.5 days, respectively; P < 0.001). Ninety percent of the outpatient group required admission for neutropenic fever, and 60% of these patients received growth factor support starting at a median of 9 days after stem cell infusion, for a median duration of 4 days. Compared to 16 patients who did not receive growth factor support, these patients had a significantly shorter time to neutrophil recovery (13 days with vs 15 days without growth factor, P = 0.02) and no difference in the total length of hospital stay (8 days with vs 10 days without growth factor, P = 0.43). CONCLUSION: For adult patients with MM undergoing aHCT, the outpatient setting is safe and reduces the total length of hospital stay and thus overall transplant costs. Growth factor support for patients with febrile neutropenia may not reduce length of stay for subsequent hospitalizations.
Peritoneal and Pleural Drains in Pediatric Hematopoietic Cell Transplant Recipients with Veno-Occlusive Disease are Safe and Do Not Adversely Impact Clinical Outcomes.
Rangarajan HG, Pai VB, Stanek JR … +6 more , Rush C, Naples J, Drope M, Polishchuk V, Abu-Arja R, Bajwa RP
Hematol Oncol Stem Cell Ther · 2023 May · PMID 37363968 · Publisher ↗
There is a lack of data on the safety and efficacy of peritoneal drain (PD) and chest tube (CT) in the management of effusions in stem cell transplant recipients with veno-occlusive disease (VOD). In this retrospective p...
There is a lack of data on the safety and efficacy of peritoneal drain (PD) and chest tube (CT) in the management of effusions in stem cell transplant recipients with veno-occlusive disease (VOD). In this retrospective pediatric study, clinical outcomes and health resource utilization (HRU) were compared in 32 patients with VOD who had a PD (PD+) post-HCT versus 27 patients who did not (PD-). Nine patients also had a CT (7 PD+ and 2 PD-). PD + patients were more likely than PD-patients to have received myeloablative conditioning (100% vs. 85.2%; p = 0.04) and have severe or very severe VOD (100% vs. 56% p < 0.01). Mechanical obstruction (38%) and hypotension (38%) were common complications, and 13% developed peritonitis. While the frequencies of cardiac dysfunction and acute kidney injury were comparable between both groups, respiratory support and its median duration were higher in PD + patients. The hospital and intensive care unit length of stay, albumin use, and the duration of defibrotide and albumin therapy was significantly longer in PD + patients. At a median follow-up of 1.04 years (range:0.03-14.6), the 2-year overall survival was similar in both groups (53.8% vs. 51.5%; p = 0.73). Although PD use was similar between 1995 and 2007 vs. 2008-2021; (47% vs. 58%; p = 0.65), day+100 mortality was improved in recent years (53.3% vs. 17.8%; p = 0.01), coinciding with the use of defibrotide (0% vs. 84%; p < 0.01). PD in pediatric patients with VOD post-HCT, although associated with increased HRU, was safe when clinically indicated and did not adversely impact clinical outcomes.
Managing Sickle Cell Disease in Patients for Whom Blood Transfusion Is Not an Option.
Tabiti BF, Ozawa S, Mian A … +3 more , Suri M, Yates HL, Hsu LL
Hematol Oncol Stem Cell Ther · 2023 Apr · PMID 37023225 · Publisher ↗
Sickle Cell Disease (SCD) is a hereditary blood disorder affecting beta hemoglobin. This disorder causes sickle-shaped red blood cells with decreased oxygen-carrying capacity resulting in vaso-occlusive crises. These cri...
Sickle Cell Disease (SCD) is a hereditary blood disorder affecting beta hemoglobin. This disorder causes sickle-shaped red blood cells with decreased oxygen-carrying capacity resulting in vaso-occlusive crises. These crises are often treated with analgesics, antibiotics, IV fluids, supplementary oxygen, and allogeneic blood transfusion. This treatment regimen becomes complicated when caring for SCD patients for whom blood transfusion is not an option. Blood transfusion may not be an option due to the patient's religious, personal, or medical concerns and in scenarios where blood is not available for transfusion. Some examples include the patient being a Jehovah's Witness, blood-borne pathogens concerns, or prior history of multiple alloantibodies and severe transfusion reactions. The number of patients in these categories is growing. The patients and their autonomy should be respected during treatment. This review focuses on the currently available modalities to best manage this subgroup of SCD patients without blood transfusion, including new professional guidelines and new therapies to reduce the severity of SCD as approved by the Food and Drug Administration since 2017.
JAK2/STAT5 Pathway Mutation Frequencies in South African BCR/ABL Negative MPN Patients.
Shires KL, Rust AJ, Harryparsad R … +2 more , Coburn JA, Gopie RE
Hematol Oncol Stem Cell Ther · 2023 Apr · PMID 37023224 · Publisher ↗
BACKGROUND: Mutations in JAK2/STAT5 proliferation pathway genes are key in the diagnosis of myeloproliferative neoplasms (MPN), with JAK2V617F being found in 50-97% of MPN subtypes. Low JAK2V617F positivity at our facili...
BACKGROUND: Mutations in JAK2/STAT5 proliferation pathway genes are key in the diagnosis of myeloproliferative neoplasms (MPN), with JAK2V617F being found in 50-97% of MPN subtypes. Low JAK2V617F positivity at our facility suggested that our South African MPN population may have a different mutational landscape. OBJECTIVES: We aimed to determine the JAK2/STAT5 mutation frequencies associated with our local MPN population, thus determining the relevance of these molecular tests in this group. We also investigated the haematopathological relevance of each test request, to assess testing practises. METHOD: This study involved the retrospective audit of 886 patients for whom JAK2V617F mutation testing had been requested for a suspected MPN diagnosis. FBC indices, erythropoietin levels and bone marrow biopsy results were used to classify the patients. JAK2V617F patient DNA was tested for calreticulin (CALR) exon9, myeloproliferative leukaemia protein (MPL) codon515 and JAK2 exon12 mutations. RESULTS: Only 23% of the patients demonstrated JAK2V617F positivity, with an additional 29 cases of CALR/MPL mutations being detected. Mutations were only detected in patients with abnormal FBC indices, as expected, yet 37% of the test requests were not associated with abnormal parameters at the time of testing. Mutation frequencies were as follows: Polycythaemia Vera: 97% JAK2V617F/3% (JAK2, CALR, MPL) triple negative; Essential thrombocythemia: 72% JAK2V617F/23%CALR/5%triple negative; Primary Myelofibrosis: 78%JAK2V617F/16%CALR/6%triple negative. CONCLUSION: Our study demonstrated that our MPN patients have a similar genetic landscape to other MPN populations, with >93% being able to be diagnosed by testing for the JAK2V617F and CALR exon9 mutations alone. Adoption of the WHO 2016 guidelines is recommended to guide testing practices.
Acquired Amegakaryocytic Thrombocytopenic Purpura: A Review of Therapeutic Options.
Hussain SA, Zafar A, Faisal H … +1 more , Ud Din MA
Hematol Oncol Stem Cell Ther · 2023 Apr · PMID 37023223 · Publisher ↗
Acquired amegakaryocytic thrombocytopenic purpura (AATP) is a rare bone marrow disorder characterized by either a marked decrease or a complete absence of megakaryocytes with the preservation of all other cell lines. To...
Acquired amegakaryocytic thrombocytopenic purpura (AATP) is a rare bone marrow disorder characterized by either a marked decrease or a complete absence of megakaryocytes with the preservation of all other cell lines. To date, more than 60 cases of AATP have been reported in the literature. Due to the rarity of this disease, no standard treatment guidelines have been established, and therapy is based on a handful of case studies and expert opinions. Herein, we provide a comprehensive review of currently utilized therapeutic options for AATP.
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