Clinical Edge Journal Scan

Key clinical point: In patients with psoriatic arthritis (PsA), efficacy of the novel interleukin (IL)-23p19 inhibitor guselkumab for joint improvement was comparable to IL-17A and subcutaneous tumor necrosis factor (TNF) inhibitors while offering a better efficacy for skin manifestations.

Major finding: Guselkumab 100 mg every 8 weeks and every 4 weeks were comparable to IL-17A and subcutaneous TNF inhibitors for American College of Rheumatology 20 response. Guselkumab showed better Psoriasis Area Severity Index 90 and 75 responses than most of the other agents.

Study details: Data come from a network meta-analysis of 26 phase 3, randomized controlled trials that evaluated 13 targeted therapies among adults with active PsA.

Disclosures: This work was supported by Janssen Research and Development. The authors including the lead author reported receiving grants, consulting fees, speaker fees, and/or honoraria from various sources. S Peterson, A Schubert, SD Chakravarty, CS Karyekar, and S Nair reported being employees of Janssen Pharmaceuticals and shareholder of Johnson & Johnson.

Source: Mease PJ et al. Rheumatology (Oxford). 2021 Mar 24. doi: 10.1093/rheumatology/keab119.

Key clinical point: In patients with psoriatic arthritis (PsA), efficacy of the novel interleukin (IL)-23p19 inhibitor guselkumab for joint improvement was comparable to IL-17A and subcutaneous tumor necrosis factor (TNF) inhibitors while offering a better efficacy for skin manifestations.

Major finding: Guselkumab 100 mg every 8 weeks and every 4 weeks were comparable to IL-17A and subcutaneous TNF inhibitors for American College of Rheumatology 20 response. Guselkumab showed better Psoriasis Area Severity Index 90 and 75 responses than most of the other agents.

Study details: Data come from a network meta-analysis of 26 phase 3, randomized controlled trials that evaluated 13 targeted therapies among adults with active PsA.

Disclosures: This work was supported by Janssen Research and Development. The authors including the lead author reported receiving grants, consulting fees, speaker fees, and/or honoraria from various sources. S Peterson, A Schubert, SD Chakravarty, CS Karyekar, and S Nair reported being employees of Janssen Pharmaceuticals and shareholder of Johnson & Johnson.

Source: Mease PJ et al. Rheumatology (Oxford). 2021 Mar 24. doi: 10.1093/rheumatology/keab119.

Key clinical point: In patients with psoriatic arthritis (PsA), efficacy of the novel interleukin (IL)-23p19 inhibitor guselkumab for joint improvement was comparable to IL-17A and subcutaneous tumor necrosis factor (TNF) inhibitors while offering a better efficacy for skin manifestations.

Major finding: Guselkumab 100 mg every 8 weeks and every 4 weeks were comparable to IL-17A and subcutaneous TNF inhibitors for American College of Rheumatology 20 response. Guselkumab showed better Psoriasis Area Severity Index 90 and 75 responses than most of the other agents.

Study details: Data come from a network meta-analysis of 26 phase 3, randomized controlled trials that evaluated 13 targeted therapies among adults with active PsA.

Disclosures: This work was supported by Janssen Research and Development. The authors including the lead author reported receiving grants, consulting fees, speaker fees, and/or honoraria from various sources. S Peterson, A Schubert, SD Chakravarty, CS Karyekar, and S Nair reported being employees of Janssen Pharmaceuticals and shareholder of Johnson & Johnson.

Source: Mease PJ et al. Rheumatology (Oxford). 2021 Mar 24. doi: 10.1093/rheumatology/keab119.

Key clinical point: Guselkumab resulted in significantly higher proportions of patients with psoriatic arthritis (PsA) with resolved enthesitis than placebo, which continued to improve through 1 year.

Major finding: A significantly higher proportion of patients with enthesitis at baseline achieved resolution by week 24 when treated with guselkumab 100 mg every 4 weeks (Q4W) and guselkumab 100 mg at week 0, 4, and then every 8 weeks (Q8W) than placebo (45% and 50% vs. 29%; P = .0301) which continued to rise in patients who continued guselkumab with 58% achieving resolution by week 52.

Study details: Findings are from a pooled analysis of DISCOVER-1 and DISCOVER-2 phase 3 trials involving patients with active PsA despite standard therapies randomly allocated to subcutaneous guselkumab 100 mg Q4W, guselkumab 100 mg Q8W, or placebo.

Disclosures: The work was supported by Janssen Research & Development, LLC. The authors reported receiving research grants, honoraria, and/or consultation/speaker fees from various sources, including Janssen. Some authors declared being employees of Janssen and owning stocks of Johnson & Johnson.

Source: McGonagle D et al. Rheumatology (Oxford). 2021 Apr 6. doi: 10.1093/rheumatology/keab285.

Key clinical point: Guselkumab resulted in significantly higher proportions of patients with psoriatic arthritis (PsA) with resolved enthesitis than placebo, which continued to improve through 1 year.

Major finding: A significantly higher proportion of patients with enthesitis at baseline achieved resolution by week 24 when treated with guselkumab 100 mg every 4 weeks (Q4W) and guselkumab 100 mg at week 0, 4, and then every 8 weeks (Q8W) than placebo (45% and 50% vs. 29%; P = .0301) which continued to rise in patients who continued guselkumab with 58% achieving resolution by week 52.

Study details: Findings are from a pooled analysis of DISCOVER-1 and DISCOVER-2 phase 3 trials involving patients with active PsA despite standard therapies randomly allocated to subcutaneous guselkumab 100 mg Q4W, guselkumab 100 mg Q8W, or placebo.

Disclosures: The work was supported by Janssen Research & Development, LLC. The authors reported receiving research grants, honoraria, and/or consultation/speaker fees from various sources, including Janssen. Some authors declared being employees of Janssen and owning stocks of Johnson & Johnson.

Source: McGonagle D et al. Rheumatology (Oxford). 2021 Apr 6. doi: 10.1093/rheumatology/keab285.

Key clinical point: Guselkumab resulted in significantly higher proportions of patients with psoriatic arthritis (PsA) with resolved enthesitis than placebo, which continued to improve through 1 year.

Major finding: A significantly higher proportion of patients with enthesitis at baseline achieved resolution by week 24 when treated with guselkumab 100 mg every 4 weeks (Q4W) and guselkumab 100 mg at week 0, 4, and then every 8 weeks (Q8W) than placebo (45% and 50% vs. 29%; P = .0301) which continued to rise in patients who continued guselkumab with 58% achieving resolution by week 52.

Study details: Findings are from a pooled analysis of DISCOVER-1 and DISCOVER-2 phase 3 trials involving patients with active PsA despite standard therapies randomly allocated to subcutaneous guselkumab 100 mg Q4W, guselkumab 100 mg Q8W, or placebo.

Disclosures: The work was supported by Janssen Research & Development, LLC. The authors reported receiving research grants, honoraria, and/or consultation/speaker fees from various sources, including Janssen. Some authors declared being employees of Janssen and owning stocks of Johnson & Johnson.

Source: McGonagle D et al. Rheumatology (Oxford). 2021 Apr 6. doi: 10.1093/rheumatology/keab285.

Key clinical point: Final analysis of OPAL Balance confirms long-term safety and efficacy of tofacitinib in patients with psoriatic arthritis.

Major finding: Only 1 instance of mortality occurred in tofacitinib group during the risk period (incidence, 0.1 patients with events [95% confidence interval, 0.0-0.3] per 100 person-years). The incidences of adverse events for herpes zoster, serious infections, opportunistic infections, adjudicated malignancies, and major adverse cardiovascular events were consistent as reported previously. Efficacy was sustained up to 36 months.

Study details: Findings are from OPAL Balance, a 36-month, long-term extension phase 3 study involving 686 adult patients with active PsA. Eligible patients (n=180) from the open-label phase entered the randomized, double-blind, 12-month methotrexate withdrawal substudy where they received open-label tofacitinib 5 mg twice daily with either masked placebo or masked methotrexate.

Disclosures: OPAL Balance was funded by Pfizer. The authors including the lead author reported receiving grants/consulting fees, speaker fees, and/or honoraria from various sources including Pfizer. Six of the authors reported being employees and shareholders of Pfizer.

Source: Nash P et al. Lancet Rheumatol. 2021 Apr 1. doi: 10.1016/S2665-9913(21)00010-2.

Key clinical point: Final analysis of OPAL Balance confirms long-term safety and efficacy of tofacitinib in patients with psoriatic arthritis.

Major finding: Only 1 instance of mortality occurred in tofacitinib group during the risk period (incidence, 0.1 patients with events [95% confidence interval, 0.0-0.3] per 100 person-years). The incidences of adverse events for herpes zoster, serious infections, opportunistic infections, adjudicated malignancies, and major adverse cardiovascular events were consistent as reported previously. Efficacy was sustained up to 36 months.

Study details: Findings are from OPAL Balance, a 36-month, long-term extension phase 3 study involving 686 adult patients with active PsA. Eligible patients (n=180) from the open-label phase entered the randomized, double-blind, 12-month methotrexate withdrawal substudy where they received open-label tofacitinib 5 mg twice daily with either masked placebo or masked methotrexate.

Disclosures: OPAL Balance was funded by Pfizer. The authors including the lead author reported receiving grants/consulting fees, speaker fees, and/or honoraria from various sources including Pfizer. Six of the authors reported being employees and shareholders of Pfizer.

Source: Nash P et al. Lancet Rheumatol. 2021 Apr 1. doi: 10.1016/S2665-9913(21)00010-2.

Key clinical point: Final analysis of OPAL Balance confirms long-term safety and efficacy of tofacitinib in patients with psoriatic arthritis.

Major finding: Only 1 instance of mortality occurred in tofacitinib group during the risk period (incidence, 0.1 patients with events [95% confidence interval, 0.0-0.3] per 100 person-years). The incidences of adverse events for herpes zoster, serious infections, opportunistic infections, adjudicated malignancies, and major adverse cardiovascular events were consistent as reported previously. Efficacy was sustained up to 36 months.

Study details: Findings are from OPAL Balance, a 36-month, long-term extension phase 3 study involving 686 adult patients with active PsA. Eligible patients (n=180) from the open-label phase entered the randomized, double-blind, 12-month methotrexate withdrawal substudy where they received open-label tofacitinib 5 mg twice daily with either masked placebo or masked methotrexate.

Disclosures: OPAL Balance was funded by Pfizer. The authors including the lead author reported receiving grants/consulting fees, speaker fees, and/or honoraria from various sources including Pfizer. Six of the authors reported being employees and shareholders of Pfizer.

Source: Nash P et al. Lancet Rheumatol. 2021 Apr 1. doi: 10.1016/S2665-9913(21)00010-2.

Key clinical point: Proportion of patients with psoriatic arthritis with at least 20% improvement in American College of Rheumatology (ACR20) response was significantly higher with upadacitinib than placebo; however, adverse events were more frequent with upadacitinib.

Major finding: The percentage of patients with ACR20 response at week 12 was higher with upadacitinib 15 mg (70.6%) and 30 mg (78.5%) vs. placebo (36.2%; P less than .001). Incidence of serious infections and serious adverse events with upadacitinib 15 mg, 30 mg, and placebo were 1.2%, 2.6%, and 0.9% and 3.3%, 6.1%, and 3.1%, respectively.

Study details: Findings are from SELECT-Psa 1, a phase 3 trial involving 1,704 patients with PsA who had an inadequate response to at least 1 nonbiologic disease-modifying antirheumatic drugs and were randomly allocated to receive either oral upadacitinib 15 or 30 mg once daily, placebo, or subcutaneous adalimumab (40 mg every other week).

Disclosures: The trial was sponsored by Abbvie. The authors reported receiving consulting fees, advisory board fees, lecture fees, travel support, grant support, and/or being an employee of and/or owning stocks in various pharmaceutical companies, including Abbvie.

Source: McInnes IB et al. N Engl J Med. 2021 Apr 1. doi: 10.1056/NEJMoa2022516.

Key clinical point: Proportion of patients with psoriatic arthritis with at least 20% improvement in American College of Rheumatology (ACR20) response was significantly higher with upadacitinib than placebo; however, adverse events were more frequent with upadacitinib.

Major finding: The percentage of patients with ACR20 response at week 12 was higher with upadacitinib 15 mg (70.6%) and 30 mg (78.5%) vs. placebo (36.2%; P less than .001). Incidence of serious infections and serious adverse events with upadacitinib 15 mg, 30 mg, and placebo were 1.2%, 2.6%, and 0.9% and 3.3%, 6.1%, and 3.1%, respectively.

Study details: Findings are from SELECT-Psa 1, a phase 3 trial involving 1,704 patients with PsA who had an inadequate response to at least 1 nonbiologic disease-modifying antirheumatic drugs and were randomly allocated to receive either oral upadacitinib 15 or 30 mg once daily, placebo, or subcutaneous adalimumab (40 mg every other week).

Disclosures: The trial was sponsored by Abbvie. The authors reported receiving consulting fees, advisory board fees, lecture fees, travel support, grant support, and/or being an employee of and/or owning stocks in various pharmaceutical companies, including Abbvie.

Source: McInnes IB et al. N Engl J Med. 2021 Apr 1. doi: 10.1056/NEJMoa2022516.

Key clinical point: Proportion of patients with psoriatic arthritis with at least 20% improvement in American College of Rheumatology (ACR20) response was significantly higher with upadacitinib than placebo; however, adverse events were more frequent with upadacitinib.

Major finding: The percentage of patients with ACR20 response at week 12 was higher with upadacitinib 15 mg (70.6%) and 30 mg (78.5%) vs. placebo (36.2%; P less than .001). Incidence of serious infections and serious adverse events with upadacitinib 15 mg, 30 mg, and placebo were 1.2%, 2.6%, and 0.9% and 3.3%, 6.1%, and 3.1%, respectively.

Study details: Findings are from SELECT-Psa 1, a phase 3 trial involving 1,704 patients with PsA who had an inadequate response to at least 1 nonbiologic disease-modifying antirheumatic drugs and were randomly allocated to receive either oral upadacitinib 15 or 30 mg once daily, placebo, or subcutaneous adalimumab (40 mg every other week).

Disclosures: The trial was sponsored by Abbvie. The authors reported receiving consulting fees, advisory board fees, lecture fees, travel support, grant support, and/or being an employee of and/or owning stocks in various pharmaceutical companies, including Abbvie.

Source: McInnes IB et al. N Engl J Med. 2021 Apr 1. doi: 10.1056/NEJMoa2022516.

Human leukocyte antigen (HLA)-mismatched hematopoetic stem cell microtransplantation (MST) has previously been evaluated in AML in combination with chemotherapy and suggests potential improvement in outcomes. Li et al evaluated MST combined with decitabine in patients with intermediate or high risk MDS compared to decitabine alone.  Out of 22 patients in the study, the cohort that received MST with decitabine had higher median OS (24 vs 14.2 months, p=0.04), although there was no statistical significant difference in overall response rate. There was no reported graft vs host disease, and adverse events were similar between the two groups. The main limitation of the study was the very small sample size; further prospective studies utilizing MST in MDS are needed to demonstrate benefit.

MDS patients often present with neutropenia and develop worsening neutropenia due to myelosuppression from HMA therapy, resulting in increased risk of infections including invasive fungal infection (IFI). Tey et al reported a retrospective analysis of the rate of IFI in patients with MDS or low blast count AML receiving azacitidine. Out of 117 patients, 61% received antifungal prophylaxis, either with posaconazole (n=70) or voriconazole (n=1). The IFI rate was 7.7% in the cohort with median time of onset of 74 days from start of azacitidine treatment (range 1-226); the IFI rate did not differ statistically between those receiving prophylaxis vs not (5.6% vs 10.9%, p=0.30). However, presence of neutropenia at three months of treatment was associated with increased IFI risk (HR 8.29, p=0.01), and IFI was associated with increased mortality in a multivariate analysis (HR 8.37, p<0.0001). Anti-fungal prophylaxis is currently standard practice for MDS patients who present or develop neutropenia; however, HMA treatment is associated with prolonged neutropenia due to myelosuppression and time to response. A more effective therapy with less myelosuppression is needed for treatment of MDS.

Human leukocyte antigen (HLA)-mismatched hematopoetic stem cell microtransplantation (MST) has previously been evaluated in AML in combination with chemotherapy and suggests potential improvement in outcomes. Li et al evaluated MST combined with decitabine in patients with intermediate or high risk MDS compared to decitabine alone.  Out of 22 patients in the study, the cohort that received MST with decitabine had higher median OS (24 vs 14.2 months, p=0.04), although there was no statistical significant difference in overall response rate. There was no reported graft vs host disease, and adverse events were similar between the two groups. The main limitation of the study was the very small sample size; further prospective studies utilizing MST in MDS are needed to demonstrate benefit.

MDS patients often present with neutropenia and develop worsening neutropenia due to myelosuppression from HMA therapy, resulting in increased risk of infections including invasive fungal infection (IFI). Tey et al reported a retrospective analysis of the rate of IFI in patients with MDS or low blast count AML receiving azacitidine. Out of 117 patients, 61% received antifungal prophylaxis, either with posaconazole (n=70) or voriconazole (n=1). The IFI rate was 7.7% in the cohort with median time of onset of 74 days from start of azacitidine treatment (range 1-226); the IFI rate did not differ statistically between those receiving prophylaxis vs not (5.6% vs 10.9%, p=0.30). However, presence of neutropenia at three months of treatment was associated with increased IFI risk (HR 8.29, p=0.01), and IFI was associated with increased mortality in a multivariate analysis (HR 8.37, p<0.0001). Anti-fungal prophylaxis is currently standard practice for MDS patients who present or develop neutropenia; however, HMA treatment is associated with prolonged neutropenia due to myelosuppression and time to response. A more effective therapy with less myelosuppression is needed for treatment of MDS.

Human leukocyte antigen (HLA)-mismatched hematopoetic stem cell microtransplantation (MST) has previously been evaluated in AML in combination with chemotherapy and suggests potential improvement in outcomes. Li et al evaluated MST combined with decitabine in patients with intermediate or high risk MDS compared to decitabine alone.  Out of 22 patients in the study, the cohort that received MST with decitabine had higher median OS (24 vs 14.2 months, p=0.04), although there was no statistical significant difference in overall response rate. There was no reported graft vs host disease, and adverse events were similar between the two groups. The main limitation of the study was the very small sample size; further prospective studies utilizing MST in MDS are needed to demonstrate benefit.

MDS patients often present with neutropenia and develop worsening neutropenia due to myelosuppression from HMA therapy, resulting in increased risk of infections including invasive fungal infection (IFI). Tey et al reported a retrospective analysis of the rate of IFI in patients with MDS or low blast count AML receiving azacitidine. Out of 117 patients, 61% received antifungal prophylaxis, either with posaconazole (n=70) or voriconazole (n=1). The IFI rate was 7.7% in the cohort with median time of onset of 74 days from start of azacitidine treatment (range 1-226); the IFI rate did not differ statistically between those receiving prophylaxis vs not (5.6% vs 10.9%, p=0.30). However, presence of neutropenia at three months of treatment was associated with increased IFI risk (HR 8.29, p=0.01), and IFI was associated with increased mortality in a multivariate analysis (HR 8.37, p<0.0001). Anti-fungal prophylaxis is currently standard practice for MDS patients who present or develop neutropenia; however, HMA treatment is associated with prolonged neutropenia due to myelosuppression and time to response. A more effective therapy with less myelosuppression is needed for treatment of MDS.

Key clinical point: Combination of decitabine and human leukocyte antigen-mismatched hematopoietic stem cell microtransplantation (HLA-MST) could be an effective and safe treatment in patients with intermediate- or high-risk myelodysplastic syndrome (MDS).

Major finding: Overall survival was significantly higher in the decitabine and HLA-MST combination vs. decitabine alone group (24.00 vs. 14.13 months; hazard ratio, 0.32; P = .04). The incidence of hematological adverse events except thrombocytopenia and nonhematological adverse events was slightly lower in decitabine+HLA-MST vs. decitabine alone group.

Study details: Findings are from a retrospective study of 22 patients with intermediate- or high-risk MDS who were treated with decitabine (n=11) or decitabine and HLA-MST combination (n=11).

Disclosures: The study was funded by National Natural Science Foundation of China; the Science and Technology Planning Project of Guangdong Province, China; the Guangzhou Regenerative Medicine and Health Guangdong Laboratory; and the Project of Guangdong Province Traditional Chinese Medicine Bureau. The authors declared absence of any commercial or financial relationships during conduct of the study.

Source: Li MM et al. Front Oncol. 2021 Mar 31. doi: 10.3389/fonc.2021.628127.

Key clinical point: Combination of decitabine and human leukocyte antigen-mismatched hematopoietic stem cell microtransplantation (HLA-MST) could be an effective and safe treatment in patients with intermediate- or high-risk myelodysplastic syndrome (MDS).

Major finding: Overall survival was significantly higher in the decitabine and HLA-MST combination vs. decitabine alone group (24.00 vs. 14.13 months; hazard ratio, 0.32; P = .04). The incidence of hematological adverse events except thrombocytopenia and nonhematological adverse events was slightly lower in decitabine+HLA-MST vs. decitabine alone group.

Study details: Findings are from a retrospective study of 22 patients with intermediate- or high-risk MDS who were treated with decitabine (n=11) or decitabine and HLA-MST combination (n=11).

Disclosures: The study was funded by National Natural Science Foundation of China; the Science and Technology Planning Project of Guangdong Province, China; the Guangzhou Regenerative Medicine and Health Guangdong Laboratory; and the Project of Guangdong Province Traditional Chinese Medicine Bureau. The authors declared absence of any commercial or financial relationships during conduct of the study.

Source: Li MM et al. Front Oncol. 2021 Mar 31. doi: 10.3389/fonc.2021.628127.

Key clinical point: Combination of decitabine and human leukocyte antigen-mismatched hematopoietic stem cell microtransplantation (HLA-MST) could be an effective and safe treatment in patients with intermediate- or high-risk myelodysplastic syndrome (MDS).

Major finding: Overall survival was significantly higher in the decitabine and HLA-MST combination vs. decitabine alone group (24.00 vs. 14.13 months; hazard ratio, 0.32; P = .04). The incidence of hematological adverse events except thrombocytopenia and nonhematological adverse events was slightly lower in decitabine+HLA-MST vs. decitabine alone group.

Study details: Findings are from a retrospective study of 22 patients with intermediate- or high-risk MDS who were treated with decitabine (n=11) or decitabine and HLA-MST combination (n=11).

Disclosures: The study was funded by National Natural Science Foundation of China; the Science and Technology Planning Project of Guangdong Province, China; the Guangzhou Regenerative Medicine and Health Guangdong Laboratory; and the Project of Guangdong Province Traditional Chinese Medicine Bureau. The authors declared absence of any commercial or financial relationships during conduct of the study.

Source: Li MM et al. Front Oncol. 2021 Mar 31. doi: 10.3389/fonc.2021.628127.

Key clinical point: Higher doses of hypomethylating agents, azacytidine (AZA) and decitabine (DAC), can help achieve higher rates of transfusion independence (TI) in patients with low-risk myelodysplastic syndrome (MDS). However, benefits should be weighed against the risk for adverse events.

Major finding: TI rate was higher with AZA (75 mg/m²/day for 7 days) than other regimens (P less than .025). The rate of grade 3/4 anemia was higher (15.8% vs. 0.0%; P less than .0001) and that of diarrhea/constipation (6.9% vs. 25.0%; P = .002) was lower with DAC (20 mg/m²/day for 3 days) vs. AZA (75 mg/m²/day for 5 days).

Study details: Findings are from a meta-analysis of 19 prospective studies including 1,076 patients with low-risk MDS.

Disclosures: The study was funded by Beijing Natural Science Foundation, the Chinese Academy of Medical Sciences (CAMS) Innovation Fund for Medical Sciences, and the Non-profit Central Research Institute Fund of CAMS. The authors declared no potential conflicts of interest.

Source: Wan Z et al. Aging (Albany NY). 2021 Mar 26. doi: 10.18632/aging.202767.

Key clinical point: Higher doses of hypomethylating agents, azacytidine (AZA) and decitabine (DAC), can help achieve higher rates of transfusion independence (TI) in patients with low-risk myelodysplastic syndrome (MDS). However, benefits should be weighed against the risk for adverse events.

Major finding: TI rate was higher with AZA (75 mg/m²/day for 7 days) than other regimens (P less than .025). The rate of grade 3/4 anemia was higher (15.8% vs. 0.0%; P less than .0001) and that of diarrhea/constipation (6.9% vs. 25.0%; P = .002) was lower with DAC (20 mg/m²/day for 3 days) vs. AZA (75 mg/m²/day for 5 days).

Study details: Findings are from a meta-analysis of 19 prospective studies including 1,076 patients with low-risk MDS.

Disclosures: The study was funded by Beijing Natural Science Foundation, the Chinese Academy of Medical Sciences (CAMS) Innovation Fund for Medical Sciences, and the Non-profit Central Research Institute Fund of CAMS. The authors declared no potential conflicts of interest.

Source: Wan Z et al. Aging (Albany NY). 2021 Mar 26. doi: 10.18632/aging.202767.

Key clinical point: Higher doses of hypomethylating agents, azacytidine (AZA) and decitabine (DAC), can help achieve higher rates of transfusion independence (TI) in patients with low-risk myelodysplastic syndrome (MDS). However, benefits should be weighed against the risk for adverse events.

Major finding: TI rate was higher with AZA (75 mg/m²/day for 7 days) than other regimens (P less than .025). The rate of grade 3/4 anemia was higher (15.8% vs. 0.0%; P less than .0001) and that of diarrhea/constipation (6.9% vs. 25.0%; P = .002) was lower with DAC (20 mg/m²/day for 3 days) vs. AZA (75 mg/m²/day for 5 days).

Study details: Findings are from a meta-analysis of 19 prospective studies including 1,076 patients with low-risk MDS.

Disclosures: The study was funded by Beijing Natural Science Foundation, the Chinese Academy of Medical Sciences (CAMS) Innovation Fund for Medical Sciences, and the Non-profit Central Research Institute Fund of CAMS. The authors declared no potential conflicts of interest.

Source: Wan Z et al. Aging (Albany NY). 2021 Mar 26. doi: 10.18632/aging.202767.

Key clinical point: Bridging therapy with hypomethylating agents (HMAs) did not provide long-term survival benefit in patients with myelodysplastic syndrome (MDS) receiving allogeneic hematopoietic stem cell transplantation (allo-HSCT).

Major finding: Overall survival was not significantly different among patients who received HMAs vs. best supportive care before allo-HSCT (hazard ratio, 0.86; P = .32).

Study details: Findings are from a meta-analysis of 7 retrospective studies including 820 patients with MDS, of which 395 patients received HMAs before allo-HSCT and the remaining patients received best supportive care.

Disclosures: The study was funded by Youth Science Foundation of National Natural Science Foundation of China. The authors declared no potential conflicts of interest.

Source: Liu L et al. Clin Exp Med. 2021 Apr 17. doi: 10.1007/s10238-021-00712-0.

Key clinical point: Bridging therapy with hypomethylating agents (HMAs) did not provide long-term survival benefit in patients with myelodysplastic syndrome (MDS) receiving allogeneic hematopoietic stem cell transplantation (allo-HSCT).

Major finding: Overall survival was not significantly different among patients who received HMAs vs. best supportive care before allo-HSCT (hazard ratio, 0.86; P = .32).

Study details: Findings are from a meta-analysis of 7 retrospective studies including 820 patients with MDS, of which 395 patients received HMAs before allo-HSCT and the remaining patients received best supportive care.

Disclosures: The study was funded by Youth Science Foundation of National Natural Science Foundation of China. The authors declared no potential conflicts of interest.

Source: Liu L et al. Clin Exp Med. 2021 Apr 17. doi: 10.1007/s10238-021-00712-0.

Key clinical point: Bridging therapy with hypomethylating agents (HMAs) did not provide long-term survival benefit in patients with myelodysplastic syndrome (MDS) receiving allogeneic hematopoietic stem cell transplantation (allo-HSCT).

Major finding: Overall survival was not significantly different among patients who received HMAs vs. best supportive care before allo-HSCT (hazard ratio, 0.86; P = .32).

Study details: Findings are from a meta-analysis of 7 retrospective studies including 820 patients with MDS, of which 395 patients received HMAs before allo-HSCT and the remaining patients received best supportive care.

Disclosures: The study was funded by Youth Science Foundation of National Natural Science Foundation of China. The authors declared no potential conflicts of interest.

Source: Liu L et al. Clin Exp Med. 2021 Apr 17. doi: 10.1007/s10238-021-00712-0.

Key clinical point: There could be a possible relationship between organizing pneumonia (OP) and karyotype der(1;7)(q10; p10) in myelodysplastic syndrome (MDS).

Major finding: Overall, 5 (3.1%) patients developed pathologically diagnosed OP, of which 3 had der(1;7)(q10; p10) abnormality. The chances of developing OP was higher among patients with vs. without der(1;7)(q10;p10) abnormality (odds ratio, 25.70; 95% confidence interval, 2.582-347.400).

Study details: Findings are from a retrospective analysis of 159 patients with high-risk MDS.

Disclosures: No funding source was identified. The authors declared no potential conflicts of interest.

Source: Matsunawa M et al. Br J Haematol. 2021 Apr 14. doi: 10.1111/bjh.17473.

Key clinical point: There could be a possible relationship between organizing pneumonia (OP) and karyotype der(1;7)(q10; p10) in myelodysplastic syndrome (MDS).

Major finding: Overall, 5 (3.1%) patients developed pathologically diagnosed OP, of which 3 had der(1;7)(q10; p10) abnormality. The chances of developing OP was higher among patients with vs. without der(1;7)(q10;p10) abnormality (odds ratio, 25.70; 95% confidence interval, 2.582-347.400).

Study details: Findings are from a retrospective analysis of 159 patients with high-risk MDS.

Disclosures: No funding source was identified. The authors declared no potential conflicts of interest.

Source: Matsunawa M et al. Br J Haematol. 2021 Apr 14. doi: 10.1111/bjh.17473.

Key clinical point: There could be a possible relationship between organizing pneumonia (OP) and karyotype der(1;7)(q10; p10) in myelodysplastic syndrome (MDS).

Major finding: Overall, 5 (3.1%) patients developed pathologically diagnosed OP, of which 3 had der(1;7)(q10; p10) abnormality. The chances of developing OP was higher among patients with vs. without der(1;7)(q10;p10) abnormality (odds ratio, 25.70; 95% confidence interval, 2.582-347.400).

Study details: Findings are from a retrospective analysis of 159 patients with high-risk MDS.

Disclosures: No funding source was identified. The authors declared no potential conflicts of interest.

Source: Matsunawa M et al. Br J Haematol. 2021 Apr 14. doi: 10.1111/bjh.17473.

Key clinical point: Quizartinib-based combinations were effective and well tolerated in both frontline and first salvage for patients with untreated myelodysplastic syndrome (MDS) and FMS-like tyrosine kinase 3-internal tandem duplication (FLT3-ITD) acute myeloid leukemia (AML).

Major finding: Frontline quizartinib/azacitidine vs. quizartinib/low-dose cytarabine (LDAC) led to a higher proportion of patients achieve composite response (87% vs. 74%) and a longer overall survival (19.2 vs. 8.5 months; P = .036). Similarly, quizartinib/azacitidine vs. quizartinib/LDAC was associated with a higher proportion of patients with relapsed/refractory AML achieve composite response (64% vs. 29%) and a trend for longer overall survival (12.8 vs. 4 months; P = .053). Overall, therapy was well tolerated in both cohorts.

Study details: In this open-label phase I/II trial, patients with untreated MDS/AML or those receiving first-salvage treatment for FLT3-ITD AML were treated with a combination of quizartinib and either azacitidine or LDAC in frontline (n=34) or as first-salvage (n=39) therapy.

Disclosures: The study was supported in part by the Cancer Center Support Grant. The authors declared no competing financial interests.

Source: Swaminathan M et al. Haematologica. 2021 Apr 15. doi: 10.3324/haematol.2020.263392.

Key clinical point: Quizartinib-based combinations were effective and well tolerated in both frontline and first salvage for patients with untreated myelodysplastic syndrome (MDS) and FMS-like tyrosine kinase 3-internal tandem duplication (FLT3-ITD) acute myeloid leukemia (AML).

Major finding: Frontline quizartinib/azacitidine vs. quizartinib/low-dose cytarabine (LDAC) led to a higher proportion of patients achieve composite response (87% vs. 74%) and a longer overall survival (19.2 vs. 8.5 months; P = .036). Similarly, quizartinib/azacitidine vs. quizartinib/LDAC was associated with a higher proportion of patients with relapsed/refractory AML achieve composite response (64% vs. 29%) and a trend for longer overall survival (12.8 vs. 4 months; P = .053). Overall, therapy was well tolerated in both cohorts.

Study details: In this open-label phase I/II trial, patients with untreated MDS/AML or those receiving first-salvage treatment for FLT3-ITD AML were treated with a combination of quizartinib and either azacitidine or LDAC in frontline (n=34) or as first-salvage (n=39) therapy.

Disclosures: The study was supported in part by the Cancer Center Support Grant. The authors declared no competing financial interests.

Source: Swaminathan M et al. Haematologica. 2021 Apr 15. doi: 10.3324/haematol.2020.263392.

Key clinical point: Quizartinib-based combinations were effective and well tolerated in both frontline and first salvage for patients with untreated myelodysplastic syndrome (MDS) and FMS-like tyrosine kinase 3-internal tandem duplication (FLT3-ITD) acute myeloid leukemia (AML).

Major finding: Frontline quizartinib/azacitidine vs. quizartinib/low-dose cytarabine (LDAC) led to a higher proportion of patients achieve composite response (87% vs. 74%) and a longer overall survival (19.2 vs. 8.5 months; P = .036). Similarly, quizartinib/azacitidine vs. quizartinib/LDAC was associated with a higher proportion of patients with relapsed/refractory AML achieve composite response (64% vs. 29%) and a trend for longer overall survival (12.8 vs. 4 months; P = .053). Overall, therapy was well tolerated in both cohorts.

Study details: In this open-label phase I/II trial, patients with untreated MDS/AML or those receiving first-salvage treatment for FLT3-ITD AML were treated with a combination of quizartinib and either azacitidine or LDAC in frontline (n=34) or as first-salvage (n=39) therapy.

Disclosures: The study was supported in part by the Cancer Center Support Grant. The authors declared no competing financial interests.

Source: Swaminathan M et al. Haematologica. 2021 Apr 15. doi: 10.3324/haematol.2020.263392.