Pediatric Dermatology: A Supplement to Pediatric News & Dermatology News

Table of Contents:

• Dupilumab curbed itch intensity, frequency in children with severe eczema
• Vitiligo treatment options abound but consider patient goals
• Beware a pair of dermatologic emergencies in children
• Database offers snapshot of common causes of pediatric allergic contact dermatitis
• Who’s at risk for depression on isotretinoin?
• Expert shares his approach to treating warts in children

Pediatric Dermatology: A Supplement to Pediatric News & Dermatology News

Table of Contents:

• Dupilumab curbed itch intensity, frequency in children with severe eczema
• Vitiligo treatment options abound but consider patient goals
• Beware a pair of dermatologic emergencies in children
• Database offers snapshot of common causes of pediatric allergic contact dermatitis
• Who’s at risk for depression on isotretinoin?
• Expert shares his approach to treating warts in children

Pediatric Dermatology: A Supplement to Pediatric News & Dermatology News

Table of Contents:

• Dupilumab curbed itch intensity, frequency in children with severe eczema
• Vitiligo treatment options abound but consider patient goals
• Beware a pair of dermatologic emergencies in children
• Database offers snapshot of common causes of pediatric allergic contact dermatitis
• Who’s at risk for depression on isotretinoin?
• Expert shares his approach to treating warts in children

Key clinical point: Venetoclax-based therapies offered good efficacy with an acceptable toxicity profile in a real-life setting of patients with relapsed/refractory acute myeloid leukemia (AML).

Major finding: During median follow-up of 10.7 months, composite complete remission was achieved in 55% of patients, of which 61% achieved minimal residual disease negativity. Overall survival and event-free survival were 10.7 and 4.5 months, respectively. Grade 4 neutropenia, thrombocytopenia, and febrile neutropenia occurred in 100%, 95%, and 45% of patients, respectively.

Study details: Data come from retrospective assessment of 47 patients with relapsed/refractory AML treated with venetoclax in combination with azacytidine, decitabine, or low-dose cytarabine.

Disclosures: This study was supported by a grant from AIRC to the MYNERVA project. The authors declared no conflicts of interest.

Source: Piccini M et al. J Clin Med. 2021 Apr 14. doi: 10.3390/jcm10081684.

Key clinical point: Venetoclax-based therapies offered good efficacy with an acceptable toxicity profile in a real-life setting of patients with relapsed/refractory acute myeloid leukemia (AML).

Major finding: During median follow-up of 10.7 months, composite complete remission was achieved in 55% of patients, of which 61% achieved minimal residual disease negativity. Overall survival and event-free survival were 10.7 and 4.5 months, respectively. Grade 4 neutropenia, thrombocytopenia, and febrile neutropenia occurred in 100%, 95%, and 45% of patients, respectively.

Study details: Data come from retrospective assessment of 47 patients with relapsed/refractory AML treated with venetoclax in combination with azacytidine, decitabine, or low-dose cytarabine.

Disclosures: This study was supported by a grant from AIRC to the MYNERVA project. The authors declared no conflicts of interest.

Source: Piccini M et al. J Clin Med. 2021 Apr 14. doi: 10.3390/jcm10081684.

Key clinical point: Venetoclax-based therapies offered good efficacy with an acceptable toxicity profile in a real-life setting of patients with relapsed/refractory acute myeloid leukemia (AML).

Major finding: During median follow-up of 10.7 months, composite complete remission was achieved in 55% of patients, of which 61% achieved minimal residual disease negativity. Overall survival and event-free survival were 10.7 and 4.5 months, respectively. Grade 4 neutropenia, thrombocytopenia, and febrile neutropenia occurred in 100%, 95%, and 45% of patients, respectively.

Study details: Data come from retrospective assessment of 47 patients with relapsed/refractory AML treated with venetoclax in combination with azacytidine, decitabine, or low-dose cytarabine.

Disclosures: This study was supported by a grant from AIRC to the MYNERVA project. The authors declared no conflicts of interest.

Source: Piccini M et al. J Clin Med. 2021 Apr 14. doi: 10.3390/jcm10081684.

Key clinical point: Age has a significant impact on the biology and outcomes of patients with acute myeloid leukemia (AML). The proportion of patients in the favorable-risk group receiving induction remission chemotherapy and rates of complete response (CR) among patients who received induction therapy decreased with age.

Major finding: Only 6% of patients older than 70 years were in the favorable-risk group, and less than 50% of patients older than 70 years received induction therapy. After induction therapy, 81.5% of patients with acute promyelocytic leukemia (APL) and 62.4% of non-APL patients achieved CR, which decreased with increasing age.

Study details: Findings are from a retrospective analysis of 3,011 adult patients with AML, of which 329 patients had APL and 2,682 were non-APL patients.

Disclosures: This study was supported by the Korean Society of Hematology AML/MDS Working Party and funding from Gachon University to H Kim. The authors declared no conflicts of interest.

Source: Yoo KH et al. PLoS One. 2021 May 7. doi: 10.1371/journal.pone.0251011.

Key clinical point: Age has a significant impact on the biology and outcomes of patients with acute myeloid leukemia (AML). The proportion of patients in the favorable-risk group receiving induction remission chemotherapy and rates of complete response (CR) among patients who received induction therapy decreased with age.

Major finding: Only 6% of patients older than 70 years were in the favorable-risk group, and less than 50% of patients older than 70 years received induction therapy. After induction therapy, 81.5% of patients with acute promyelocytic leukemia (APL) and 62.4% of non-APL patients achieved CR, which decreased with increasing age.

Study details: Findings are from a retrospective analysis of 3,011 adult patients with AML, of which 329 patients had APL and 2,682 were non-APL patients.

Disclosures: This study was supported by the Korean Society of Hematology AML/MDS Working Party and funding from Gachon University to H Kim. The authors declared no conflicts of interest.

Source: Yoo KH et al. PLoS One. 2021 May 7. doi: 10.1371/journal.pone.0251011.

Key clinical point: Age has a significant impact on the biology and outcomes of patients with acute myeloid leukemia (AML). The proportion of patients in the favorable-risk group receiving induction remission chemotherapy and rates of complete response (CR) among patients who received induction therapy decreased with age.

Major finding: Only 6% of patients older than 70 years were in the favorable-risk group, and less than 50% of patients older than 70 years received induction therapy. After induction therapy, 81.5% of patients with acute promyelocytic leukemia (APL) and 62.4% of non-APL patients achieved CR, which decreased with increasing age.

Study details: Findings are from a retrospective analysis of 3,011 adult patients with AML, of which 329 patients had APL and 2,682 were non-APL patients.

Disclosures: This study was supported by the Korean Society of Hematology AML/MDS Working Party and funding from Gachon University to H Kim. The authors declared no conflicts of interest.

Source: Yoo KH et al. PLoS One. 2021 May 7. doi: 10.1371/journal.pone.0251011.

Key clinical point: Treatment with FMS-like tyrosine kinase inhibitor (FLT3i)-based induction and allogeneic stem cell transplantation (allo-SCT) in the first complete remission (CR1) improved survival in patients with newly diagnosed acute myeloid leukemia (AML) with very low FLT3 allelic burden (0.1 or lower).

Major finding: The 5-year overall survival (OS) rates among patients who received FLT3i induction and allo-SCT in CR1 vs. those who neither received FLT3i nor allo-SCT in CR1 were 100% vs. 27% (P = .02). The 5-year OS rate among patients who did not receive FLT3i-based induction but underwent allo-SCT in CR1 was 71%. Moreover, patients who received FLT3i-based induction and underwent allo-SCT achieved the highest OS.

Study details: Findings are from the retrospective analysis of 50 patients with newly diagnosed FLT3-mutated AML. Patients received frontline chemotherapy without FLT3i (n=30) or induction therapy with FLT3i (n=20).

Disclosures: This study was partly supported by the MD Anderson Cancer Centre Support Grant. Some investigators including the lead author reported research funding, personal fees, grants, honoraria, consultancy, and advisory roles for various pharmaceutical companies.

Source: Yilmaz M et al. Am J Hematol. 2021 Apr 23. doi: 10.1002/ajh.26202.

Key clinical point: Treatment with FMS-like tyrosine kinase inhibitor (FLT3i)-based induction and allogeneic stem cell transplantation (allo-SCT) in the first complete remission (CR1) improved survival in patients with newly diagnosed acute myeloid leukemia (AML) with very low FLT3 allelic burden (0.1 or lower).

Major finding: The 5-year overall survival (OS) rates among patients who received FLT3i induction and allo-SCT in CR1 vs. those who neither received FLT3i nor allo-SCT in CR1 were 100% vs. 27% (P = .02). The 5-year OS rate among patients who did not receive FLT3i-based induction but underwent allo-SCT in CR1 was 71%. Moreover, patients who received FLT3i-based induction and underwent allo-SCT achieved the highest OS.

Study details: Findings are from the retrospective analysis of 50 patients with newly diagnosed FLT3-mutated AML. Patients received frontline chemotherapy without FLT3i (n=30) or induction therapy with FLT3i (n=20).

Disclosures: This study was partly supported by the MD Anderson Cancer Centre Support Grant. Some investigators including the lead author reported research funding, personal fees, grants, honoraria, consultancy, and advisory roles for various pharmaceutical companies.

Source: Yilmaz M et al. Am J Hematol. 2021 Apr 23. doi: 10.1002/ajh.26202.

Key clinical point: Treatment with FMS-like tyrosine kinase inhibitor (FLT3i)-based induction and allogeneic stem cell transplantation (allo-SCT) in the first complete remission (CR1) improved survival in patients with newly diagnosed acute myeloid leukemia (AML) with very low FLT3 allelic burden (0.1 or lower).

Major finding: The 5-year overall survival (OS) rates among patients who received FLT3i induction and allo-SCT in CR1 vs. those who neither received FLT3i nor allo-SCT in CR1 were 100% vs. 27% (P = .02). The 5-year OS rate among patients who did not receive FLT3i-based induction but underwent allo-SCT in CR1 was 71%. Moreover, patients who received FLT3i-based induction and underwent allo-SCT achieved the highest OS.

Study details: Findings are from the retrospective analysis of 50 patients with newly diagnosed FLT3-mutated AML. Patients received frontline chemotherapy without FLT3i (n=30) or induction therapy with FLT3i (n=20).

Disclosures: This study was partly supported by the MD Anderson Cancer Centre Support Grant. Some investigators including the lead author reported research funding, personal fees, grants, honoraria, consultancy, and advisory roles for various pharmaceutical companies.

Source: Yilmaz M et al. Am J Hematol. 2021 Apr 23. doi: 10.1002/ajh.26202.

Key clinical point: Mutation in isocitrate dehydrogenase 2 (IDH2) gene predicted a favorable response to daunorubicin, cytarabine, and cladribine (DAC) regimen in patients with normal karyotype acute myeloid leukemia (NK-AML).

Major finding: IDH2 mutation had a positive impact on overall survival in patients treated with DAC regimen (54% vs. 33%; P = .0087) but not in those treated with daunorubicin+cytarabine (DA; 21% vs. 23%; P = .22) regimen. Moreover, DAC induction was independently associated with a reduced risk for death when the observations were censored at allogeneic hematopoietic stem cell transplant (hazard ratio, 0.21; P = .02).

Study details: Findings are from a retrospective analysis of 398 patients with NK-AML (IDH2⁺, n=50) treated with DA (48%), DAC (44%), or DA+fludarabine (8%) regimens.

Disclosures: This study was supported by grants from the Polish Ministry of Science and Education, Polish National Center for Research and Development, and Nicolaus Copernicus University in Bydgoszcz. The authors declared no conflicts of interest.

Source: Libura M et al. Sci Rep. 2021 May 11. doi: 10.1038/s41598-021-88120-y.

Key clinical point: Mutation in isocitrate dehydrogenase 2 (IDH2) gene predicted a favorable response to daunorubicin, cytarabine, and cladribine (DAC) regimen in patients with normal karyotype acute myeloid leukemia (NK-AML).

Major finding: IDH2 mutation had a positive impact on overall survival in patients treated with DAC regimen (54% vs. 33%; P = .0087) but not in those treated with daunorubicin+cytarabine (DA; 21% vs. 23%; P = .22) regimen. Moreover, DAC induction was independently associated with a reduced risk for death when the observations were censored at allogeneic hematopoietic stem cell transplant (hazard ratio, 0.21; P = .02).

Study details: Findings are from a retrospective analysis of 398 patients with NK-AML (IDH2⁺, n=50) treated with DA (48%), DAC (44%), or DA+fludarabine (8%) regimens.

Disclosures: This study was supported by grants from the Polish Ministry of Science and Education, Polish National Center for Research and Development, and Nicolaus Copernicus University in Bydgoszcz. The authors declared no conflicts of interest.

Source: Libura M et al. Sci Rep. 2021 May 11. doi: 10.1038/s41598-021-88120-y.

Key clinical point: Mutation in isocitrate dehydrogenase 2 (IDH2) gene predicted a favorable response to daunorubicin, cytarabine, and cladribine (DAC) regimen in patients with normal karyotype acute myeloid leukemia (NK-AML).

Major finding: IDH2 mutation had a positive impact on overall survival in patients treated with DAC regimen (54% vs. 33%; P = .0087) but not in those treated with daunorubicin+cytarabine (DA; 21% vs. 23%; P = .22) regimen. Moreover, DAC induction was independently associated with a reduced risk for death when the observations were censored at allogeneic hematopoietic stem cell transplant (hazard ratio, 0.21; P = .02).

Study details: Findings are from a retrospective analysis of 398 patients with NK-AML (IDH2⁺, n=50) treated with DA (48%), DAC (44%), or DA+fludarabine (8%) regimens.

Disclosures: This study was supported by grants from the Polish Ministry of Science and Education, Polish National Center for Research and Development, and Nicolaus Copernicus University in Bydgoszcz. The authors declared no conflicts of interest.

Source: Libura M et al. Sci Rep. 2021 May 11. doi: 10.1038/s41598-021-88120-y.

Key clinical point: In patients with newly diagnosed and relapsed/refractory (R/R) acute myeloid leukemia (AML) evolving from myeloproliferative neoplasms (MPNs), treatment with venetoclax-based (VEN-b) regimens led to modest clinical responses with no substantial improvement in survival, but high treatment-associated hematologic toxicity and mortality.

Major finding: Median overall survival from initiation of VEN-b regimen was 4 (95% confidence interval, 2-8) months with 8-week mortality being 32%. Only 7 patients with newly diagnosed AML achieved a response, with 3 achieving complete remission, 3 achieving complete remission with incomplete count recovery, and 1 achieving partial remission. Grade 3 or higher infections were observed in 84% of patients during the initial treatment cycle, with severe hemorrhagic complications in 45% of patients.

Study details: Findings are from a retrospective analysis of 31 patients with newly diagnosed (n=14) or R/R (n=17) post-MPN-AML who were treated with VEN-b therapies.

Disclosures: This study was supported partly by a Cancer Center Support Grant from the National Cancer Institute, National Institutes of Health to MD Anderson Cancer Center. Some investigators reported financial and nonfinancial ties with various pharmaceutical companies.

Source: Masarova L et al. Blood Adv. 2021 Apr 22. doi: 10.1182/bloodadvances.2020003934.

Key clinical point: In patients with newly diagnosed and relapsed/refractory (R/R) acute myeloid leukemia (AML) evolving from myeloproliferative neoplasms (MPNs), treatment with venetoclax-based (VEN-b) regimens led to modest clinical responses with no substantial improvement in survival, but high treatment-associated hematologic toxicity and mortality.

Major finding: Median overall survival from initiation of VEN-b regimen was 4 (95% confidence interval, 2-8) months with 8-week mortality being 32%. Only 7 patients with newly diagnosed AML achieved a response, with 3 achieving complete remission, 3 achieving complete remission with incomplete count recovery, and 1 achieving partial remission. Grade 3 or higher infections were observed in 84% of patients during the initial treatment cycle, with severe hemorrhagic complications in 45% of patients.

Study details: Findings are from a retrospective analysis of 31 patients with newly diagnosed (n=14) or R/R (n=17) post-MPN-AML who were treated with VEN-b therapies.

Disclosures: This study was supported partly by a Cancer Center Support Grant from the National Cancer Institute, National Institutes of Health to MD Anderson Cancer Center. Some investigators reported financial and nonfinancial ties with various pharmaceutical companies.

Source: Masarova L et al. Blood Adv. 2021 Apr 22. doi: 10.1182/bloodadvances.2020003934.

Key clinical point: In patients with newly diagnosed and relapsed/refractory (R/R) acute myeloid leukemia (AML) evolving from myeloproliferative neoplasms (MPNs), treatment with venetoclax-based (VEN-b) regimens led to modest clinical responses with no substantial improvement in survival, but high treatment-associated hematologic toxicity and mortality.

Major finding: Median overall survival from initiation of VEN-b regimen was 4 (95% confidence interval, 2-8) months with 8-week mortality being 32%. Only 7 patients with newly diagnosed AML achieved a response, with 3 achieving complete remission, 3 achieving complete remission with incomplete count recovery, and 1 achieving partial remission. Grade 3 or higher infections were observed in 84% of patients during the initial treatment cycle, with severe hemorrhagic complications in 45% of patients.

Study details: Findings are from a retrospective analysis of 31 patients with newly diagnosed (n=14) or R/R (n=17) post-MPN-AML who were treated with VEN-b therapies.

Disclosures: This study was supported partly by a Cancer Center Support Grant from the National Cancer Institute, National Institutes of Health to MD Anderson Cancer Center. Some investigators reported financial and nonfinancial ties with various pharmaceutical companies.

Source: Masarova L et al. Blood Adv. 2021 Apr 22. doi: 10.1182/bloodadvances.2020003934.

Key clinical point: Treatment with a combination of hypomethylating agents (HMA) and venetoclax (VEN) was similarly effective in patients with acute myeloid leukemia (AML) with or without spliceosome mutations with specific mutational pairs demonstrating favorable outcomes.

Major finding: Overall response rate (89% vs. 79%; P = .32), composite complete response (79% vs. 75%; P = .65), and median overall survival (OS; 35 vs. 14 months; P = .58) were not significantly different in patients with vs. without spliceosome mutations. Co-occurrence of IDH2 and SRSF2 mutations was associated with favorable outcomes (1- and 2-year OS, 100% and 88%, respectively).

Study details: Findings are from a retrospective study of 119 adult patients with AML treated with frontline HMA+VEN-based therapy. Mutation in the spliceosome gene was observed in 39 patients.

Disclosures: No specific funding source was identified. Some investigators reported research funding, support, honoraria, consultancy, and advisory roles for various pharmaceutical companies.

Source: Lachowiez CA et al. Blood Adv. 2021 Apr 22. doi: 10.1182/bloodadvances.2020004173.

Key clinical point: Treatment with a combination of hypomethylating agents (HMA) and venetoclax (VEN) was similarly effective in patients with acute myeloid leukemia (AML) with or without spliceosome mutations with specific mutational pairs demonstrating favorable outcomes.

Major finding: Overall response rate (89% vs. 79%; P = .32), composite complete response (79% vs. 75%; P = .65), and median overall survival (OS; 35 vs. 14 months; P = .58) were not significantly different in patients with vs. without spliceosome mutations. Co-occurrence of IDH2 and SRSF2 mutations was associated with favorable outcomes (1- and 2-year OS, 100% and 88%, respectively).

Study details: Findings are from a retrospective study of 119 adult patients with AML treated with frontline HMA+VEN-based therapy. Mutation in the spliceosome gene was observed in 39 patients.

Disclosures: No specific funding source was identified. Some investigators reported research funding, support, honoraria, consultancy, and advisory roles for various pharmaceutical companies.

Source: Lachowiez CA et al. Blood Adv. 2021 Apr 22. doi: 10.1182/bloodadvances.2020004173.

Key clinical point: Treatment with a combination of hypomethylating agents (HMA) and venetoclax (VEN) was similarly effective in patients with acute myeloid leukemia (AML) with or without spliceosome mutations with specific mutational pairs demonstrating favorable outcomes.

Major finding: Overall response rate (89% vs. 79%; P = .32), composite complete response (79% vs. 75%; P = .65), and median overall survival (OS; 35 vs. 14 months; P = .58) were not significantly different in patients with vs. without spliceosome mutations. Co-occurrence of IDH2 and SRSF2 mutations was associated with favorable outcomes (1- and 2-year OS, 100% and 88%, respectively).

Study details: Findings are from a retrospective study of 119 adult patients with AML treated with frontline HMA+VEN-based therapy. Mutation in the spliceosome gene was observed in 39 patients.

Disclosures: No specific funding source was identified. Some investigators reported research funding, support, honoraria, consultancy, and advisory roles for various pharmaceutical companies.

Source: Lachowiez CA et al. Blood Adv. 2021 Apr 22. doi: 10.1182/bloodadvances.2020004173.

Key clinical point: Low-intensity chemotherapy with clofarabine or cladribine and low-dose cytarabine (LDAC) alternating with decitabine was effective and safe for the treatment of older patients with newly diagnosed acute myeloid leukemia (AML).

Major finding: Overall, response was observed in 66% of patients, with complete remission (CR) and CR with incomplete count recovery in 59% and 7% of patients, respectively. The 4- and 8-week mortality was low at 2% and 11%, respectively. During median follow-up of 60 months, the median overall survival was 12.5 months.

Study details: This study assessed 248 older patients (median age, 69 years) with newly diagnosed AML who were treated with either clofarabine (n=119) or cladribine (n=129) combined with LDAC alternating with decitabine.

Disclosures: This study was funded by the MD Anderson Cancer Center Support and the Charif Souki Cancer Research Fund. Some investigators including the lead author reported ties with various pharmaceutical companies.

Source: Kadia TM et al. Am J Hematol. 2021 Apr 26. doi: 10.1002/ajh.26206.

Key clinical point: Low-intensity chemotherapy with clofarabine or cladribine and low-dose cytarabine (LDAC) alternating with decitabine was effective and safe for the treatment of older patients with newly diagnosed acute myeloid leukemia (AML).

Major finding: Overall, response was observed in 66% of patients, with complete remission (CR) and CR with incomplete count recovery in 59% and 7% of patients, respectively. The 4- and 8-week mortality was low at 2% and 11%, respectively. During median follow-up of 60 months, the median overall survival was 12.5 months.

Study details: This study assessed 248 older patients (median age, 69 years) with newly diagnosed AML who were treated with either clofarabine (n=119) or cladribine (n=129) combined with LDAC alternating with decitabine.

Disclosures: This study was funded by the MD Anderson Cancer Center Support and the Charif Souki Cancer Research Fund. Some investigators including the lead author reported ties with various pharmaceutical companies.

Source: Kadia TM et al. Am J Hematol. 2021 Apr 26. doi: 10.1002/ajh.26206.

Key clinical point: Low-intensity chemotherapy with clofarabine or cladribine and low-dose cytarabine (LDAC) alternating with decitabine was effective and safe for the treatment of older patients with newly diagnosed acute myeloid leukemia (AML).

Major finding: Overall, response was observed in 66% of patients, with complete remission (CR) and CR with incomplete count recovery in 59% and 7% of patients, respectively. The 4- and 8-week mortality was low at 2% and 11%, respectively. During median follow-up of 60 months, the median overall survival was 12.5 months.

Study details: This study assessed 248 older patients (median age, 69 years) with newly diagnosed AML who were treated with either clofarabine (n=119) or cladribine (n=129) combined with LDAC alternating with decitabine.

Disclosures: This study was funded by the MD Anderson Cancer Center Support and the Charif Souki Cancer Research Fund. Some investigators including the lead author reported ties with various pharmaceutical companies.

Source: Kadia TM et al. Am J Hematol. 2021 Apr 26. doi: 10.1002/ajh.26206.

Key clinical point: In patients with relapsed/refractory acute myeloid leukemia (AML), mivebresib (MIV) was tolerated and showed antileukemic activity as monotherapy (MIV-mono) and in combination with venetoclax (MIV-Ven).

Major finding: In the MIV-mono cohort, response included complete remission with incomplete blood count recovery (5%) and resistant disease (79%). In patients receiving MIV-Ven, responses were complete remission (7%), partial remission (7%), leukemia-free state (7%), resistant disease (40%), and aplasia (3%). Treatment-emergent adverse events (TEAEs) were reported in 100% of patients, with serious TEAEs in 74%, 88%, and 40% of patients in MIV-mono, MIV-Ven, and patients who switched from MIV-mono to MIV-Ven groups, respectively.

Study details: Findings are from phase 1 study including 44 adult patients with relapsed/refractory AML who received either MIV-mono (n=19) or MIV-Ven (n=25). Because of disease progression, 5 patients switched from MIV-mono to MIV-Ven.

Disclosures: This study was funded by AbbVie. Investigators including the lead author reported ties with various pharmaceutical companies including AbbVie.

Source: Borthakur G et al. Cancer. 2021 May 2. doi: 10.1002/cncr.33590.

Key clinical point: In patients with relapsed/refractory acute myeloid leukemia (AML), mivebresib (MIV) was tolerated and showed antileukemic activity as monotherapy (MIV-mono) and in combination with venetoclax (MIV-Ven).

Major finding: In the MIV-mono cohort, response included complete remission with incomplete blood count recovery (5%) and resistant disease (79%). In patients receiving MIV-Ven, responses were complete remission (7%), partial remission (7%), leukemia-free state (7%), resistant disease (40%), and aplasia (3%). Treatment-emergent adverse events (TEAEs) were reported in 100% of patients, with serious TEAEs in 74%, 88%, and 40% of patients in MIV-mono, MIV-Ven, and patients who switched from MIV-mono to MIV-Ven groups, respectively.

Study details: Findings are from phase 1 study including 44 adult patients with relapsed/refractory AML who received either MIV-mono (n=19) or MIV-Ven (n=25). Because of disease progression, 5 patients switched from MIV-mono to MIV-Ven.

Disclosures: This study was funded by AbbVie. Investigators including the lead author reported ties with various pharmaceutical companies including AbbVie.

Source: Borthakur G et al. Cancer. 2021 May 2. doi: 10.1002/cncr.33590.

Key clinical point: In patients with relapsed/refractory acute myeloid leukemia (AML), mivebresib (MIV) was tolerated and showed antileukemic activity as monotherapy (MIV-mono) and in combination with venetoclax (MIV-Ven).

Major finding: In the MIV-mono cohort, response included complete remission with incomplete blood count recovery (5%) and resistant disease (79%). In patients receiving MIV-Ven, responses were complete remission (7%), partial remission (7%), leukemia-free state (7%), resistant disease (40%), and aplasia (3%). Treatment-emergent adverse events (TEAEs) were reported in 100% of patients, with serious TEAEs in 74%, 88%, and 40% of patients in MIV-mono, MIV-Ven, and patients who switched from MIV-mono to MIV-Ven groups, respectively.

Study details: Findings are from phase 1 study including 44 adult patients with relapsed/refractory AML who received either MIV-mono (n=19) or MIV-Ven (n=25). Because of disease progression, 5 patients switched from MIV-mono to MIV-Ven.

Disclosures: This study was funded by AbbVie. Investigators including the lead author reported ties with various pharmaceutical companies including AbbVie.

Source: Borthakur G et al. Cancer. 2021 May 2. doi: 10.1002/cncr.33590.

Key clinical point: Addition of tosedostat to low-dose cytosine arabinoside (LDAC) did not confer any additional clinical benefit compared with LDAC alone in older patients with acute myeloid leukemia (AML) unfit for intensive chemotherapy.

Major finding: There was no significant benefit of adding tosedostat to LDAC vs. LDAC alone on overall response (25% vs. 18%; P = .22) and 2-year overall survival (16% vs. 12%; P = .8). Overall rates of grade 3 or higher toxicity were low, but diarrhea and cardiac toxicity were significantly higher with tosedostat.

Study details: Findings are from LI-1 trial including 243 older (age, 60 years or older) patients with de novo or secondary AML or high-risk myelodysplastic syndrome who were unfit for intensive chemotherapy. Patients were randomly allocated to receive either LDAC (n=121) or LDAC+tosedostat (n=122).

Disclosures: This study was supported by CTI Biopharma, Blood Cancer UK, the Haematology Clinical Trials Unit, and the Centre for Trials Research, Cardiff University. No author disclosures were reported.

Source: Dennis M et al. Br J Haematol. 2021 May 7. doi: 10.1111/bjh.17501.

Key clinical point: Addition of tosedostat to low-dose cytosine arabinoside (LDAC) did not confer any additional clinical benefit compared with LDAC alone in older patients with acute myeloid leukemia (AML) unfit for intensive chemotherapy.

Major finding: There was no significant benefit of adding tosedostat to LDAC vs. LDAC alone on overall response (25% vs. 18%; P = .22) and 2-year overall survival (16% vs. 12%; P = .8). Overall rates of grade 3 or higher toxicity were low, but diarrhea and cardiac toxicity were significantly higher with tosedostat.

Study details: Findings are from LI-1 trial including 243 older (age, 60 years or older) patients with de novo or secondary AML or high-risk myelodysplastic syndrome who were unfit for intensive chemotherapy. Patients were randomly allocated to receive either LDAC (n=121) or LDAC+tosedostat (n=122).

Disclosures: This study was supported by CTI Biopharma, Blood Cancer UK, the Haematology Clinical Trials Unit, and the Centre for Trials Research, Cardiff University. No author disclosures were reported.

Source: Dennis M et al. Br J Haematol. 2021 May 7. doi: 10.1111/bjh.17501.

Key clinical point: Addition of tosedostat to low-dose cytosine arabinoside (LDAC) did not confer any additional clinical benefit compared with LDAC alone in older patients with acute myeloid leukemia (AML) unfit for intensive chemotherapy.

Major finding: There was no significant benefit of adding tosedostat to LDAC vs. LDAC alone on overall response (25% vs. 18%; P = .22) and 2-year overall survival (16% vs. 12%; P = .8). Overall rates of grade 3 or higher toxicity were low, but diarrhea and cardiac toxicity were significantly higher with tosedostat.

Study details: Findings are from LI-1 trial including 243 older (age, 60 years or older) patients with de novo or secondary AML or high-risk myelodysplastic syndrome who were unfit for intensive chemotherapy. Patients were randomly allocated to receive either LDAC (n=121) or LDAC+tosedostat (n=122).

Disclosures: This study was supported by CTI Biopharma, Blood Cancer UK, the Haematology Clinical Trials Unit, and the Centre for Trials Research, Cardiff University. No author disclosures were reported.

Source: Dennis M et al. Br J Haematol. 2021 May 7. doi: 10.1111/bjh.17501.