An investigational, orally dissolving film formulation of dexmedetomidine (BXCL501, BioXcel Therapeutics) may offer rapid relief from acute agitation related to schizophrenia or bipolar disorder (BD), results of two phase 3, randomized, placebo-controlled trials show.
 

For both disorders, BXCL501 showed “superiority over placebo” by meeting the primary endpoint of reduction of agitation as measured by the excited component of the Positive and Negative Syndrome Scale (PANSS), study investigator Leslie Citrome, MD, MPH, department of psychiatry and behavioral sciences, New York Medical College, Valhalla, said in an interview.

The findings were presented at the annual meeting of the American Psychiatric Association, which was held as a virtual live event.
 

Noninvasive option

Acute agitation in patients with schizophrenia or BD is often encountered in emergency departments (EDs) and inpatient units. When nondrug tactics fail to calm the patient, drug options include injectable antipsychotics or benzodiazepines. BXCL501 is a thin, orally dissolving film for sublingual or buccal use.

“Dexmedetomidine is a highly-selective alpha-2a receptor agonist and we haven’t really had one of those before in psychiatry for this purpose. And we haven’t had much in the way of orally dissolving thin films that are absorbed in the oral mucosa so this represents an opportunity to provide a potential intervention that does not require an injection and yet could possibly be of use in people who are agitated,” Dr. Citrome said.

The study, known as SERENITY I, included 380 adults (mean age 45.6 years, 63% male) with schizophrenia, schizoaffective disorder, or schizophreniform disorder, and acute agitation in the ED (total score ≥ 14 on the PANSS-Excited Component (PEC) scale at baseline and a score ≥ 4 on at least one of the five PEC items).

Patients were randomly allocated to a single oral dose of BXCL501: 120 mcg, 180 mcg, or placebo. A total of 372 patients (97.9%) completed the study.

Mean PEC total score was 17.6 at baseline. The mean change from baseline in the PEC total score at 2 hours (primary endpoint) was -8.5 and -10.3 with BXCL501 120 mcg and 180 mcg, respectively, versus -4.8 for placebo (P < .0001 vs. placebo).

PEC response rates (≥ 40% reduction from baseline) were 80.6% and 89.6% with BXCL501 120 mcg and 180 mcg versus 47.6% with placebo (P < .0001 vs. placebo).

Compared with placebo, significant improvement in the Clinical Global Impression–Improvement scale (CGI-I) was observed with both BXCL501 doses at 1 and 2 hours after dosing and in the Agitation and Calmness Evaluation Scale (ACES) at 2 hours post dosing.

The incidence of adverse events (AE) was 39.5%, 37.3%, and 15.1% with BXCL501 120 mg, 180 mg, and placebo groups.

All AEs were mild or moderate. The most common AEs with BXCL501 were somnolence, dizziness, dry mouth, hypotension, orthostatic hypotension, hypoesthesia, and paresthesia. No drug-related severe or serious AEs occurred.
 

Nipping it in the bud

SERENITY II had a similar design. This study included 380 adults (mean age 48, 55% female) with bipolar I or II disorder and acute agitation in the ED (total score ≥14 on the PEC scale at baseline and a score ≥4 on at least one PEC item). A total of 362 (95.3%) of patients completed the study.

Mean PEC total score was 18 at baseline. The mean change from baseline in the PEC total score at 2 hours (primary endpoint) was -9.0 and -10.4 with BXCL501 120 mcg and 180 mcg, respectively, versus -4.9 for placebo (P < .0001 vs. placebo).

Bipolar patients also saw significant improvement on the secondary outcomes of CGI-I and ACES, with an adverse event profile similar to that seen in patients with schizophrenia.

BXCL501 demonstrated “rapid, robust and clinically meaningful efficacy” in both patient populations and represents a “novel, noninvasive and well tolerated treatment of agitation,” the investigators concluded in their APA abstracts.

“Patients who are agitated are in psychic pain and they want relief from this psychic pain. We’re also worried that they might get worse and that agitation escalates to aggression potentially requiring restraints. We want to avoid that,” Dr. Citrome said.

“By nipping it in the bud with a pharmacological intervention, we can ease their psychic pain and we can manage a potentially dangerous situation. Offering an oral medicine that would work quickly would be ideal in my mind and patients might potentially be more accepting of that than an injection,” Dr. Citrome said.

Based on the SERENITY I and II data, BioXcel Therapeutics has submitted a new drug application to the Food and Drug Administration.
 

Negotiation first, medication second 

Reached for comment, Samoon Ahmad, MD, professor, department of psychiatry, New York (N.Y.) University, cautioned that, “when we talk about treating an agitated patient, medication is only part of the picture.

“There is a negotiating process with the patient. Number one, you offer them an environment that is conducive to making them feel calm, safe, and secure and that they are being listened to. Providing all of those things sometimes can be very helpful,” said Dr. Ahmad, who serves as unit chief of inpatient psychiatry at Bellevue Hospital Center in New York City.

“If someone starts throwing chairs at you or assaulting you, that is not really the time to negotiate a medicine; you basically have to restrain the patient, and many times give them intramuscular medicine,” Dr. Ahmad said.

He also noted that patients in the SERENITY trials had moderate to severe acute agitation.

“These are people you can potentially negotiate with. But again, when a patient crosses a certain line, you have to immediately do something and that could be intramuscular injection or something oral, which they may spit right in your face, which has happened numerous times,” Dr. Ahmad said.

“I don’t think intramuscular options will ever go away but an oral agent could be a useful tool as well,” said Dr. Ahmad, founder of the Integrative Center for Wellness in New York City.

He cautioned that clinicians are not going to be using this medicine in their offices. “If a patient walks in and is floridly psychotic, you will need to call 911. We’re really talking about its use either in the ED or acute inpatient setting,” Dr. Ahmad said.

A version of this article first appeared on Medscape.com.

An investigational, orally dissolving film formulation of dexmedetomidine (BXCL501, BioXcel Therapeutics) may offer rapid relief from acute agitation related to schizophrenia or bipolar disorder (BD), results of two phase 3, randomized, placebo-controlled trials show.
 

For both disorders, BXCL501 showed “superiority over placebo” by meeting the primary endpoint of reduction of agitation as measured by the excited component of the Positive and Negative Syndrome Scale (PANSS), study investigator Leslie Citrome, MD, MPH, department of psychiatry and behavioral sciences, New York Medical College, Valhalla, said in an interview.

The findings were presented at the annual meeting of the American Psychiatric Association, which was held as a virtual live event.
 

Noninvasive option

Acute agitation in patients with schizophrenia or BD is often encountered in emergency departments (EDs) and inpatient units. When nondrug tactics fail to calm the patient, drug options include injectable antipsychotics or benzodiazepines. BXCL501 is a thin, orally dissolving film for sublingual or buccal use.

“Dexmedetomidine is a highly-selective alpha-2a receptor agonist and we haven’t really had one of those before in psychiatry for this purpose. And we haven’t had much in the way of orally dissolving thin films that are absorbed in the oral mucosa so this represents an opportunity to provide a potential intervention that does not require an injection and yet could possibly be of use in people who are agitated,” Dr. Citrome said.

The study, known as SERENITY I, included 380 adults (mean age 45.6 years, 63% male) with schizophrenia, schizoaffective disorder, or schizophreniform disorder, and acute agitation in the ED (total score ≥ 14 on the PANSS-Excited Component (PEC) scale at baseline and a score ≥ 4 on at least one of the five PEC items).

Patients were randomly allocated to a single oral dose of BXCL501: 120 mcg, 180 mcg, or placebo. A total of 372 patients (97.9%) completed the study.

Mean PEC total score was 17.6 at baseline. The mean change from baseline in the PEC total score at 2 hours (primary endpoint) was -8.5 and -10.3 with BXCL501 120 mcg and 180 mcg, respectively, versus -4.8 for placebo (P < .0001 vs. placebo).

PEC response rates (≥ 40% reduction from baseline) were 80.6% and 89.6% with BXCL501 120 mcg and 180 mcg versus 47.6% with placebo (P < .0001 vs. placebo).

Compared with placebo, significant improvement in the Clinical Global Impression–Improvement scale (CGI-I) was observed with both BXCL501 doses at 1 and 2 hours after dosing and in the Agitation and Calmness Evaluation Scale (ACES) at 2 hours post dosing.

The incidence of adverse events (AE) was 39.5%, 37.3%, and 15.1% with BXCL501 120 mg, 180 mg, and placebo groups.

All AEs were mild or moderate. The most common AEs with BXCL501 were somnolence, dizziness, dry mouth, hypotension, orthostatic hypotension, hypoesthesia, and paresthesia. No drug-related severe or serious AEs occurred.
 

Nipping it in the bud

SERENITY II had a similar design. This study included 380 adults (mean age 48, 55% female) with bipolar I or II disorder and acute agitation in the ED (total score ≥14 on the PEC scale at baseline and a score ≥4 on at least one PEC item). A total of 362 (95.3%) of patients completed the study.

Mean PEC total score was 18 at baseline. The mean change from baseline in the PEC total score at 2 hours (primary endpoint) was -9.0 and -10.4 with BXCL501 120 mcg and 180 mcg, respectively, versus -4.9 for placebo (P < .0001 vs. placebo).

Bipolar patients also saw significant improvement on the secondary outcomes of CGI-I and ACES, with an adverse event profile similar to that seen in patients with schizophrenia.

BXCL501 demonstrated “rapid, robust and clinically meaningful efficacy” in both patient populations and represents a “novel, noninvasive and well tolerated treatment of agitation,” the investigators concluded in their APA abstracts.

“Patients who are agitated are in psychic pain and they want relief from this psychic pain. We’re also worried that they might get worse and that agitation escalates to aggression potentially requiring restraints. We want to avoid that,” Dr. Citrome said.

“By nipping it in the bud with a pharmacological intervention, we can ease their psychic pain and we can manage a potentially dangerous situation. Offering an oral medicine that would work quickly would be ideal in my mind and patients might potentially be more accepting of that than an injection,” Dr. Citrome said.

Based on the SERENITY I and II data, BioXcel Therapeutics has submitted a new drug application to the Food and Drug Administration.
 

Negotiation first, medication second 

Reached for comment, Samoon Ahmad, MD, professor, department of psychiatry, New York (N.Y.) University, cautioned that, “when we talk about treating an agitated patient, medication is only part of the picture.

“There is a negotiating process with the patient. Number one, you offer them an environment that is conducive to making them feel calm, safe, and secure and that they are being listened to. Providing all of those things sometimes can be very helpful,” said Dr. Ahmad, who serves as unit chief of inpatient psychiatry at Bellevue Hospital Center in New York City.

“If someone starts throwing chairs at you or assaulting you, that is not really the time to negotiate a medicine; you basically have to restrain the patient, and many times give them intramuscular medicine,” Dr. Ahmad said.

He also noted that patients in the SERENITY trials had moderate to severe acute agitation.

“These are people you can potentially negotiate with. But again, when a patient crosses a certain line, you have to immediately do something and that could be intramuscular injection or something oral, which they may spit right in your face, which has happened numerous times,” Dr. Ahmad said.

“I don’t think intramuscular options will ever go away but an oral agent could be a useful tool as well,” said Dr. Ahmad, founder of the Integrative Center for Wellness in New York City.

He cautioned that clinicians are not going to be using this medicine in their offices. “If a patient walks in and is floridly psychotic, you will need to call 911. We’re really talking about its use either in the ED or acute inpatient setting,” Dr. Ahmad said.

A version of this article first appeared on Medscape.com.

An investigational, orally dissolving film formulation of dexmedetomidine (BXCL501, BioXcel Therapeutics) may offer rapid relief from acute agitation related to schizophrenia or bipolar disorder (BD), results of two phase 3, randomized, placebo-controlled trials show.
 

For both disorders, BXCL501 showed “superiority over placebo” by meeting the primary endpoint of reduction of agitation as measured by the excited component of the Positive and Negative Syndrome Scale (PANSS), study investigator Leslie Citrome, MD, MPH, department of psychiatry and behavioral sciences, New York Medical College, Valhalla, said in an interview.

The findings were presented at the annual meeting of the American Psychiatric Association, which was held as a virtual live event.
 

Noninvasive option

Acute agitation in patients with schizophrenia or BD is often encountered in emergency departments (EDs) and inpatient units. When nondrug tactics fail to calm the patient, drug options include injectable antipsychotics or benzodiazepines. BXCL501 is a thin, orally dissolving film for sublingual or buccal use.

“Dexmedetomidine is a highly-selective alpha-2a receptor agonist and we haven’t really had one of those before in psychiatry for this purpose. And we haven’t had much in the way of orally dissolving thin films that are absorbed in the oral mucosa so this represents an opportunity to provide a potential intervention that does not require an injection and yet could possibly be of use in people who are agitated,” Dr. Citrome said.

The study, known as SERENITY I, included 380 adults (mean age 45.6 years, 63% male) with schizophrenia, schizoaffective disorder, or schizophreniform disorder, and acute agitation in the ED (total score ≥ 14 on the PANSS-Excited Component (PEC) scale at baseline and a score ≥ 4 on at least one of the five PEC items).

Patients were randomly allocated to a single oral dose of BXCL501: 120 mcg, 180 mcg, or placebo. A total of 372 patients (97.9%) completed the study.

Mean PEC total score was 17.6 at baseline. The mean change from baseline in the PEC total score at 2 hours (primary endpoint) was -8.5 and -10.3 with BXCL501 120 mcg and 180 mcg, respectively, versus -4.8 for placebo (P < .0001 vs. placebo).

PEC response rates (≥ 40% reduction from baseline) were 80.6% and 89.6% with BXCL501 120 mcg and 180 mcg versus 47.6% with placebo (P < .0001 vs. placebo).

Compared with placebo, significant improvement in the Clinical Global Impression–Improvement scale (CGI-I) was observed with both BXCL501 doses at 1 and 2 hours after dosing and in the Agitation and Calmness Evaluation Scale (ACES) at 2 hours post dosing.

The incidence of adverse events (AE) was 39.5%, 37.3%, and 15.1% with BXCL501 120 mg, 180 mg, and placebo groups.

All AEs were mild or moderate. The most common AEs with BXCL501 were somnolence, dizziness, dry mouth, hypotension, orthostatic hypotension, hypoesthesia, and paresthesia. No drug-related severe or serious AEs occurred.
 

Nipping it in the bud

SERENITY II had a similar design. This study included 380 adults (mean age 48, 55% female) with bipolar I or II disorder and acute agitation in the ED (total score ≥14 on the PEC scale at baseline and a score ≥4 on at least one PEC item). A total of 362 (95.3%) of patients completed the study.

Mean PEC total score was 18 at baseline. The mean change from baseline in the PEC total score at 2 hours (primary endpoint) was -9.0 and -10.4 with BXCL501 120 mcg and 180 mcg, respectively, versus -4.9 for placebo (P < .0001 vs. placebo).

Bipolar patients also saw significant improvement on the secondary outcomes of CGI-I and ACES, with an adverse event profile similar to that seen in patients with schizophrenia.

BXCL501 demonstrated “rapid, robust and clinically meaningful efficacy” in both patient populations and represents a “novel, noninvasive and well tolerated treatment of agitation,” the investigators concluded in their APA abstracts.

“Patients who are agitated are in psychic pain and they want relief from this psychic pain. We’re also worried that they might get worse and that agitation escalates to aggression potentially requiring restraints. We want to avoid that,” Dr. Citrome said.

“By nipping it in the bud with a pharmacological intervention, we can ease their psychic pain and we can manage a potentially dangerous situation. Offering an oral medicine that would work quickly would be ideal in my mind and patients might potentially be more accepting of that than an injection,” Dr. Citrome said.

Based on the SERENITY I and II data, BioXcel Therapeutics has submitted a new drug application to the Food and Drug Administration.
 

Negotiation first, medication second 

Reached for comment, Samoon Ahmad, MD, professor, department of psychiatry, New York (N.Y.) University, cautioned that, “when we talk about treating an agitated patient, medication is only part of the picture.

“There is a negotiating process with the patient. Number one, you offer them an environment that is conducive to making them feel calm, safe, and secure and that they are being listened to. Providing all of those things sometimes can be very helpful,” said Dr. Ahmad, who serves as unit chief of inpatient psychiatry at Bellevue Hospital Center in New York City.

“If someone starts throwing chairs at you or assaulting you, that is not really the time to negotiate a medicine; you basically have to restrain the patient, and many times give them intramuscular medicine,” Dr. Ahmad said.

He also noted that patients in the SERENITY trials had moderate to severe acute agitation.

“These are people you can potentially negotiate with. But again, when a patient crosses a certain line, you have to immediately do something and that could be intramuscular injection or something oral, which they may spit right in your face, which has happened numerous times,” Dr. Ahmad said.

“I don’t think intramuscular options will ever go away but an oral agent could be a useful tool as well,” said Dr. Ahmad, founder of the Integrative Center for Wellness in New York City.

He cautioned that clinicians are not going to be using this medicine in their offices. “If a patient walks in and is floridly psychotic, you will need to call 911. We’re really talking about its use either in the ED or acute inpatient setting,” Dr. Ahmad said.

A version of this article first appeared on Medscape.com.

Key clinical point: Long-term survival in children with acute myeloid leukemia (AML) who receive low-dose chemotherapy (LDC) for remission induction is comparable to that of children who receive standard-dose chemotherapy (SDC).

Major finding:  No significant differences were observed between the LDC and SDC groups in 5-year event-free survival (61.4% ± 8.7% vs 65.2% ± 7.4%, respectively; P = .462), overall survival (72.7% ± 6.9% vs 72.5% ± 6.2%, respectively; P = .933), and the incidence of relapse (20.5% ± 4.5% vs 17.6% ± 3.9%, respectively; P = .484).

Study details: Long-term follow-up of 83 patients with AML treated with a LDC regimen (and 100 children with AML treated with a standard-dose chemotherapy (SDC) regimen.

Disclosures: This work was supported by NSCF, the Key Research Program of the Chinese Academy of Sciences, the Jiangsu Province Foundation, the National Clinical Research Center for Hematological Disorders, and the Youth Innovation Promotion Association of Chinese Academy of Sciences. The authors declared no conflicts of interest.

Source: Hu Y et al. Blood Adv. 2021 Apr 13. doi: 10.1182/bloodadvances.2020003453.

Key clinical point: Long-term survival in children with acute myeloid leukemia (AML) who receive low-dose chemotherapy (LDC) for remission induction is comparable to that of children who receive standard-dose chemotherapy (SDC).

Major finding:  No significant differences were observed between the LDC and SDC groups in 5-year event-free survival (61.4% ± 8.7% vs 65.2% ± 7.4%, respectively; P = .462), overall survival (72.7% ± 6.9% vs 72.5% ± 6.2%, respectively; P = .933), and the incidence of relapse (20.5% ± 4.5% vs 17.6% ± 3.9%, respectively; P = .484).

Study details: Long-term follow-up of 83 patients with AML treated with a LDC regimen (and 100 children with AML treated with a standard-dose chemotherapy (SDC) regimen.

Disclosures: This work was supported by NSCF, the Key Research Program of the Chinese Academy of Sciences, the Jiangsu Province Foundation, the National Clinical Research Center for Hematological Disorders, and the Youth Innovation Promotion Association of Chinese Academy of Sciences. The authors declared no conflicts of interest.

Source: Hu Y et al. Blood Adv. 2021 Apr 13. doi: 10.1182/bloodadvances.2020003453.

Key clinical point: Long-term survival in children with acute myeloid leukemia (AML) who receive low-dose chemotherapy (LDC) for remission induction is comparable to that of children who receive standard-dose chemotherapy (SDC).

Major finding:  No significant differences were observed between the LDC and SDC groups in 5-year event-free survival (61.4% ± 8.7% vs 65.2% ± 7.4%, respectively; P = .462), overall survival (72.7% ± 6.9% vs 72.5% ± 6.2%, respectively; P = .933), and the incidence of relapse (20.5% ± 4.5% vs 17.6% ± 3.9%, respectively; P = .484).

Study details: Long-term follow-up of 83 patients with AML treated with a LDC regimen (and 100 children with AML treated with a standard-dose chemotherapy (SDC) regimen.

Disclosures: This work was supported by NSCF, the Key Research Program of the Chinese Academy of Sciences, the Jiangsu Province Foundation, the National Clinical Research Center for Hematological Disorders, and the Youth Innovation Promotion Association of Chinese Academy of Sciences. The authors declared no conflicts of interest.

Source: Hu Y et al. Blood Adv. 2021 Apr 13. doi: 10.1182/bloodadvances.2020003453.

Key clinical point: Allogeneic hematopoietic stem cell transplantation (allo-HSCT) may improve survival outcomes in acute myeloid leukemia (AML) patients with ASXL1 mutations.

Major finding: In multivariate analysis, ASXL1 mutation was found to be an independent prognostic factor for overall survival (OS; hazard ratio [HR], 2.248; P = .017). Furthermore, Allo-HSCT was associated with significant improvements in overall survival (HR 7.568; P less than .001) and disease-free survival (HR, 2.611; P less than .001) in ASXL1-mutated ALL patients.

Study details: Analysis of the prognostic value of ASXL1 mutations and the role of allo-HSCT in 581 patients with AML.

Disclosures: The study was supported by grants from the National Natural Science Foundation of China, the Natural Science Foundation of Jiangsu Province, the Social Development Project of Jiangsu Province, the Priority Academic Program Development of Jiangsu Higher Education Institutions, and the National Key Research and Development Program. The authors declared no conflicts of interest.

Source: Zhou L et al. Hematology. 2021 Apr 10. doi: 10.1080/16078454.2021.1905356.

Key clinical point: Allogeneic hematopoietic stem cell transplantation (allo-HSCT) may improve survival outcomes in acute myeloid leukemia (AML) patients with ASXL1 mutations.

Major finding: In multivariate analysis, ASXL1 mutation was found to be an independent prognostic factor for overall survival (OS; hazard ratio [HR], 2.248; P = .017). Furthermore, Allo-HSCT was associated with significant improvements in overall survival (HR 7.568; P less than .001) and disease-free survival (HR, 2.611; P less than .001) in ASXL1-mutated ALL patients.

Study details: Analysis of the prognostic value of ASXL1 mutations and the role of allo-HSCT in 581 patients with AML.

Disclosures: The study was supported by grants from the National Natural Science Foundation of China, the Natural Science Foundation of Jiangsu Province, the Social Development Project of Jiangsu Province, the Priority Academic Program Development of Jiangsu Higher Education Institutions, and the National Key Research and Development Program. The authors declared no conflicts of interest.

Source: Zhou L et al. Hematology. 2021 Apr 10. doi: 10.1080/16078454.2021.1905356.

Key clinical point: Allogeneic hematopoietic stem cell transplantation (allo-HSCT) may improve survival outcomes in acute myeloid leukemia (AML) patients with ASXL1 mutations.

Major finding: In multivariate analysis, ASXL1 mutation was found to be an independent prognostic factor for overall survival (OS; hazard ratio [HR], 2.248; P = .017). Furthermore, Allo-HSCT was associated with significant improvements in overall survival (HR 7.568; P less than .001) and disease-free survival (HR, 2.611; P less than .001) in ASXL1-mutated ALL patients.

Study details: Analysis of the prognostic value of ASXL1 mutations and the role of allo-HSCT in 581 patients with AML.

Disclosures: The study was supported by grants from the National Natural Science Foundation of China, the Natural Science Foundation of Jiangsu Province, the Social Development Project of Jiangsu Province, the Priority Academic Program Development of Jiangsu Higher Education Institutions, and the National Key Research and Development Program. The authors declared no conflicts of interest.

Source: Zhou L et al. Hematology. 2021 Apr 10. doi: 10.1080/16078454.2021.1905356.

Key clinical point: No significant difference was seen in the 1-year relapse-free survival (RFS) relapse-free survival rate (RFS) and overall survival (OS) between intermediate-dose cytarabine (IDAC) and high-dose cytarabine (HiDAC) in patients with acute myeloid leukemia (AML).

Major finding: One-year RFS was 63.33% in the IDAC group vs. 46.87% in the HiDAC group (P = .137). One-year OS was 93.33% in the IDAC group vs. 84.37% in the HiDAC group (P = .691). IDAC group vs HiDAC group had significantly shorter duration of grade 3–4 thrombocytopenia (mean duration, 14.69 vs. 23.84 days; P = .045).

Study details: The data come from a retrospective study involvoing 62 patients with AML (30 patients in IDAC and 32 patients in HiDAC regimen).

Disclosures: The authors declared no conflicts of interest.

Source: Tangchitpianvit K et al. Hematology. 2021 Apr 14. doi: 10.1080/16078454.2021.1912949.

Key clinical point: No significant difference was seen in the 1-year relapse-free survival (RFS) relapse-free survival rate (RFS) and overall survival (OS) between intermediate-dose cytarabine (IDAC) and high-dose cytarabine (HiDAC) in patients with acute myeloid leukemia (AML).

Major finding: One-year RFS was 63.33% in the IDAC group vs. 46.87% in the HiDAC group (P = .137). One-year OS was 93.33% in the IDAC group vs. 84.37% in the HiDAC group (P = .691). IDAC group vs HiDAC group had significantly shorter duration of grade 3–4 thrombocytopenia (mean duration, 14.69 vs. 23.84 days; P = .045).

Study details: The data come from a retrospective study involvoing 62 patients with AML (30 patients in IDAC and 32 patients in HiDAC regimen).

Disclosures: The authors declared no conflicts of interest.

Source: Tangchitpianvit K et al. Hematology. 2021 Apr 14. doi: 10.1080/16078454.2021.1912949.

Key clinical point: No significant difference was seen in the 1-year relapse-free survival (RFS) relapse-free survival rate (RFS) and overall survival (OS) between intermediate-dose cytarabine (IDAC) and high-dose cytarabine (HiDAC) in patients with acute myeloid leukemia (AML).

Major finding: One-year RFS was 63.33% in the IDAC group vs. 46.87% in the HiDAC group (P = .137). One-year OS was 93.33% in the IDAC group vs. 84.37% in the HiDAC group (P = .691). IDAC group vs HiDAC group had significantly shorter duration of grade 3–4 thrombocytopenia (mean duration, 14.69 vs. 23.84 days; P = .045).

Study details: The data come from a retrospective study involvoing 62 patients with AML (30 patients in IDAC and 32 patients in HiDAC regimen).

Disclosures: The authors declared no conflicts of interest.

Source: Tangchitpianvit K et al. Hematology. 2021 Apr 14. doi: 10.1080/16078454.2021.1912949.

Key clinical point: Presence of acute kidney injury (AKI) may have an adverse impact on the clinical course of patients with acute myeloid leukemia (AML) treated with induction chemotherapy.

Major finding: 72 (18%) AML patients had AKI during induction chemotherapy. AML patients with AKI vs without AKI had more days with fever (7 vs. 5, P = .028) and require treatment intensive care unit more often (45.8% vs. 10.6%, P less than .001). AML patients with AKI also had a significantly lower complete remission rate following induction chemotherapy and shorter median overall survival.

Study details: Retrospective analysis of data from 401 patients with AML undergoing induction chemotherapy between 2007 and 2019.

Disclosures: Open access funding was enabled and organized by Projekt DEAL. The authors declared no conflicts of interest.

Source: Ballo O et al. Ann Hematol. 2021 March 11. doi: 10.1007/s00277-021-04482-3.

Key clinical point: Presence of acute kidney injury (AKI) may have an adverse impact on the clinical course of patients with acute myeloid leukemia (AML) treated with induction chemotherapy.

Major finding: 72 (18%) AML patients had AKI during induction chemotherapy. AML patients with AKI vs without AKI had more days with fever (7 vs. 5, P = .028) and require treatment intensive care unit more often (45.8% vs. 10.6%, P less than .001). AML patients with AKI also had a significantly lower complete remission rate following induction chemotherapy and shorter median overall survival.

Study details: Retrospective analysis of data from 401 patients with AML undergoing induction chemotherapy between 2007 and 2019.

Disclosures: Open access funding was enabled and organized by Projekt DEAL. The authors declared no conflicts of interest.

Source: Ballo O et al. Ann Hematol. 2021 March 11. doi: 10.1007/s00277-021-04482-3.

Key clinical point: Presence of acute kidney injury (AKI) may have an adverse impact on the clinical course of patients with acute myeloid leukemia (AML) treated with induction chemotherapy.

Major finding: 72 (18%) AML patients had AKI during induction chemotherapy. AML patients with AKI vs without AKI had more days with fever (7 vs. 5, P = .028) and require treatment intensive care unit more often (45.8% vs. 10.6%, P less than .001). AML patients with AKI also had a significantly lower complete remission rate following induction chemotherapy and shorter median overall survival.

Study details: Retrospective analysis of data from 401 patients with AML undergoing induction chemotherapy between 2007 and 2019.

Disclosures: Open access funding was enabled and organized by Projekt DEAL. The authors declared no conflicts of interest.

Source: Ballo O et al. Ann Hematol. 2021 March 11. doi: 10.1007/s00277-021-04482-3.

Key clinical point: Maintenance treatment with tyrosine kinase inhibitors (TKIs) following Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is safe and effective in patients with FLT3 mutated acute myeloid leukemia (AML).

Major finding: After a median follow-up of 10 months after allo-HSCT, 29 patients (71%) were alive and in complete remission (CR). In the subgroup of pretransplant TKI-treated patients, 25 patients (78%) were alive and in CR. 17 patients (41%) experienced side effects and 7 patients (17%) discontinued TKIs as a result of adverse events.

Study details: The data come from a retrospective observational study involving 41 patients treated with posttransplant TKIs (sorafenib, n = 23;  midostaurin, n = 18). 32  patients (79%) had also received TKIs before allo-HSCT.

Disclosures: The authors declared no conflicts of interest.

Source: Shimony S et al. Leuk Lymphoma. 2021 Apr 21. doi: 10.1080/10428194.2021.1913145.

Key clinical point: Maintenance treatment with tyrosine kinase inhibitors (TKIs) following Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is safe and effective in patients with FLT3 mutated acute myeloid leukemia (AML).

Major finding: After a median follow-up of 10 months after allo-HSCT, 29 patients (71%) were alive and in complete remission (CR). In the subgroup of pretransplant TKI-treated patients, 25 patients (78%) were alive and in CR. 17 patients (41%) experienced side effects and 7 patients (17%) discontinued TKIs as a result of adverse events.

Study details: The data come from a retrospective observational study involving 41 patients treated with posttransplant TKIs (sorafenib, n = 23;  midostaurin, n = 18). 32  patients (79%) had also received TKIs before allo-HSCT.

Disclosures: The authors declared no conflicts of interest.

Source: Shimony S et al. Leuk Lymphoma. 2021 Apr 21. doi: 10.1080/10428194.2021.1913145.

Key clinical point: Maintenance treatment with tyrosine kinase inhibitors (TKIs) following Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is safe and effective in patients with FLT3 mutated acute myeloid leukemia (AML).

Major finding: After a median follow-up of 10 months after allo-HSCT, 29 patients (71%) were alive and in complete remission (CR). In the subgroup of pretransplant TKI-treated patients, 25 patients (78%) were alive and in CR. 17 patients (41%) experienced side effects and 7 patients (17%) discontinued TKIs as a result of adverse events.

Study details: The data come from a retrospective observational study involving 41 patients treated with posttransplant TKIs (sorafenib, n = 23;  midostaurin, n = 18). 32  patients (79%) had also received TKIs before allo-HSCT.

Disclosures: The authors declared no conflicts of interest.

Source: Shimony S et al. Leuk Lymphoma. 2021 Apr 21. doi: 10.1080/10428194.2021.1913145.

Key clinical point: The addition of gemtuzumab ozogamycin (GO) to standard chemotherapy (SC) did not improve survival in younger patients with de novo acute myeloid leukemia (AML) and intermediate-risk (IR) cytogenetics but was associated with significantly higher toxicities.

Major finding: During a median follow-up of 35 months, event-free survival (hazard ratio [HR], 1.35; P = .116) and overall survival (HR, 1.35; P = .146) were not different in patients receiving GO+SC vs. SC alone. Duration of neutropenia (P = .03) and thrombopenia (P less than .001) and grade 3/4 liver toxicities (P = .03) were significantly higher in patients receiving GO vs. SC alone.

Study details: Phase 3 AML 2006-IR trial included 238 younger patients (age, 18-60 years) with de novo AML and IR cytogenetics randomly assigned to receive cytarabine and daunorubicin with or without GO. GO arm was prematurely closed after 254 inclusions because of toxicity and early deaths.

Disclosures: This study was supported by the French government PHRC 2006 and PFIZER group. Some investigators reported being on advisory boards, receiving research and travel funding, honoraria, and personal fees from various pharmaceutical companies, including Pfizer.

Source: Bouvier A et al. Eur J Haematol. 2021 Mar 25. doi: 10.1111/ejh.13626.

Key clinical point: The addition of gemtuzumab ozogamycin (GO) to standard chemotherapy (SC) did not improve survival in younger patients with de novo acute myeloid leukemia (AML) and intermediate-risk (IR) cytogenetics but was associated with significantly higher toxicities.

Major finding: During a median follow-up of 35 months, event-free survival (hazard ratio [HR], 1.35; P = .116) and overall survival (HR, 1.35; P = .146) were not different in patients receiving GO+SC vs. SC alone. Duration of neutropenia (P = .03) and thrombopenia (P less than .001) and grade 3/4 liver toxicities (P = .03) were significantly higher in patients receiving GO vs. SC alone.

Study details: Phase 3 AML 2006-IR trial included 238 younger patients (age, 18-60 years) with de novo AML and IR cytogenetics randomly assigned to receive cytarabine and daunorubicin with or without GO. GO arm was prematurely closed after 254 inclusions because of toxicity and early deaths.

Disclosures: This study was supported by the French government PHRC 2006 and PFIZER group. Some investigators reported being on advisory boards, receiving research and travel funding, honoraria, and personal fees from various pharmaceutical companies, including Pfizer.

Source: Bouvier A et al. Eur J Haematol. 2021 Mar 25. doi: 10.1111/ejh.13626.

Key clinical point: The addition of gemtuzumab ozogamycin (GO) to standard chemotherapy (SC) did not improve survival in younger patients with de novo acute myeloid leukemia (AML) and intermediate-risk (IR) cytogenetics but was associated with significantly higher toxicities.

Major finding: During a median follow-up of 35 months, event-free survival (hazard ratio [HR], 1.35; P = .116) and overall survival (HR, 1.35; P = .146) were not different in patients receiving GO+SC vs. SC alone. Duration of neutropenia (P = .03) and thrombopenia (P less than .001) and grade 3/4 liver toxicities (P = .03) were significantly higher in patients receiving GO vs. SC alone.

Study details: Phase 3 AML 2006-IR trial included 238 younger patients (age, 18-60 years) with de novo AML and IR cytogenetics randomly assigned to receive cytarabine and daunorubicin with or without GO. GO arm was prematurely closed after 254 inclusions because of toxicity and early deaths.

Disclosures: This study was supported by the French government PHRC 2006 and PFIZER group. Some investigators reported being on advisory boards, receiving research and travel funding, honoraria, and personal fees from various pharmaceutical companies, including Pfizer.

Source: Bouvier A et al. Eur J Haematol. 2021 Mar 25. doi: 10.1111/ejh.13626.

Key clinical point: In patients with acute myeloid leukemia (AML), incomplete count recovery (CRi) or presence of measurable residual disease (MRD) before allogeneic hematopoietic cell transplantation (allo-HCT) was associated with inferior posttransplant outcomes vs. those in complete remission (CR) or without MRD.

Major finding: Patients in CRi vs. CR were at a higher risk for death (hazard ratio [HR], 1.27; P less than .001) and nonrelapse mortality (HR, 1.33; P = .002). Additionally, presence vs. absence of MRD was associated with shorter overall survival (HR, 1.52; P less than .001) and increased relapse (HR, 1.78; P less than .001).

Study details: This observational study included 2,492 adult patients with AML (CR, n=1,799; CRi, n=693) from the Center for International Blood and Marrow Transplantation Research (CIBMTR) registry, who underwent first allo-HCT between 2007 and 2015.

Disclosures: CIBMTR is primarily supported by the National Cancer Institute, National Heart, Lung, and Blood Institute, National Institute of Allergy and Infectious Diseases, Health Resources and Services Administration, and Office of Naval Research. The lead author had no disclosures. Some coinvestigators reported ties with various pharmaceutical companies.

Source: Percival ME et al. Bone Marrow Transplant. 2021 Apr 16. doi: 10.1038/s41409-021-01261-6.

Key clinical point: In patients with acute myeloid leukemia (AML), incomplete count recovery (CRi) or presence of measurable residual disease (MRD) before allogeneic hematopoietic cell transplantation (allo-HCT) was associated with inferior posttransplant outcomes vs. those in complete remission (CR) or without MRD.

Major finding: Patients in CRi vs. CR were at a higher risk for death (hazard ratio [HR], 1.27; P less than .001) and nonrelapse mortality (HR, 1.33; P = .002). Additionally, presence vs. absence of MRD was associated with shorter overall survival (HR, 1.52; P less than .001) and increased relapse (HR, 1.78; P less than .001).

Study details: This observational study included 2,492 adult patients with AML (CR, n=1,799; CRi, n=693) from the Center for International Blood and Marrow Transplantation Research (CIBMTR) registry, who underwent first allo-HCT between 2007 and 2015.

Disclosures: CIBMTR is primarily supported by the National Cancer Institute, National Heart, Lung, and Blood Institute, National Institute of Allergy and Infectious Diseases, Health Resources and Services Administration, and Office of Naval Research. The lead author had no disclosures. Some coinvestigators reported ties with various pharmaceutical companies.

Source: Percival ME et al. Bone Marrow Transplant. 2021 Apr 16. doi: 10.1038/s41409-021-01261-6.

Key clinical point: In patients with acute myeloid leukemia (AML), incomplete count recovery (CRi) or presence of measurable residual disease (MRD) before allogeneic hematopoietic cell transplantation (allo-HCT) was associated with inferior posttransplant outcomes vs. those in complete remission (CR) or without MRD.

Major finding: Patients in CRi vs. CR were at a higher risk for death (hazard ratio [HR], 1.27; P less than .001) and nonrelapse mortality (HR, 1.33; P = .002). Additionally, presence vs. absence of MRD was associated with shorter overall survival (HR, 1.52; P less than .001) and increased relapse (HR, 1.78; P less than .001).

Study details: This observational study included 2,492 adult patients with AML (CR, n=1,799; CRi, n=693) from the Center for International Blood and Marrow Transplantation Research (CIBMTR) registry, who underwent first allo-HCT between 2007 and 2015.

Disclosures: CIBMTR is primarily supported by the National Cancer Institute, National Heart, Lung, and Blood Institute, National Institute of Allergy and Infectious Diseases, Health Resources and Services Administration, and Office of Naval Research. The lead author had no disclosures. Some coinvestigators reported ties with various pharmaceutical companies.

Source: Percival ME et al. Bone Marrow Transplant. 2021 Apr 16. doi: 10.1038/s41409-021-01261-6.

Key clinical point: Prior treatment with gemtuzumab ozogamicin (GO) before allogeneic hematopoietic cell transplantation (allo-HCT) in pediatric patients with acute myeloid leukemia (AML) increased risk for veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) but did not affect survival.

Major finding: Compared with nonexposure, exposure to GO was associated with increased risk for VOD/SOS at 100 days (odds ratio, 2.26; P = .004). However, posttransplant overall survival (P = .43), disease-free survival (P = .20), relapse (P = .32), and nonrelapse mortality (P = .51) did not differ in patients with or without prior GO treatment.

Study details: Findings are from retrospective assessment of pediatric patients with AML who received myeloablative allo-HCT between 2008 and 2011 with (n=148) or without (n=348) prior GO treatment.

Disclosures: This study was funded by Pfizer. D Chirnomas, CJ Hoang, and FR Loberiza Jr declared being current/former employees of and/or had equity ownership in Pfizer. Other authors declared no conflicts of interest.

Source: Duncan C et al. Pediatr Blood Cancer. 2021 Apr 19. doi: 10.1002/pbc.29067.

Key clinical point: Prior treatment with gemtuzumab ozogamicin (GO) before allogeneic hematopoietic cell transplantation (allo-HCT) in pediatric patients with acute myeloid leukemia (AML) increased risk for veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) but did not affect survival.

Major finding: Compared with nonexposure, exposure to GO was associated with increased risk for VOD/SOS at 100 days (odds ratio, 2.26; P = .004). However, posttransplant overall survival (P = .43), disease-free survival (P = .20), relapse (P = .32), and nonrelapse mortality (P = .51) did not differ in patients with or without prior GO treatment.

Study details: Findings are from retrospective assessment of pediatric patients with AML who received myeloablative allo-HCT between 2008 and 2011 with (n=148) or without (n=348) prior GO treatment.

Disclosures: This study was funded by Pfizer. D Chirnomas, CJ Hoang, and FR Loberiza Jr declared being current/former employees of and/or had equity ownership in Pfizer. Other authors declared no conflicts of interest.

Source: Duncan C et al. Pediatr Blood Cancer. 2021 Apr 19. doi: 10.1002/pbc.29067.

Key clinical point: Prior treatment with gemtuzumab ozogamicin (GO) before allogeneic hematopoietic cell transplantation (allo-HCT) in pediatric patients with acute myeloid leukemia (AML) increased risk for veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) but did not affect survival.

Major finding: Compared with nonexposure, exposure to GO was associated with increased risk for VOD/SOS at 100 days (odds ratio, 2.26; P = .004). However, posttransplant overall survival (P = .43), disease-free survival (P = .20), relapse (P = .32), and nonrelapse mortality (P = .51) did not differ in patients with or without prior GO treatment.

Study details: Findings are from retrospective assessment of pediatric patients with AML who received myeloablative allo-HCT between 2008 and 2011 with (n=148) or without (n=348) prior GO treatment.

Disclosures: This study was funded by Pfizer. D Chirnomas, CJ Hoang, and FR Loberiza Jr declared being current/former employees of and/or had equity ownership in Pfizer. Other authors declared no conflicts of interest.

Source: Duncan C et al. Pediatr Blood Cancer. 2021 Apr 19. doi: 10.1002/pbc.29067.

Key clinical point: Survival outcomes have improved significantly in patients with de novo acute myeloid leukemia (AML) over a 4-decade period from 1980 to 2017; however, least improvement was observed in patients aged 70 years or older.

Major finding: Overall, 5-year survival increased from 9% during 1980-1989 to 15% in 1990-1999, 22% in 2000-2009, and 28% in 2010-2017 (all P less than .001). However, improvement in 5-year survival was poorest in patients aged 70 years or older with 1% in 1980-1989 to 5% in 2010-2017.

Study details: Findings are from a U.S. population-based study that evaluated 29,107 patients from the Surveillance, Epidemiology, and End Results registries, who were diagnosed with de novo AML between 1980 and 2017.

Disclosures: No funding source was identified. Some investigators including the lead author reported personal fees, research funding, honoraria, or other support from various pharmaceutical companies.

Source: Sasaki K et al. Cancer. 2021 Apr 5. doi: 10.1002/cncr.33458.

Key clinical point: Survival outcomes have improved significantly in patients with de novo acute myeloid leukemia (AML) over a 4-decade period from 1980 to 2017; however, least improvement was observed in patients aged 70 years or older.

Major finding: Overall, 5-year survival increased from 9% during 1980-1989 to 15% in 1990-1999, 22% in 2000-2009, and 28% in 2010-2017 (all P less than .001). However, improvement in 5-year survival was poorest in patients aged 70 years or older with 1% in 1980-1989 to 5% in 2010-2017.

Study details: Findings are from a U.S. population-based study that evaluated 29,107 patients from the Surveillance, Epidemiology, and End Results registries, who were diagnosed with de novo AML between 1980 and 2017.

Disclosures: No funding source was identified. Some investigators including the lead author reported personal fees, research funding, honoraria, or other support from various pharmaceutical companies.

Source: Sasaki K et al. Cancer. 2021 Apr 5. doi: 10.1002/cncr.33458.

Key clinical point: Survival outcomes have improved significantly in patients with de novo acute myeloid leukemia (AML) over a 4-decade period from 1980 to 2017; however, least improvement was observed in patients aged 70 years or older.

Major finding: Overall, 5-year survival increased from 9% during 1980-1989 to 15% in 1990-1999, 22% in 2000-2009, and 28% in 2010-2017 (all P less than .001). However, improvement in 5-year survival was poorest in patients aged 70 years or older with 1% in 1980-1989 to 5% in 2010-2017.

Study details: Findings are from a U.S. population-based study that evaluated 29,107 patients from the Surveillance, Epidemiology, and End Results registries, who were diagnosed with de novo AML between 1980 and 2017.

Disclosures: No funding source was identified. Some investigators including the lead author reported personal fees, research funding, honoraria, or other support from various pharmaceutical companies.

Source: Sasaki K et al. Cancer. 2021 Apr 5. doi: 10.1002/cncr.33458.