Acute Myeloid Leukemia (AML)
Conditions
Keywords
IDH1 R132 mutation
Brief summary
The goal of this non-interventional study is to evaluate quality of life (QoL) in adult patients with newly diagnosed IDH1 R132-mutated AML who are not eligible to receive standard induction chemotherapy and who are treated with ivosidenib in combination with azacitidine in a real-world setting in Germany. The main questions it aims to answer are: * Evaluate QoL by validated and widely used Functional Assessment of Cancer Therapy - Leukemia (FACT-Leu) questionnaire and European Quality of Life 5 Dimensions 5 Level Version (EQ-5D-5L) questionnaire during treatment and follow-up period * Assesment of effectiveness in routine treatment (e.g. overall survival, event-free survival, overall response rate) * Assessment of drug safety (all adverse events) * Description of treatment reality in detail
Interventions
DRUGIvosidenib
inhibitor of mutant IDH1
Sponsors
iOMEDICO AG
Study design
Observational model
CASE_ONLY
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Age 18 years or older. * Newly diagnosed Acute Myeloid Leukemia (AML). * Having an isocitrate dehydrogenase 1 (IDH1) R132 mutation. * Not eligible to receive standard induction chemotherapy. * Decision for treatment with ivosidenib in combination with azacitidine as per current SmPC. * Signed written informed consent\* \*Patients are allowed to be enrolled up to 6 weeks after ivosidenib plus azacitidine dose * For patients participating in PROs: Willingness and capability to participate in PRO assessment in German language * Other criteria according to current SmPC.
Exclusion criteria
* Participation in an interventional clinical trial within 30 days prior to enrollment or simultaneous participation in an interventional clinical trial except for the follow-up period. * Patients unable to consent * Other contraindications according to current SmPC.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Evaluate quality of life | Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months | Validation of FACT-Leu. Change from baseline (treatment start) of FACT-Leu total score over time |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Subjective well-being: Validation of FACT-Leu | Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months | Change from baseline (treatment start) of FACT-Leu subscale score over time |
| Subjective well-being: Validation of FACT-Leu and EQ-5D-5L questionnaire | Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months | FACT-Leu total score over time over time |
| Subjective well-being: Validation of EQ-5D-5L questionnaire | Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months | Change from baseline of the EQ-5D-5L visual analogue scale score over time |
| Assesment of effectiveness in routine treatment | Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months | Assesment of overall survival (OS), |
| Assessment of drug safety | Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months | Assesment of adverse events (AEs), serious adverse events (SAEs), adverse drug reactions (ADRs), serious adverse drug reactions (SADRs), Adverse events of special interest (AESIs) |
| Assessment of parameters of treatment decision making | From date of patient enrollment until start of treatment | Frequency of distinct parameters affecting therapy choice including reasons why patient is ineligible for standard induction chemotherapy. |
| Ivosidenib and azacitidine treatment: Dose intensity | Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months | Descriptive statistics will be provided for dose intensity for ivosidinib and azacitidine |
| Ivosidenib and azacitidine treatment: Frequency and type of dose modification | Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months | Frequency tables will be provided for frequency and type of dose modification for ivosidinib and azacitidine |
| Ivosidenib and azacitidine treatment: Reason for dose modifications | Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months | Frequency tables of reasons for dose modifications will be provided for ivosidinib and azacitidine |
| Ivosidenib and azacitidine treatment: Duration of treatment in total and for each substance | Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months | Descriptive statistics will be provided for duration of treatment in total and for each substance dose |
| Ivosidenib and azacitidine treatment: Reason for end of treatment (EOT) | Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months | Frequency tables of EOT-reasons will be provided |
| Treatment reality in detail: Transfusion dependency | Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months | Proportion of transfusion dependent patients to independency and vice versa will be provided |
| Treatment reality in detail: Concomitant medication | Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months | Frequency table of concomitant medication in total and frequency of concomitant medications known to induce QT prolongation (e.g., antiarrhythmic medicines, fluoroquinolones, triazole anti-fungals, 5-HT3 receptor antagonists) as well as strong CYP3A4 inducers or dabigatran will be provided |
| Treatment reality in detail: Subsequent antineoplastic therapies | Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months | Description of frequency and type of subsequent antineoplastic therapies by line of therapy (number and substance) |
| Treatment reality in detail: Frequency of hospitalizations/emergency room visits | Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months | Frequency table of hospitalization and emergency room visits will be provided |
| Treatment reality in detail: Reasons for hospitalizations/emergency room visits | Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months | Frequency table of reasons for hospitalization and emergency room visits will be provided |
| Treatment reality in detail: Length of hospital stay | Baseline until end of study (during ivosidenib treatment and Follow-Up); Up to 54 months | Descriptive statistic for length of hospital stay will be provided |
Countries
Germany
Outcome results
None listed