Contributions
Abstract: PB2393
Type: Publication Only
Background
Ph+ R/R BCP ALL is a rare disease with poor prognosis. Tyrosine kinase inhibitors (TKIs) are usually recommended for these patients but exact treatment patterns are unclear.
Aims
This study was aimed to understand the current treatment pattern and unmet need in adults with Ph+ R/R BCP ALL in EU-5 countries.
Methods
A Delphi-based methodology was employed: a survey sent to clinicians and a country-specific panel were used to discuss the survey results to generate point estimates. Physicians must be board certified with at least 5 years’ experience in R/R ALL. The percentage of using each treatment was estimated for patients who were relapsed/refractory to at least one 2nd-generation TKI or were intolerant to 2nd-generation TKIs and intolerant/refractory to imatinib. Limitations of the current treatments for Ph+ R/R ALL were examined.
Results
Four physicians from the UK, 4 from Germany, and 3 from each of the remaining 3 countries were enrolled with a median of 10 years’ experience in R/R ALL. Regimens are widely distributed in each country and across the countries (Table). TKI (mainly ponatinib) combined with chemotherapy is the most commonly used regimen. Most physicians agree or strongly agree with these limitations of the current treatments:
- The risk of developing resistance to TKIs is very high;
- Survival is short, particularly once the disease becomes resistant to TKIs;
- Chemotherapy alone does not significantly extend overall survival;
- Patients may experience different adverse events (AEs) with each TKI.
Regimen | Proportion of Patients (%) | ||||
France | Germany | Italy | Spain | UK | |
TKI only |
| 15 | 86 | 17 | 10 |
TKI + Clofarabine based chemo |
|
|
| 1 | 15 |
TKI + FLAG-IDA based chemo |
|
| 2 | 10 | 50 |
TKI + Hyper-CVAD | 20 |
| 7 | 10 |
|
TKI + Other chemo (e.g. vincristine + steroids) | 67 | 35 |
| 17 | 25 |
TKI + Blinatumomab | 7 | 7 |
|
|
|
Blinatumomab |
| 29 |
| 22 |
|
Inotuzumab | 6 | 14 |
| 5 |
|
Chemo only |
|
| 5 |
|
|
Best supportive care only |
|
|
| 18 |
|
TKI |
|
|
|
|
|
Ponatinib | 60 | 71 | 100 | 90 | 100 |
Dasatinib | 7 | 15 | 10 | ||
Nilotinib | 33 | 14.5 | |||
Conclusion
TKI plus chemotherapy is the most commonly used regimen for adults with Ph+ R/R BCP ALL in the EU-5 countries. There remains significant unmet need due to limited survival, resistance to TKIs and AEs associated with the current treatments.
Session topic: 36. Quality of life, palliative care, ethics and health economics
Keyword(s): Acute lymphoblastic leukemia
Abstract: PB2393
Type: Publication Only
Background
Ph+ R/R BCP ALL is a rare disease with poor prognosis. Tyrosine kinase inhibitors (TKIs) are usually recommended for these patients but exact treatment patterns are unclear.
Aims
This study was aimed to understand the current treatment pattern and unmet need in adults with Ph+ R/R BCP ALL in EU-5 countries.
Methods
A Delphi-based methodology was employed: a survey sent to clinicians and a country-specific panel were used to discuss the survey results to generate point estimates. Physicians must be board certified with at least 5 years’ experience in R/R ALL. The percentage of using each treatment was estimated for patients who were relapsed/refractory to at least one 2nd-generation TKI or were intolerant to 2nd-generation TKIs and intolerant/refractory to imatinib. Limitations of the current treatments for Ph+ R/R ALL were examined.
Results
Four physicians from the UK, 4 from Germany, and 3 from each of the remaining 3 countries were enrolled with a median of 10 years’ experience in R/R ALL. Regimens are widely distributed in each country and across the countries (Table). TKI (mainly ponatinib) combined with chemotherapy is the most commonly used regimen. Most physicians agree or strongly agree with these limitations of the current treatments:
- The risk of developing resistance to TKIs is very high;
- Survival is short, particularly once the disease becomes resistant to TKIs;
- Chemotherapy alone does not significantly extend overall survival;
- Patients may experience different adverse events (AEs) with each TKI.
Regimen | Proportion of Patients (%) | ||||
France | Germany | Italy | Spain | UK | |
TKI only |
| 15 | 86 | 17 | 10 |
TKI + Clofarabine based chemo |
|
|
| 1 | 15 |
TKI + FLAG-IDA based chemo |
|
| 2 | 10 | 50 |
TKI + Hyper-CVAD | 20 |
| 7 | 10 |
|
TKI + Other chemo (e.g. vincristine + steroids) | 67 | 35 |
| 17 | 25 |
TKI + Blinatumomab | 7 | 7 |
|
|
|
Blinatumomab |
| 29 |
| 22 |
|
Inotuzumab | 6 | 14 |
| 5 |
|
Chemo only |
|
| 5 |
|
|
Best supportive care only |
|
|
| 18 |
|
TKI |
|
|
|
|
|
Ponatinib | 60 | 71 | 100 | 90 | 100 |
Dasatinib | 7 | 15 | 10 | ||
Nilotinib | 33 | 14.5 | |||
Conclusion
TKI plus chemotherapy is the most commonly used regimen for adults with Ph+ R/R BCP ALL in the EU-5 countries. There remains significant unmet need due to limited survival, resistance to TKIs and AEs associated with the current treatments.
Session topic: 36. Quality of life, palliative care, ethics and health economics
Keyword(s): Acute lymphoblastic leukemia