Contributions
Abstract: PB2275
Type: Publication Only
Background
In patients with myelofibrosis (MF), ruxolitinib has proven effective in ameliorating symptoms such as night sweats, itching and abdominal discomfort. Responses are frequently seen within the first month of treatment. To date, however, data on symptom improvement has been reported as decreasing means of symptom scores over time in large patient cohorts. By nature, these data cannot address critical questions arising in clinical practice: if a patient fails to show symptom improvement within the first month or the first three months of treatment, what are the chances that he or she will still respond if treatment is continued?
Aims
Therapeutic decisions for individual patients may therefore be better informed by knowledge of conditional probabilities.
Methods
We thus used conditional probability algorithms to analyze symptom improvement in patients enrolled in the JaKoMo trial, a phase IV prospective, non-interventional study of MF patients receiving ruxolitinib therapy. Symptom burden was measured by the Total Symptom Score (TSS) of the MPN-SAF (Myeloproliferative Neoplasm - Symptom Assesment Form), a scale that measures 10 items and can range from 0 – 100.
Results
Data at baseline, one month, three and six months follow up were available for 192 MF patients. At baseline, JaKoMo patients reported a TSS of 29.9. Two separate response analyses were conducted.
In a first study, response was defined as a change of at least 5 points in the TSS. Following a month of ruxolitinib treatment, 105 (55%) of patients responded by decreasing the total symptom score by 5 points or more. Importantly, of the remaining 87 patients, 23 patients (26.4%) responded during the following 2 months, and these responses were maintained for at least the following 3 months. Of the 64 of patients that had not responded by 3 months, a further 8 (13%) responded at 6 months.
We subsequently used a more stringent criterium to define response, a decrease of at least 50% in the TSS of an individual patient. By this criterion, 62 patients (32%) responded following 1 month of treatment, while 130 patients (68%) did not. Of these 130 patients, 26 (20%) responded following two more months of treatment. Likewise, of the 104 remaining patients without a response until 3 months, 17 (16%) responded during the following 3 months. An important strength of our study is that the JaKoMo patient cohort is recruited largely from office-based hematologists, which renders the data more broadly applicable than studies conducted in selected, academic hospital settings.
Conclusion
Our data demonstrate that a subset of MF patients will experience relief from symptoms under continued ruxolitinib therapy, even if improvement was initially not observed during the first 4 to 12 weeks of treatment. Given the potential myelosuppressive effect of ruxolitinib, these data may be used to weigh risks and benefits in informing therapeutic decisions.
Session topic: 16. Myeloproliferative neoplasms - Clinical
Abstract: PB2275
Type: Publication Only
Background
In patients with myelofibrosis (MF), ruxolitinib has proven effective in ameliorating symptoms such as night sweats, itching and abdominal discomfort. Responses are frequently seen within the first month of treatment. To date, however, data on symptom improvement has been reported as decreasing means of symptom scores over time in large patient cohorts. By nature, these data cannot address critical questions arising in clinical practice: if a patient fails to show symptom improvement within the first month or the first three months of treatment, what are the chances that he or she will still respond if treatment is continued?
Aims
Therapeutic decisions for individual patients may therefore be better informed by knowledge of conditional probabilities.
Methods
We thus used conditional probability algorithms to analyze symptom improvement in patients enrolled in the JaKoMo trial, a phase IV prospective, non-interventional study of MF patients receiving ruxolitinib therapy. Symptom burden was measured by the Total Symptom Score (TSS) of the MPN-SAF (Myeloproliferative Neoplasm - Symptom Assesment Form), a scale that measures 10 items and can range from 0 – 100.
Results
Data at baseline, one month, three and six months follow up were available for 192 MF patients. At baseline, JaKoMo patients reported a TSS of 29.9. Two separate response analyses were conducted.
In a first study, response was defined as a change of at least 5 points in the TSS. Following a month of ruxolitinib treatment, 105 (55%) of patients responded by decreasing the total symptom score by 5 points or more. Importantly, of the remaining 87 patients, 23 patients (26.4%) responded during the following 2 months, and these responses were maintained for at least the following 3 months. Of the 64 of patients that had not responded by 3 months, a further 8 (13%) responded at 6 months.
We subsequently used a more stringent criterium to define response, a decrease of at least 50% in the TSS of an individual patient. By this criterion, 62 patients (32%) responded following 1 month of treatment, while 130 patients (68%) did not. Of these 130 patients, 26 (20%) responded following two more months of treatment. Likewise, of the 104 remaining patients without a response until 3 months, 17 (16%) responded during the following 3 months. An important strength of our study is that the JaKoMo patient cohort is recruited largely from office-based hematologists, which renders the data more broadly applicable than studies conducted in selected, academic hospital settings.
Conclusion
Our data demonstrate that a subset of MF patients will experience relief from symptoms under continued ruxolitinib therapy, even if improvement was initially not observed during the first 4 to 12 weeks of treatment. Given the potential myelosuppressive effect of ruxolitinib, these data may be used to weigh risks and benefits in informing therapeutic decisions.
Session topic: 16. Myeloproliferative neoplasms - Clinical