Decitabine Low-Dose Schedule (100 mg/m2/Course) in Myelodysplastic Syndrome (MDS). Comparison of 3 Different Dose Schedules.

Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 2522-2522 ◽  
Author(s):  
Hagop Kantarjian ◽  
Susan O’Brien ◽  
Francis Giles ◽  
Farhad Ravandi-Kashani ◽  
Stefan Faderl ◽  
...  
Keyword(s):  
Low Dose ◽  
Overall Response Rate ◽  
Response Rate ◽  
Optimal Schedule ◽  
Dose Schedule ◽  
Overall Response ◽  
Transfusion Independence ◽  
Secondary Mds ◽  
To Receive

Abstract Background Decitabine (DAC), a hypomethylating agent, has shown activity in MDS. DAC 150 mg/m2 by continuous infusion has been associated with CR rates of 10% to 20%. We investigated optimizing the dose schedule of DAC in MDS. Study Group and Methods Patients (pts) with IPSS intermediate 1–2 & high risk MDS were randomized to one of 3 schedules of DAC: 1) 20 mg/m2 IV over 1 hour daily x 5; 2) 10 mg/m2 IV over 1 hour daily x 10; or 3)10 mg/m2 subcutaneously (SQ) BID x 5. A total of 95 pts are to be treated; Bayesian randomization is implemented based on CR rates. Courses were given every 28 days. Delays to allow counts recovery were permitted every 3 courses, or if myelosuppression without disease, or severe myelosuppression complications. Pts were allowed to receive erythropoietin 40,000 units weekly for anemia, or GCSF if needed. Response criteria for CR & PR were as for AML (PR requiring also ↓ blasts by >50%). Clinical benefit (CB) referred to one or more of: platelets î by ≥ 50% and >30 x 109/L, or granulocytes increase by ≥ 100% and to >109/L, or hemoglobin î by ≥ 2 g/dl or transfusion independence, or splenomegaly ↓ by 50% or more, or monocytes ↓ by 50% or more (pretreatment >5 x 109/L). Results 92 pts have been treated; median age 65 (31–90) yrs; 66% >60 yrs old. IPSS: intermediate-1 25%; intermediate-2 38%; high 19%; CMML 17% Cytogenetic abnormalities 57%; secondary MDS 17%; marrow blasts > 10% in 31%. 27 pts had prior erythropoietin; 17 had prior GCSF; 22 had other prior therapies. Presently, 89 pts have received 1 course. Results: 32 CR (36%); 7 PR (8%); 13 marrow CR + CB (15%); 16 CB (18%); overall response 68/89=76%. Median courses to CR 3 (range 1 to 6). Median follow-up of 9 months; 48 pts continue on DAC. Compared with a 114 pts with MDS who received intensive chemotherapy (2000–2004), CR rate was lower with DAC (36% vs. 45%), overall response rate was favorable; 6-week mortality was lower with DAC (1% vs. 21%); and estimated survival favorable (p = 0.00007). CR rates by schedule: 5 days IV 24/58 (41%); 5 days SQ 4/14 (28%); 10 day IV 4/17 (24%). There was more myelosuppression with 10 day IV. After 55 patients were randomized, the 5 day IV arm was determined statistically superior, therefore, remaining patients were not randomized, but were treated with 5 days IV therapy. Conclusions DAC has significant anti-MDS activity; 2) optimal schedule: 20 mg/m2 IV over 1 hour daily x 5; 3) timely repeated courses needed for optimal response.

Decitabine Low-Dose Schedule (100 mg/m2/course) in Myelodysplastic Syndrome (MDS). Comparison of 3 Different Dose Schedules.

Blood ◽  
2004 ◽  
Vol 104 (11) ◽  
pp. 1437-1437 ◽  
Author(s):  
Hagop M. Kantarjian ◽  
Farhad Ravandi ◽  
Susan O’Brien ◽  
Francis Giles ◽  
Stefan Faderl ◽  
...  
Keyword(s):  
Myeloid Leukemia ◽  
Low Dose ◽  
Optimal Dose ◽  
Dose Schedule ◽  
Overall Response ◽  
Transfusion Independence ◽  
Historical Group ◽  
Secondary Mds ◽  
To Receive

Abstract Decitabine, a hypomethylating agent, has shown activity in MDS, acute myeloid leukemia (AML), and chronic myeloid leukemia. In this study, we investigated optimizing the dose schedule. Patients with IPSS intermediate 1–2 and high risk were randomized to one of 3 schedules of decitabine: 1) 20 mg/m2 IV over 1 hour daily x 5; 2) 10 mg/m2 IV over 1 hour daily x 10; or 3) 10 mg/m2 subcutaneously (SQ) BID x 5. A total of 90 patients are to be treated. Randomization is equal until the 45th patient after which a Bayesian (play by the winner) randomization is implemented based on CR rates. Courses were given every 28 days regardless of counts, as long as counts recovered to pretreatment + evidence of disease on repeat marrow + no significant myelosuppression complications. Delays to allow for recovery of counts were permitted every 3 courses, or in the presence of myelosuppression without disease or severe myelosuppression-related complications. Patients were allowed to receive erythropoietin 40,000 units weekly for anemia, or GCSF if needed during febrile neutropenia. Response criteria for CR and PR were as for AML (PR requiring also decrease blasts by >50%). Clinical benefit (CB) referred to one or more of the following: platelets increase by 50% and above 30 x 109/L, or granulocytes increase by 100% and to above 109/L, or hemoglobin increase by 2 g/dl or transfusion independence, or splenomegaly decrease by 50% or more, or monocytes decrease by 50% or more (pretreatment >5 x 109/L). 43 patients have been treated; median age 63 years (range 39 to 90); 60 (26%) were ≥ 60 years old. IPSS risk: intermediate 1 –13 (30%); intermediate 2–13 (30%); high-(23%); CMML-7(16%). Cytogenetic abnormalities were present in 56%; secondary MDS in 23%; marrow blasts ≥ 10% in 30%. 22 patients had prior erythropoietin; 9 had prior GCSF; 12 had other prior therapies (thalidomide 6, azacytidine 2, other 4). Presently, 36 patients have received at least 1 course of therapy. Results were: 10 CR (28%); 3 PR (9%); 18 CB (50%); overall response 31/36=86%. 22(51%) patients required hospitalizations for fever and neutropenia. Median courses to CR was 1 (range 1 to 3); 5 patients (50%) needed 2 or more courses to achieve CR. With a median follow-up of 4 months, 5 have evolved into AML; 4 have died (2 AML; 2 MDS); 35 patients continue on decitabine therapy. Compared with a historical group of 54 patients with MDS who received intensive chemotherapy (2000–2003) and matched for age, cytogenetics and IPSS/FAB, the CR rate was lower with decitabine (28% vs 47%), but the overall response rate was favorable; the 8-week mortality was also lower with decitabine (7% vs 26%); and estimated survival favorable (6-month rates 80% vs 67%). CR rates by schedule were: 5 days IV 6/15 (40%); 5 days SQ 2/11 (18%); 10 day IV 2/10 (20%). There was more myelosuppression with the 10 day IV schedule. We conclude that 1) decitabine at this low-dose schedule has major anti- MDS activity in the setting of poorer risk MDS; 2) the optimal dose schedule is being defined; 3) side effects are acceptable; 4) timely and repeated courses of decitabine therapy is required for optimal response results.

Prospective observational trial of low-dose skin electron therapy in mycosis fungoides using rotational technique.

2020 ◽  
Vol 38 (15_suppl) ◽  
pp. e22077-e22077
Author(s):  
Neil Newman ◽  
Austin Noah Kirschner ◽  
Chirayu Patel
Keyword(s):  
Treatment Response ◽  
Mycosis Fungoides ◽  
Clinical Benefit ◽  
Low Dose ◽  
Overall Response Rate ◽  
Response Rate ◽  
Time To Treatment ◽  
Overall Response ◽  
Electron Therapy

e22077 Background: Low dose total skin electron therapy (TSET) utilizing 12 Gy in 12 fractions has been shown to provide a durable treatment response for dermal lesions caused by mycosis fungoides secondary to cutaneous T cell lymphoma (CTCL). We sought to prospectively assess the durability of response and quality of life (QOL) for patients receiving low dose TSET using the rotational technique technique. Methods: We prospectively enrolled patients with CTCL on an IRB approved study from 2016 to 2019 who had pathologically confirmed CTCL from stage IB-III. Patients completed a baseline validated Skindex-29 survey encompassing 29 questions assessing QOL. Physicians graded the appearance of the skin using the modified severity-weighted assessment tool (mSWAT) at baseline. Patients received 12 Gy in 12 Fractions on consecutive dates and patients were treated with a dual field rotational technique. The mSWAT was reassessed on ensuing follow-up visits along with the Skindex. The primary outcome was overall response rate with the secondary outcomes being time to treatment response, duration of clinical benefit (time to requiring an additional intervention), as well as improvements in Skindex questionnaire items. Results: We enrolled 20 patients and recorded an overall response rate (ORR) of 90% with four complete responses. The time to treatment response was 9.7 weeks. While the baseline mSWAT was 55.6 it declined to a median mSWAT of 2.2 at last follow-up (p < 0.001) with there being a median decline of nearly 97% in the scores. The median duration of clinical benefit was 15.7 months. There was a precipitous decline in the Skindex total score and every subdomain when comparing each follow-up visit (p = 0.004) with post-hoc analysis demonstrating the decline between the baseline visit and first follow-up as the primary driver of the decline (p < 0.0001). Conclusions: This prospective study demonstrates the subjective and objective clinical benefits of utilizing low dose TSE with a dual beam rotational technique. This control rates and QOL will continue to be observed for ongoing feasibility as a treatment modality.

Survival Benefits of Patients with Non-Hodgkin’s Lymphoma Treated with Rituximab Combined with Chemotherapy.

Blood ◽  
2004 ◽  
Vol 104 (11) ◽  
pp. 4625-4625
Author(s):  
Zhixiang Shen ◽  
Junmin Li ◽  
Aihua Wang ◽  
Yu Chen
Keyword(s):  
B Cell ◽  
Overall Response Rate ◽  
Response Rate ◽  
Cell Lymphoma ◽  
Progression Free Survival ◽  
B Cell Lymphoma ◽  
Free Survival ◽  
Overall Response ◽  
Large B Cell

Abstract Purpose: Rituximab combined with chemotherapy has been recommended as first-line or second-line standard regimen in most subtypes of B-cell lymphoma in China by the 2004 National Comprehensive Cancer Network lymphoma therapy guideline. We have conducted a multicenter trial to evaluate the efficacy and safety of rituximab in combination with standard chemotherapy (CHOP) in patients with previously untreated or relapsed indolent and aggressive NHL. Methods: Patients received 4–8 cycles of rituximab plus CHOP every 21 days. For each cycle, rituximab (375mg/m2) was given on day 1 and CHOP started on day 3. CHOP consisted of cyclophosphamide 750mg/m2, doxorubicin 50mg/m2, and vincristine 1.4mg/m2 (maximum 2mg/dose) given intravenously on day 3, and oral prednisone 100mg on days 3–7. Results: A total of 221 patients were enrolled on the trial, 128 males and 93 females with a mean age of 49 years (range 10–83 years). The main lymphoma subtypes were small lymphocytic (15 patients, 7%), follicular (27 patients, 12%), and diffuse large B-cell (160 patients, 72%). In total, 56 patients had indolent NHL and 165 aggressive NHL. The overall response rate for all patients was 86% with 57% complete responses. In patients with indolent NHL the overall and complete response rates were 95% and 55% respectively. After a median 12 months follow up, progression-free survival in patients with indolent NHL was 88%±5% at 1 year and 83%±6% at 2 years. In the 160 patients with diffuse large B-cell lymphoma, the overall response rate was 88% with 61% complete responses, and after a mean follow-up of 6 months, predicted 1-year and 2-year progression-free survival were 88%±5% and 83%±7% respectively. Infusion-related adverse events occurred in 4% of patients, associated with the first infusion of rituximab. Subanalyses according to subtype, stage, IPI and other factors will be presented. Conclusion: Rituximab plus chemotherapy is an effective, well-tolerated treatment that achieves high response rates and long progression-free survival in both indolent and aggressive NHL.

Randomized Comparison of Cladribine Containing Regimens and COP in Previously Untreated Patients with Small Lymphocytic, Marginal Zone and Follicular Lymphoma.

Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 4739-4739
Author(s):  
Ewa Kalinka ◽  
Jaroslaw Wajs ◽  
Kazimierz Sulek ◽  
Tadeusz Robak ◽  
Maria Blasinska-Morawiec ◽  
...  
Keyword(s):  
Side Effects ◽  
Overall Response Rate ◽  
Marginal Zone ◽  
Response Rate ◽  
Low Grade ◽  
Front Line ◽  
Histological Subtypes ◽  
First Line ◽  
To Receive

Abstract The aim of the study was to comparatively assess first-line treatment with cladribine alone or in combination with cyclophosphamide (CCR, cladribine-containing regimens) and COP (cyclophosphamide, vincristine, prednisone) in different subtypes of low-grade lymphoma. End points were complete remission (CR), overall response rate (ORR), incidence of chemotherapy-related side effects as well as freedom from progression (FFP) and overall survival (OS). From June’2000 to June’2005, 178 previously untreated patients (pts) were randomly allocated to receive 6 monthly courses of either CCR or COP in 17 centers in Poland. This analysis included 107 pts who have completed scheduled chemotherapy, including 45 pts with small lymphocytic (SLL, median age=64 years), 26 marginal zone (MZL, median age=58 years) and 36 follicular (FL, median age=65 years) lymphoma. Compared to COP, CCR induced higher CR rates in all treated groups (65% vs 15%, p=.005; 57% vs 10%, p=.02; 58% vs 12%, p=.03, respectively) but differences in ORR were not significant (92% vs 69%; 92% vs 60%; 79% vs 62%, respectively). Incidence of side effects did not differ significantly in CCR- as compared to COP-treated pts, e.i. infections (10% vs 7%; 14% vs 20%; 15% vs 0%, respectively), myelosuppression (31% vs 7%; 21% vs 20%; 30% vs 0%, respectively), and non-hematological adverse events (10% vs 14%; 7% vs 30%; 7% vs 22%, respectively). With a median follow-up of 12 months, median FFP was superior in CCR- as compared to COP-treated treated pts with SLL (43 vs 12 months, log-rank p<.03) or MZL (37 vs 7 months, log-rank p<.03) but not with FL (17 vs 22 months). Although the median OS has not been reached in any of the histological group so far, no difference in its duration is detected between CCR- or COP-treated pts. In summary, for pts with SLL, MZL and FL, first-line CCR regimens provided better CR and similar toxicity rates as compared to COP, which translated into longer FFP in SLL and MZL but not in FL pts. Although these results warrant larger number of pts and longer follow-up, they might suggest the choice of different front-line chemotherapy with or without immunotherapy in particular histological subtypes of low-grade lymphoma.

A Phase I/II Trial of Bortezomib, Low Dose Intravenous Melphalan and Dexamethasone for Patients with Relapsed Multiple Myeloma.

Blood ◽  
2006 ◽  
Vol 108 (11) ◽  
pp. 3542-3542 ◽  
Author(s):  
Rakesh Popat ◽  
Catherine Williams ◽  
Mark Cook ◽  
Charles Craddock ◽  
Supratik Basu ◽  
...  
Keyword(s):  
Multiple Myeloma ◽  
Phase I ◽  
Median Time ◽  
Low Dose ◽  
Overall Response Rate ◽  
Response Rate ◽  
Chemotherapeutic Agents ◽  
Time To Progression ◽  
Overall Response ◽  
Grade 3

Abstract Background: Bortezomib is an effective treatment for patients with relapsed multiple myeloma with an overall response rate (MR+PR+CR) of 46% and time to progression of 6.2 months (APEX study). We and others have previously demonstrated potent in-vitro synergy with chemotherapeutic agents such as melphalan and it is likely that this will translate into improved responses in the clinical setting. Methods: This was a multi-centre, non-randomised Phase I/II clinical trial for patients with relapsed multiple myeloma. Bortezomib 1.3mg/m2 was given on Days 1,4,8 and 11 of a 28 day cycle, and intravenous melphalan on Day 2 for a maximum of 8 cycles. In the Phase I component melphalan was given at 2.5, 5,7.5 and 10mg/m2 in a dose escalation scheme and the maximum tolerated dose (MTD) of 7.5mg/m2 was taken forward to an expanded Phase II component. Dexamethasone 20mg on the day of and the day after each dose of bortezomib was permitted for progressive or stable disease after 2 or 4 cycles respectively. Responses were classified by EBMT criteria. Results: To date 39 patients have been enrolled (median age 61years [range 40–77]) with a median of 3 lines of prior therapy [range 1–5] of which 26 (67%) have had one previous autologous stem cell procedure and 4 (10%) have had two. 23 (59%) have had prior exposure to thalidomide and 4 (10%) to bortezomib. 36 have now completed at least 1 cycle and are therefore evaluable for response. The overall response rate (CR+PR+MR) across all treatment levels was 75% rising to 81% (CR 11%; nCR 3%; VGPR 8%; PR 39%; MR 19%) with the addition of dexamethasone in 13 cases for suboptimal response. Rapid responses were seen with the median time to response being 1 month [range 1–6]. The median time to progression is 10.1 months and the median overall survival has not yet been reached at a median follow-up of 7.4 months. Of the patients that have had disease progression 7 (35%) had responses of longer duration than their previous therapy. The MTD was defined by unacceptable delays in administering treatment due to myelosuppresion. The toxicities have been acceptable with 13 SAEs reported of which 8 were hospitalisation due to infection. The most common grade 3–4 adverse events were: thrombocytopenia (53%), infections (25%), neutropenia (17%) and neuropathy (17%). Three grade 3 cardiac events were seen (myocardial infarction, atrial fibrillation and cardiac failure) and GCSF was administered to 13 patients as treatment and prophylaxis of grade 4 neutropenia. 13 patients were withdrawn from the study due to toxicity of which 7 were for neuropathy and 3 for delayed haematological recovery. Of note, 11 patients (28%) had pre-existing grade 1 neuropathy prior to starting therapy. Summary: The combination of bortezomib, low dose intravenous melphalan and dexamethasone appears to be highly effective in patients with relapsed multiple myeloma where a response rate of 81% is seen with 14% achieving nCR/CR. The toxicity profile associated is predictable, manageable and predominantly haematological. Recruitment is ongoing to a total of 53 patients.

Bortezomib, Low Dose Intravenous Melphalan and Dexamethasone for Patients with Relapsed Multiple Myeloma: Final Results of a Phase I/II Clinical Trial.

Blood ◽  
2007 ◽  
Vol 110 (11) ◽  
pp. 2713-2713
Author(s):  
Rakesh Popat ◽  
Catherine Williams ◽  
Mark Cook ◽  
Charles Craddock ◽  
Supratik Basu ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Multiple Myeloma ◽  
Phase I ◽  
Median Time ◽  
Low Dose ◽  
Overall Response Rate ◽  
Response Rate ◽  
Time To Progression ◽  
Overall Response ◽  
Grade 3

Abstract Background: Bortezomib is an effective treatment for patients with relapsed multiple myeloma with an overall response rate (≥PR) of 43% and time to progression of 6.2 months (APEX study). We and others have previously demonstrated potent in-vitro synergy with chemotherapeutic agents such as melphalan and it is likely that this will translate into improved responses in the clinical setting. Methods: This was a multi-centre, non-randomised Phase I/II clinical trial for patients with relapsed multiple myeloma. Bortezomib 1.3mg/m2 was given on Days 1,4,8 and 11 of a 28 day cycle, and intravenous melphalan on Day 2 for a maximum of 8 cycles. In the Phase I component melphalan was given at 2.5, 5,7.5 and 10mg/m2 in a dose escalation scheme and the maximum tolerated dose (MTD) of 7.5mg/m2 was taken forward to an expanded Phase II component. Dexamethasone 20mg on the day of and the day after each dose of bortezomib was permitted for progressive or stable disease after 2 or 4 cycles respectively. Responses were defined by EBMT criteria. Results: 53 patients were enrolled (median age 61years [range 40–77]) with a median of 3 lines of prior therapy [range 1–5] of which 26 (67%) have had one previous autologous stem cell procedure and 4 (10%) have had two. 23 (59%) have had prior exposure to thalidomide and 4 (10%) to bortezomib. The overall response rate (≥PR) across all treatment levels (n=52) was 65% rising to 69% (CR 19%; nCR 4%; VGPR 6%; PR 40%; MR 15%) with the addition of dexamethasone in 27 cases for suboptimal response. Of the 32 patients treated at the MTD the overall response rate (≥PR) was 78% (CR 28%; nCR 6%; VGPR 6%; PR 38%; MR 9%). Rapid responses were seen with the median time to response being 1 month [range 1–6]. The median time to progression was 10 months and the median overall survival has not yet been reached at a median follow-up of 17 months. Of the patients that have had disease progression 7 (35%) had responses of longer duration than their previous therapy. The MTD was defined by unacceptable delays in administering treatment due to myelosuppresion. The toxicities have been acceptable with 13 SAEs reported of which 8 were hospitalisation due to infection. The most common grade 3–4 adverse events were: thrombocytopenia (53%), infections (25%), neutropenia (17%) and neuropathy (17%). Three grade 3 cardiac events were seen (myocardial infarction, atrial fibrillation and cardiac failure) and GCSF was administered to 13 patients as treatment and prophylaxis of grade 4 neutropenia. 19 patients were withdrawn from the study due to toxicity of which 7 were for neuropathy and 3 for delayed haematological recovery. Of note, 11 patients (28%) had pre-existing grade 1 neuropathy prior to starting therapy. Summary: The combination of bortezomib, low dose intravenous melphalan and dexamethasone appears to be highly effective in patients with relapsed multiple myeloma with a response rate (≥PR) at the MTD of 78% including 34% nCR/CR. The toxicity profile is predominantly haematological.

Pananemia 2008 multicenter observational study on erythropoietin beta treatment in patients with cancer: Efficacy, patient satisfaction, and impact on psychological distress

2009 ◽  
Vol 27 (15_suppl) ◽  
pp. e20695-e20695
Author(s):  
S. Del Prete ◽  
R. Addeo ◽  
L. Leo ◽  
S. Cinieri ◽  
V. Lorusso ◽  
...  
Keyword(s):  
Patient Satisfaction ◽  
Psychological Distress ◽  
Cancer Patients ◽  
Overall Response Rate ◽  
Response Rate ◽  
Multicenter Observational Study ◽  
Overall Response ◽  
To Receive ◽  
Self Administered Questionnaire ◽  
Anemia Treatment

e20695 Background: Cancer-related anemia, results in both a need for transfusions and a decreased functional capacity and quality of life. Treating anemia associated with chemotherapy and many cancers is often necessary. However, patient satisfaction with anemia treatment, and the possible correlation between anemia and psychological distress frequently present in these patients is limited by the lack of validated instruments. Methods: Between January 2008 and December 2008, 591 cancer patients in treatment with erythropoietin beta for anemia were targeted to complete the Psychological Distress Inventory (PDI), a 13-item self-administered questionnaire, and the Patient Satisfaction Questionnaire ( PSQ) at 4 week intervals, a 10-item, self-administered questionnaire. Data from weeks 5 and 9 were analyzed. Patients were required to receive at least four weekly injections of Epo, expecting to receive ≥ 8 additional weeks of chemotherapy, and able to complete questionnaires. Results: Among patients fulfilling eligibility criteria and having received at least four Epo Beta administrations, most (57.5%) of them were female, with a median age of 66 (52.4 - 76.5), and a median KPS of 85 (range: 50–100). 399 patients had a stage IV cancers. Hemoglobin values increased from mean baseline levels of 9.55 g/dL, to attain levels 10.31 at week 5, and 11.05 after 8 weeks of therapy; 247 (42%) patients received iron supplementation. For PDI, the overall response rate was 93 % (548/591) at baseline, 100 % (517/517) at week 5, and 99% (491/492) at week 9. The percentage of patients with psychological difficulties decreased during the treatment. For PSQ, the overall response rate was 100% (517/517) at week 5, and 100 % (492/492) at week 9. The PSQ questionnaires showed that a conspicuous group of patients (124/517) marked troubles to accept the treatment. Conclusions: Our results suggest that in anemic cancer patients psychological distress and anemia were related. PSQ reflect the burden of injection anemia treatment on cancer patients. Final data analysis will be presented. No significant financial relationships to disclose.

scholarly journals Combining 308-monochromatic excimer phototherapy with monthly IM triamcinolone acetonide for the treatment of resistant alopecia totalis

2021 ◽  
pp. 37
Author(s):  
Keyword(s):  
General Practice ◽  
Triamcinolone Acetonide ◽  
Overall Response Rate ◽  
Response Rate ◽  
Young Patients ◽  
Interventional Study ◽  
Younger Patients ◽  
History Of ◽  
To Receive

Background: Treatment of resistant alopecia totalis AT is a major problem in general practice. Some studies reported the use of either excimer-308 or intra-muscular triamcinolone acetonide as a monotherapy, with conflicting results. Objective: To evaluate the therapeutic effect of combining 308-excimer phototherapy and intramuscular triamcinolone acetonide for the treatment of alopecia totalis. Methods and Material: Ten patients with alopecia totalis were evaluated in this prospective interventional study. All patients were assigned to receive the thera-peutic regimen that includes monthly IM triamcinolone acetonide (TAC) for a maximum of six pulses and twice-weekly excimer phototherapy for 24 sessions. Results: The overall response rate for this regimen was 90%, with four patients 40% achieving complete regrowth of hair (100%). Three patients have exhibited a satisfactory response (>70% regrowth). Unsatisfactory response ( >10-< 70% regrowth) was reported in two patients . Younger patients responded better, as did those with a shorter history of the disease P < 0.05. At follow-up, which continued for 8–12 months, recurrence was noted in two (22.2%) of the nine responders. Conclusions: Combining excimer phototherapy with triamcinolone acetonide showed a promising effect on resistant AT. This treatment modality was effective and well tolerated particularly in young patients.

scholarly journals FINAL Results of an Phase, Multicenter, Randomied, Controlled OPEN LEVEL Trial: Decitabine Therapy in Patients with Myelodysplastic Syndromes

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 3865-3865
Author(s):  
Zonghong Shao ◽  
Hui Liu ◽  
Hao Jiang ◽  
Hongyan Tong ◽  
Ruixiang Xiang ◽  
...  
Keyword(s):  
Clinical Trials ◽  
High Risk ◽  
Adverse Events ◽  
Myelodysplastic Syndromes ◽  
Overall Response Rate ◽  
Response Rate ◽  
Conflicts Of Interest ◽  
Study Drug ◽  
Overall Response ◽  
To Receive

Abstract Background: DNA hypomethylating agent, decitabine, has become the current standard therapy for patients with higher-risk myelodysplastic syndromes (MDS). Decitabine was launched in China in August 2009 without clinical trials. According to some retrospective studies, the efficacy and safety are similar to those reported in other countries, but there is still a lack of large-scale prospective clinical trials. So we start a prospective clinical trial in China to compare the effect and safety of decitabine in MDS, which was registered at clinicaltrials.gov (NCT02013102). Design: Adults with intermediate or high risk MDS by the International Prognostic Scoring System (IPSS≥0.5) were randomized to receive either decitabine 20 mg/m2 IV daily for 5 days (arm Ⅰ) or decitabine 12 mg/m2 IV daily for 8 days (arm Ⅱ) every four weeks. Patients continued to receive study drug for 4 cycles until death, disease progression, intercurrent illness preventing further administration of treatment, unacceptable adverse event or decision by the patient to withdraw from the study. And supportive care were permitted. The primary end point was overall response rate (ORR, CR+mCR+PR) by International Working Group (IWG 2006) criteria, secondary end points included CR, mCR, PR, HI, safety, et al. Results: We enrolled a total of 198 patients between 8/2013 and 12/2017, among which 7 patients didn't take decitabine, and 191 were included in the analysis. 94 in arm Ⅰ recieved decitabine and 97 in arm Ⅱ. 32.8% of patients withdrew from the study for a variety of reasons, including progression and death (5.1%), personal decision (13.6%), adverse events (6.6%), and other causes (7.6%). The median age of patients in arm Ⅰ was 54.88 years old and 54.82 years old in arm II. The median follow-up was 106 days for patients in both arms. The patients received a mean 2.5 cycles of decitabine therapy for arm Ⅰ and 2.0 cycles for arm Ⅱ. The overall response rate was 39.3% in total, and 41.5% and 38.1% (p=0.6598) for patients in arm Ⅰ and arm Ⅱ, respectively. And CR was 18.1% and 14.4% (p= 0.5584) , PR was 6.4% and 3.1% (p=0.3257) , mCR was 17.0% and 20.6% (p=0.5814) , HI was 3.2% and 1.0% (p=0.3633) , for patients in armⅠand armⅡ, respectively (Table 1). Among all patients, 38.7% were intermediate-1 risk, 40.3% were intermediate-2 risk, 20.4% were high risk. Analysis of response by MDS patient subtypes is shown in Table 2. Those who were higher risk experienced higher ORR and CR, while the difference is not significant between two arms (p>0.05). As expected, cytopenias were the most frequent complications (76.4%). Grade 3-4 neutropenia, thrombocytopenia and anemia considered to be at least possibly related to the study drug occurred at rates of 23.0%, 34.6%, and 34.6% of patients, respectively. Nonhematologic adverse events were also common including abnormal metabolism and nutrition (23.40% vs 18.56%), abnormal gastrointestinal function (29.79% vs 41.24%), cardiac disorders (11.70% vs 14.43%), infection and infectious diseases (32.98% vs 36.08%), abnormal skin and subcutaneous tissue and so on, which were no significant differences between two ams. During the study there were 17 SAE, only 7 cases were possibly related to drug therapy, such as pulmonary infection, Sepsis, myelosuppression, intracranial hemorrhage, hepatic failure, and arrhythmia. Conclusions: The use of 5-day and 8-day schedule decitabine is safe and effective in patients with intermediate and high risk MDS, among which there was no significant differences. Disclosures No relevant conflicts of interest to declare.

scholarly journals Real-World Effectiveness and Safety of Eltrombopag in Patients with Aplastic Anemia: A Chinese Nationwide Survey

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 5020-5020
Author(s):  
Wenrui Yang ◽  
Bing Han ◽  
Hong Chang ◽  
Bingyi Wu ◽  
Fankai Meng ◽  
...  
Keyword(s):  
Aplastic Anemia ◽  
Real World ◽  
Overall Response Rate ◽  
Response Rate ◽  
Line Treatment ◽  
First Line ◽  
Overall Response ◽  
Treatment Naïve ◽  
First Line Treatment

Immunosuppressive therapy (IST) based on antithymoglobin (ATG) and cyclosporin (CsA) is the first-line treatment for severe aplastic anemia (SAA) patients who are unfit for transplantation,and the overall response rate (ORR) is about 70%.The utility of eltrombopag (EPAG),a TPO receptor agonist, achieved robust hematologic response in refractory and treatment-naïve SAA patients in clinical trials and some other studies. However, only a few data came from Asia countries where higher incidence of AA has been reported. A retrospective study on the use of EPAG in AA was conducted in mainland China. Aplastic anemia (transfusion dependent non-severe, severe, and very severe) patients who started eltrombopag before Feb 2019 and continued for at least 3 months either as first-line treatment or as rescue treatment, were enrolled. The maximum daily dosage of EPAG used continuously for at least 2 weeks is defined as the stable dosage. Response criteria were referred to that used in previous reports (Townsley DM, NEJM 2017; BCSH, BJH 2016). Fifty-six patients from eleven centers were enrolled in this study, including 26 males and 30 females at the median age of 39 (7-80) years. All patients were transfusion-dependent by the time of EPAG administration, and there were 14 VSAA, 24 SAA and 18 transfusion dependent non-severe aplastic anemia (TD-NSAA). Nineteen treatment-naïve patients received EPAG and IST (ATG+CsA, n=10; CsA/CsA+androgen, n=9) as first-line treatment. Thirty-seven patients were refractory to IST. Eltrombopag was administered at a median dose of 75 (25-150) mg per day for 7 (3-31) month. The median follow-up time was 9 (3-40) months. The overall response rate in patients receiving EPAG as first-line therapy was 78.9% (15/19), and most patients achieved complete response (CR) (10/15). Among the 10 patients receiving ATG+CsA, 6 patients achieved hematologic response (HR) at 3 months post-treatment, including 3 CR. Six patients were diagnosed as VSAA and three achieved HR. For the 9 patients treated with CsA/CsA+androgen, 8 achieved HR (88.9%) and 4 were CR (44.4%) at 3 months. By last follow-up, the cumulative HR rate was 70% in ATG+CsA group and 89% in CsA/CsA+ androgen group. Among the 14 responders, 11 patients receiving EPAG at a stable dosage ≤75mg/d and achieved HR at 3 months. The overall response rate in IST-refractory patients was 46% (17/37), with trilineage response in 27% patients at 3 months. For the 18 ATG+CsA refractory SAA patients,trilineage HR occurred in 4 patients (22.2%, 4/18), bi-lineage HR in one patient and single lineage HR in one patient. Thus, the total HR was 33.3% (6/18) at 3 months and increased to 44% (8/18) by last follow-up. Among the 19 CsA/CsA+ androgen refractory patients, 6 (31.5%, 6/19) achieved trilineage HR, one achieved bi-lineage HR and 4 achieved single lineage response. Total HR rate was 57.9% (11/19) at 3 months after EPAG initiation and 68% (13/19) by last follow-up, including 9 patients with trilineage HR. Among 17 responders, 13 received a stable EPAG dose of≤75mg/d. Most patients tolerated EPAG well. Adverse events occurred in 29 patients (52%) and most were mild to moderate, including gastrointestinal symptom (n=15, e.g. abdominal pain, nausea), impaired liver function (n=5), skin changes (n=7, e.g. skin pruritus and rash) and musculoskeletal pain (n=6), and venous thrombus (n=2). Eltrombopag dosage was reduced in 2 patients due to severe digestive symptoms at 100 mg/d and discontinued in one patient who suffered from upper limb venous thrombus. In conclusion, EPAG is effective and safe in treating Chinese AA patients at a daily dose of 75mg and less. The real-world result of EPAG in Chinese patients is similar to those reported in Western countries. Disclosures No relevant conflicts of interest to declare.

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