scholarly journals Incidence and Treatment of Myelodysplastic Syndrome in the US: Treatment Approaches, Optimization of Care and the Need for Additional Therapeutic Agents

Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 1287-1287 ◽  
Author(s):  
Erin P. Demakos ◽  
Lewis R. Silverman ◽  
Moira E. Lawrence ◽  
Thomas J. McKearn ◽  
Scott Megaffin ◽  
...  
Keyword(s):  
Clin Oncol ◽  
Unmet Need ◽  
Median Number ◽  
Medical Community ◽  
Interventional Treatment ◽  
Watch And Wait ◽  
Medical Claim ◽  
Target Number ◽  
The Us ◽  
Number Of Cycles

Abstract Background : The incidence of myelodysplastic syndromes (MDS) - a heterogeneous group of malignant myeloid stem cell disorders - increases with age and commonly affects older people. The prevalence of MDS in the US has been constantly rising as a result of increasing longevity of the overall population. Analyses of healthcare claims data using associated medical claims information (ICD-9-CM diagnosis codes) are a common way to estimate the number patients (pts) receiving care in specific disease states. We examined the total of unique US claims for MDS submitted over a 3-year period and also analyzed the claims according to type of treatment. Methods : We conducted a retrospective cohort study of patients (pts) with an MDS-associated medical claim (ICD-9-CM diagnosis code 238.7x) in the observation (OB) period (calendar years 2009-2011). In each year of the OB period, pts were classified according to type of treatment: watch and wait (ie, receiving no drug therapy) or interventional treatment [ie, supportive care treatment with erythropoietin stimulating agents (ESA) or growth factors (GF) and active drug treatment (ie, the hypomethylating agents (HMA) azacitidine (AZA) and decitabine (DEC) and the immunomodulating agent lenalidomide (REV)]. A subgroup of newly diagnosed MDS pts was also identified in Years 2 and 3, but this group was not included in Year 1 of the OB period (calendar year 2009); this group of new-to-treatment patients had a claim for MDS in Years 2 and 3 of the OB period. MDS incidence rates were then determined within Years 2 and 3 of the OB period for this group. The total number of physicians treating pts coded for MDS was also collected. Results : We identified more than 100,000 unique pts with an MDS-associated claim in each of the 3 years of the OB period. Our calculated incidence of newly treated MDS pts (34,000) in Years 2 and 3 is consistent with recently reported estimates (Cogle et al, Blood 2011; Goldberg et al, J Clin Oncol 2010) but higher than the SEER database. Over the 3-year OB period, the number of diagnosed and treated MDS pts grew year on year and grew at a slower rate than that of the US population. Watch-and-wait is the mainstay treatment for 47% of MDS pts. We found that 6000 pts per month are treated with an HMA by 2100 physicians, or 2.8 pts per physician and 14,000 pts receive therapy for MDS comprised of ESA, GF, AZA, DEC or REV per year. AZA and DEC were the predominant HMA treatments prescribed for higher-risk MDS. Approximately 30% of HMA-treated reached the target number of 6 cycles. HMA therapies were used in 13.1% of pts. A larger percentage of the AZA- and DEC-treated pts (69.1%) stopped therapy before reaching the target number of doses, with approximately 32% of pts who initiated therapy dropping out after the first cycle. Conclusions : The total number of pts coded for MDS-associated ICD-9 billing in the US each year is substantial. The estimated incidence of 34,000 patients per year with MDS in the US in 2010 and 2011 is similar to results found in other epidemiological databases. The majority of pts are not treated with the optimal number of cycles with an HMA. The average physician treats only a limited number of pts with MDS, which may influence treatment decisions. These data suggest a need for further educational efforts to optimize care with additional insight into past population-based estimates of diagnosis and treatment (Cogle et al, Blood 2011; Goldberg et al, J Clin Oncol 2010). The data showing early discontinuation or failure of HMA therapy accentuates the poor prognosis of patients post-HMA, who have a predicted median survival of 4-6 months (Prebet et al, J Clin Oncol 2011). These data may serve to better inform the medical community of the unmet need of pts who are not successfully treated with first-line MDS therapies, underscoring the need for optimization of care and the need for additional agents beyond those currently available. Table HMA Cycle and Administration Metrics per Line of Therapy Treatment Metric Moving Annual Total 2010 2011 2012 Dacogen Median number of cycles 4-day admin schedule 5-day admin schedule 3.00 14.7% 64.0% 4.00 11.4% 67.6% 3.00 12.6% 66.2% Vidaza Median number of cycles 5-day admin schedule 7+ day admin schedule 4.00 47% 20% 4.00 49% 20% 4.00 46% 20% Disclosures Demakos: Onconova Therapeutics, Inc: Consultancy. Silverman:Onconova Therapeutics, Inc: Consultancy. Lawrence:Onconova Therapeutics, Inc. : Employment. McKearn:Onconova Therapeutics, Inc. : Employment. Megaffin:Onconova Therapeutics, Inc. : Employment. Percy:Onconova Therapeutics, Inc. : Employment. Petrone:Onconova Therapeutics, Inc. : Employment, Stock Options Other.

Breastfeeding

2018 ◽  
Author(s):  
Marcie Richardson
Keyword(s):  
Infant Nutrition ◽  
Milk Composition ◽  
World Health ◽  
Medical Community ◽  
Baby Friendly Hospital Initiative ◽  
Skin Contact ◽  
The Us ◽  
The World ◽  
Breastfeeding Rates ◽  
Skin To Skin

Breastfeeding is endorsed by the medical community as the optimal nutrition for infants during the first 6-12 months of life.1,2,3  Breastfeeding rates in the US and worldwide have varied over time and still vary geographically.4 There is robust literature addressing the physiology of lactation, composition of breast milk, and health advantages of breastfeeding for both the mother and infant as well as strategies for clinicians to promote and support breastfeeding. This chapter reviews breastfeeding history, how milk is made, why breastfeeding matters, and the somewhat controversial the World Health Organization’s Baby Friendly Hospital Initiative (BFHI)5 for successful initiation of lactation as well as some special situations.    Key words:  breastfeeding, infant nutrition, human milk composition, breastfeeding advantages, lactation, lactation support, Baby Friendly Hospital Initiative, skin to skin contact

scholarly journals Impact of Induction Treatment on Stem Cell Collection: A COVID Related Study

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 4848-4848
Author(s):  
Brad Rybinski ◽  
Ashraf Z. Badros ◽  
Aaron P. Rapoport ◽  
Mehmet Hakan Kocoglu
Keyword(s):  
Stem Cells ◽  
Stem Cell ◽  
Research Funding ◽  
Median Number ◽  
Cell Transplant ◽  
Cd34 Cells ◽  
Significant Difference ◽  
Number Of Cycles ◽  
Time Required ◽  
Stem Cell Collection

Abstract Introduction: Standard induction therapy for multiple myeloma consists of 3-6 cycles of bortezomib, lenalidomide, and dexamethasone (VRd) or carfilzomib, lenalidomide and dexamethasone (KRd). Receiving greater than 6 cycles of a lenalidomide containing regimen is thought to negatively impact the ability to collect sufficient CD34+ stem cells for autologous stem cell transplant (Kumar, Dispenzieri et al. 2007, Bhutani, Zonder et al. 2013). Due to the COVID-19 pandemic, at least 20 patients at University of Maryland Greenebaum Comprehensive Cancer Center (UMGCC) had transplant postponed, potentially resulting in prolonged exposure to lenalidomide containing induction regimens. Here, in the context of modern stem cell mobilization methods, we describe a retrospective study that suggests prolonged induction does not inhibit adequate stem cell collection for transplant. Methods: By chart review, we identified 56 patients with multiple myeloma who received induction with VRd or KRd and underwent apheresis or stem cell transplant at UMGCC between 10/1/19 and 10/1/20. Patients were excluded if they received more than 2 cycles of a different induction regimen, had a past medical history of an inborn hematological disorder, or participated in a clinical trial of novel stem cell mobilization therapy. We defined 1 cycle of VRd or KRd as 1 cycle of "lenalidomide containing regimen". In accordance with routine clinical practice, we defined standard induction as having received 3-6 cycles of lenalidomide containing regimen and prolonged induction as having received 7 or more cycles. Results: 29 patients received standard induction (Standard induction cohort) and 27 received prolonged induction (Prolonged induction cohort) with lenalidomide containing regimens. The median number of cycles received by the Standard cohort was 6 (range 4-6), and the median number of cycles received by the Prolonged cohort was 8 (range 7-13). The frequency of KRd use was similar between patients who received standard induction and prolonged induction (27.58% vs. 25.93%, respectively). Standard induction and Prolonged induction cohorts were similar with respect to clinical characteristics (Fig 1), as well as the mobilization regimen used for stem cell collection (p = 0.6829). 55/56 patients collected sufficient stem cells for 1 transplant (≥ 4 x 10 6 CD34 cells/kg), and 40/56 patients collected sufficient cells for 2 transplants (≥ 8 x 10 6 CD34 cells/kg). There was no significant difference in the total CD34+ stem cells collected at completion of apheresis between standard and prolonged induction (10.41 and 10.45 x 10 6 CD34 cells/kg, respectively, p = 0.968, Fig 2). Furthermore, there was no significant correlation between the number of cycles of lenalidomide containing regimen a patient received and total CD34+ cells collected (R 2 = 0.0073, p = 0.5324). Although prolonged induction did not affect final stem yield, prolonged induction could increase the apheresis time required for adequate collection or result in more frequent need for plerixafor rescue. There was no significant difference in the total number of stem cells collected after day 1 of apheresis between patients who received standard or prolonged induction (8.72 vs. 7.96 x 10 6 cells/kg, respectively, p = 0.557). However, patients who received prolonged induction were more likely to require 2 days of apheresis (44% vs. 25%, p = 0.1625) and there was a trend toward significance in which patients who received prolonged induction underwent apheresis longer than patients who received standard induction (468 vs 382 minutes, respectively, p = 0.0928, Fig 3). In addition, longer apheresis time was associated with more cycles of lenalidomide containing regimen, which neared statistical significance (R 2 = 0.0624, p = 0.0658, Fig 4). There was no significant difference between standard and prolonged induction with respect to the frequency of plerixafor rescue. Conclusions: Prolonged induction with lenalidomide containing regimens does not impair adequate stem cell collection for autologous transplant. Prolonged induction may increase the apheresis time required to collect sufficient stem cells for transplant, but ultimately clinicians should be re-assured that extending induction when necessary is not likely to increase the risk of collection failure. Figure 1 Figure 1. Disclosures Badros: Janssen: Research Funding; J&J: Research Funding; BMS: Research Funding; GlaxoSmithKline: Research Funding.

scholarly journals Professor Liberato J.A. DiDio - A great anatomist of the 20th century and an advocate of medicine without borders

2018 ◽  
Vol 146 (5-6) ◽  
pp. 351-355
Author(s):  
Gordana Teofilovski-Parapid ◽  
Maria Miglino
Keyword(s):  
20Th Century ◽  
Gold Medal ◽  
Surgical Anatomy ◽  
Medical Community ◽  
Professor Emeritus ◽  
Medical College ◽  
Editorial Boards ◽  
Topographical Anatomy ◽  
The Us ◽  
Belo Horizonte

Liberato J. A. DiDio (1920?2004) was one of the most prominent figures of anatomy belonging to the 20th century?s second half and an open-minded man. In 1984, during the era of communism in Yugoslavia, he opened the doors of the Medical College of Ohio (MCO) in Toledo, OH, USA, to a Serbian doctor. During the troubled times for people and anatomists in Serbia in 1994, he saved their association from being expelled from the International Federation of Anatomical Associations. In 1999, only a few months after the bombing of Yugoslavia, he helped them to get the organization of the XVIII International Symposium on Morphological Sciences in 2005, the meeting of the leaders in the field. Serbian anatomists and clinicians proved that he was right when considering them on a par with their peers in the international anatomical and medical community. Professor DiDio first showed talent with Gold Medal ? top graduate at his high school, and La Royale Award (Graduation Golden Ring) ? top graduate MD. He was trained in Brazil, Italy, and the US. He was the Founding Chairman ? Department of Topographical Anatomy, Faculdade de Ciencias Medicas, Belo Horizonte, Minas Gerais, Brazil; Head of Gross and Surgical Anatomy, Northwestern University Medical, Dental, and Graduate Schools; Founding Chairman, Department of Anatomy, MCO; Professor Emeritus at the age of 70 (1990), Assistant to the President of the MCO, Consultant to the President and the Emeritus Dean (1992?2004). He was a member of editorial boards of 34 journals, academic adviser in 92 M.S. and Ph.D. theses.

scholarly journals Resecting the Dominant Lesion: Patient Outcomes after Surgery and Radiosurgery vs Stand-Alone Radiosurgery in the Setting of Multiple Brain Metastases

2021 ◽  
Author(s):  
Maria Punchak ◽  
Stephen P Miranda ◽  
Alexis Gutierrez ◽  
Steven Brem ◽  
Donald O'Rourke ◽  
...  
Keyword(s):  
Brain Metastases ◽  
Surgical Resection ◽  
Gamma Knife Radiosurgery ◽  
Median Number ◽  
University Of Pennsylvania ◽  
Multiple Brain Metastases ◽  
The Us ◽  
Median Diameter ◽  
Similar Survival ◽  
The University

Abstract BACKGROUND: Brain metastases are the most common central nervous system (CNS) tumors, occurring in 300,000 people per year in the US. The benefit of surgical resection, over radiosurgery, for dominant lesions remains unclear. METHODS: The University of Pennsylvania Health System database was retrospectively reviewed for patients presenting with multiple brain metastases from 1/1/16 to 8/31/18 with one dominant lesion > 2 cm in diameter, who underwent initial treatment with either resection of the dominant lesion or Gamma Knife radiosurgery (GKS). Inclusion criteria were age > 18, >1 brain metastasis, and presence of a dominant lesion (>2 cm). We analyzed factors associated with mortality. RESULTS: 129 patients were identified (surgery=84, GKS=45). The median number of intracranial metastases was 3 (IQR: 2-5). The median diameter of the largest lesion was 31 mm (IQR: 25-38) in the surgery group vs 21 mm (IQR: 20-24) in the GKS group (p<0.001). Mortality did not differ between surgery and GKS patients (69.1% vs 77.8%, p = 0.292). In a multivariate survival analysis, there was no difference in mortality between the surgery and GKS cohorts (aHR: 1.35, 95% CI: 0.74-2.45 p=0.32). Pre-operative KPS (aHR: 0.97, 95% CI: 0.95-0.99, p=0.004), CNS radiotherapy (aHR: 0.33, 95% CI: 0.19-0.56 p<0.001), chemotherapy (aHR: 0.27, 95% CI: 0.15-0.47, p<0.001), and immunotherapy (aHR: 0.41, 95% CI: 0.25-0.68, p=0.001) were associated with decreased mortality. CONCLUSION: In our institution, patients with multiple brain metastases and one symptomatic dominant lesion demonstrated similar survival after GKS when compared with up-front surgical resection of the dominant lesion.

AIDS and Doctors

PEDIATRICS ◽  
1989 ◽  
Vol 83 (6) ◽  
pp. A38-A38
Author(s):  
R. J. M.
Keyword(s):  
Health Care ◽  
Ethical Issues ◽  
Immune Deficiency Syndrome ◽  
Deficiency Syndrome ◽  
Medical Community ◽  
Columbia University ◽  
Physical Handicap ◽  
The Us ◽  
Attending Physician ◽  
Care Worker

Santa Monica, CA—. . .While there isn't a single documented case of a patient catching acquired immune deficiency syndrome from a doctor or other healthcare worker, medical caregivers with AIDS—and those who have tested positive for the AIDS virus but don't have the disease—raise difficult and sensitive questions for the medical community. The central question: whether these people should continue to treat and maintain contact with patients. "What you have are fundamental ethical issues of fairness, justice and moral responsibility," says Ronald Bayer, a Columbia University bioethics professor. . . As of last September 3, 182 health-care workers were known to have AIDS, including 356 physicians, according to the US Centers for Disease Control. . . most hospitals that have discovered an attending physician or health-care worker with AIDS do try to re-assign the individuals to other duties, especially if they are involved in performing invasive procedures. But. the courts have consistently held that AIDS is a physical handicap protected under state and federal anti-discrimination laws. . . . Procedures used to handle the question of what to do about doctors with AIDS will only work if these doctors identify themselves. But a handful of infected physicians have learned the hard way what happens when word of their condition leaks out. . . (After a newspaper report revealed that a Dallas-area pediatrician had AIDS last year) local medical authorities assured parents that the doctor wasn't a threat to their children. But the assurances did no good, and his practice collapsed. "Any risk is too great when you're dealing with my children," (said the parent of a patient.). . .(The pediatrician's) advice to other doctors with AIDS: "Don't tell anyone."

UnCHAARTED territory: The role of docetaxel rechallenge following chemohormonal therapy for metastatic castrate-sensitive prostate cancer.

2021 ◽  
Vol 39 (6_suppl) ◽  
pp. 117-117
Author(s):  
Mary Mahler ◽  
Esmail Mutahar Al-Ezzi ◽  
Noa Shani Shrem ◽  
Eric Winquist ◽  
Christina M. Canil ◽  
...  
Keyword(s):  
Prostate Cancer ◽  
Overall Survival ◽  
Lymph Node ◽  
Median Time ◽  
Median Number ◽  
Time To Progression ◽  
Chemohormonal Therapy ◽  
Radium 223 ◽  
Psa Response ◽  
Number Of Cycles

117 Background: Since docetaxel has been advanced to the metastatic castrate-sensitive prostate cancer (mCSPC) setting, there is a lack of evidence guiding its re-introduction upon castrate-resistant (CR) progression. We sought to identify clinical characteristics and outcomes of patients subjected to docetaxel rechallenge (DR) following prior docetaxel exposure in the mCSPC realm. Methods: Patients rechallenged with docetaxel following treatment in the mCSPC setting were identified from three academic centres in Ontario, Canada. Retrospective chart reviews were performed to identify clinical, treatment and outcome variables. Results: Of the 45 patients with DR initiated between 06/2015 and 07/2020, the median age was 65, 60% had a Gleason score of ≥8, and 64% had an ECOG of ≤1. 56% had bone only metastasis, 4% lymph node only metastasis, 29% bone and lymph node metastasis, and 11% had visceral metastasis. In the mCSPC setting, 98% of patients received 6 cycles of docetaxel with 13% requiring dose delays. Of 43 informative patients, all had a PSA response to chemohormonal therapy. 91% achieved at least a 50% PSA response (PSA50), of which 40% had a 50-89% PSA reduction and 51% had a ≥90% PSA reduction. 29% of patients obtained a PSA nadir of < 0.2 ng/mL. 16% had CR progression in < 6 months, 56% in 6-12 months, and 28% in > 12 months. DR was initiated after a median of 20.8 months (range 6.0-40.4) following the last dose of docetaxel for mCSPC, and was given as first line treatment for CR disease to 7%, second line to 51%, third line to 40%, and fourth line or beyond to 2% of patients. 69% of patients had received an androgen-receptor axis targeted therapy prior to DR, 18% radium 223, and 7% had received a trial drug. Notably, no patients had received cabazitaxel prior to DR. The median number of cycles of docetaxel received at rechallenge was 5 (range 1-11) with 18% of patients requiring treatment delays. 64% of patients stopped treatment due to progression, 16% due to side effects, 7% at the patient’s request, 7% due to completion of the planned number of cycles, and 6% due to death or other causes. Among 44 informative patients, 23% achieved at least a PSA50, with 18% having a 50-90% PSA reduction, and 5% having a ≥90% PSA reduction. The median time to progression (biochemical, radiographic, or death) was 2.3 months (95%CI 1.7-4.4) and the median overall survival was 11.0 months (95%CI 8.5-14.3). Conclusions: DR following exposure to docetaxel in the mCSPC setting resulted in a PSA50 in only around one quarter of patients. Both the median time to progression and overall survival were found to be short. With future investigations, we hope to identify clinical variables that will help predict which patients might benefit most from DR.

scholarly journals Real-world experience with Temozolomide & Sativex in patients with recurrent High Grade Gliomas

2019 ◽  
Vol 21 (Supplement_4) ◽  
pp. iv13-iv14
Author(s):  
Lillie Shahabi ◽  
Kerlann Le Calvez ◽  
Seema Dadhania ◽  
Catherine Blake ◽  
James Wang ◽  
...  
Keyword(s):  
Median Overall Survival ◽  
Progressive Disease ◽  
Phase 1 ◽  
Median Number ◽  
Phase 1 Study ◽  
Malignant Brain Tumour ◽  
Mri Scans ◽  
Recent Phase ◽  
Grade 3 ◽  
Number Of Cycles

Abstract Background Glioblastoma (GBM) is the most common malignant brain tumour in adults with a median survival from progression of 8 months. A recent phase 1 study of temozolomide (TMZ) and 1:1 CBD:THC (Sativex) offers evidence of efficacy in patients with recurrent GBM (NCT01812603). Methods All patients receiving TMZ & Sativex (75mg/m2 daily d1 – 21 q28; Sativex continuously) for relapsed GBM or grade 3 astrocytoma at our centre were identified. Patient, tumor and treatment characteristics were recorded, and response based on sequential MRI scans using modified RANO criteria assessed. Results 13 patients were treated over 18 months. The median age was 56; 69% were male. All had received initial chemo-radiotherapy (12 patients: 60 or 59.4Gy/30–33#; 1 patient: 45Gy/15#). 6 patients underwent reresection at recurrence. 4 patients were treated at first progression, 7 at second progression, and 2 at third or later progression. The median number of cycles of TMZ and sativex was 2. The combination treatment was tolerated well by all patients treated, with no Grade 3 or 4 toxicities, the only complaints being of discomfort in mouth after spray and ‘spaced out feeling’. Patients stopped treatment due to evidence of progressive disease on sequential MRI sign or physical deterioration. Median Overall Survival (OS) from initiation was 5.9 months (177 days); Progression Free Surival (PFS) at 3 months was 50%. Conclusion These results highlight some discrepancies in OS in comparison to the previous trial (NCT01812603), but our patients were treated at second/ third recurrence. We agree that the combination is well tolerated.

scholarly journals Phase I clinical trial of the combination of eribulin and everolimus in patients with metastatic triple-negative breast cancer

2019 ◽  
Vol 21 (1) ◽  
Author(s):  
Jin Sun Lee ◽  
Susan E. Yost ◽  
Suzette Blanchard ◽  
Daniel Schmolze ◽  
Hongwei Holly Yin ◽  
...  
Keyword(s):  
Breast Cancer ◽  
Phase I ◽  
Dose Level ◽  
Triple Negative Breast Cancer ◽  
Triple Negative ◽  
Median Number ◽  
Primary Objective ◽  
Best Response ◽  
Grade 3 ◽  
Number Of Cycles

Abstract Background Alteration of the PI3K/AKT/mTOR pathway is a common genomic abnormality detected in triple-negative breast cancer (TNBC). Everolimus acts synergistically with eribulin in TNBC cell lines and xenograft models. This phase I trial was designed to test the safety and tolerability of combining eribulin and everolimus in patients with metastatic TNBC. Methods The primary objective of this study was to evaluate the safety and toxicities of the combination. Patients with metastatic TNBC who had up to four lines of prior chemotherapies were enrolled. The combination of eribulin and everolimus was tested using three dosing levels: A1 (everolimus 5 mg daily; eribulin 1.4 mg/m2 days 1 and 8 every 3 weeks), A2 (everolimus 7.5 mg daily; eribulin 1.4 mg/m2, days 1 and 8 every 3 weeks), and B1 (everolimus 5 mg daily; eribulin 1.1 mg/m2 days 1 and 8 every 3 weeks). Results Twenty-seven patients with median age 55 years were enrolled. Among 8 evaluable patients who received dose level A1, 4 had dose-limiting toxicities (DLTs). Among 3 evaluable patients treated with dose level A2, 2 had DLTs. Among 12 evaluable patients who received dose level B1, 4 had DLTs. The DLTs were neutropenia, stomatitis, and hyperglycemia. Over the study period, 59% had a ≥ grade 3 toxicity, 44% had ≥ grade 3 hematologic toxicities, and 22% had grade 4 hematologic toxicities. The most common hematological toxicities were neutropenia, leukopenia, and lymphopenia. Thirty-three percent had grade 3 non-hematologic toxicities. The most common non-hematological toxicities were stomatitis, hyperglycemia, and fatigue. The median number of cycles completed was 4 (range 0–8). Among 25 eligible patients, 9 patients (36%) achieved the best response as partial response, 9 (36%) had stable disease, and 7 (28%) had progression. The median time to progression was 2.6 months (95% CI [2.1, 4.0]), and median overall survival (OS) was 8.3 months (95% CI [5.5, undefined]). Conclusion Eribulin 1.1 mg/m2 days 1 and 8 every 3 weeks with everolimus 5 mg daily was defined as the highest dose with acceptable toxicity (RP2D). The combination is safe, and efficacy is modest. A post hoc analysis showed that participants that used dexamethasone mouthwash stayed on treatment for one additional cycle. Trial registration ClinicalTrials.gov, NCT02120469. Registered 18 April 2014

scholarly journals The Efficacy of Cladribine (2-CdA) in Advanced Systemic Mastocytosis

2020 ◽  
Vol 36 (4) ◽  
pp. 661-666 ◽  
Author(s):  
Grzegorz Helbig ◽  
Anna Koclęga ◽  
Władysław B. Gaweł ◽  
Martyna Włodarczyk ◽  
Marek Rodzaj ◽  
...  
Keyword(s):  
Overall Response Rate ◽  
Response Rate ◽  
Systemic Mastocytosis ◽  
Median Number ◽  
Hemoglobin Level ◽  
Median Dose ◽  
Skeletal Involvement ◽  
Duration Of Response ◽  
Hematological Neoplasm ◽  
Number Of Cycles

Abstract Systemic mastocytosis (SM) is a rare clonal disorder with multi-organ involvements and shortened life expectancy. To date, no curative treatment for SM exists. Cladribine (2-CdA) is a purine analogue showing activity against neoplastic mast cells and its use was found to be effective in some patients with SM. Nine patients (six males and three females) with advanced SM at median age of 63 years (range 33–67) who received at least one course of 2-CdA were included in a retrospective analysis. Study patients were classified as having aggressive SM (ASM; n = 7) and SM with an associated hematological neoplasm (SM-AHN; n = 2). The “C” findings were as follows: (1) absolute neutrophil count (ANC) < 1 × 109/l (n = 1) and/or hemoglobin level < 10 g/dl (n = 4) and/or platelet count < 100 × 109/l (n = 4); (2) hepatomegaly with ascites (n = 4); (3) skeletal involvement (n = 2); (4) palpable splenomegaly with hypersplenism (n = 3) and (5) malabsorption with weight loss (n = 5). Treatment consisted of 2-CdA at dose 0.14 mg/kg/day intravenously over a 2-h infusion for 5 consecutive days. Median dose per cycle was 45 mg (range 35–60). Median number of cycles was 6 (range 1–7). Overall response rate (ORR) was 66% (6/9 pts) including three partial responses and three clinical improvements. ORR was 100% and 66% for SM-AHN and ASM, respectively. Median duration of response was 1.98 years (range 0.2–11.2). At the last contact, five patients died, four have little disease activity, but remain treatment- free. 2-CdA seems to be beneficial in some patients with SM, however the response is incomplete.

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