scholarly journals Newly Developed Psoriasis in a Patient with Telangiectasia Macularis Eruptiva Perstans and Systemic Mastocytosis Treated with Interferon

2021 ◽  
pp. 558-562
Author(s):  
Tatiana Péčová ◽  
Tatiana Burjanivová ◽  
Bibiana Malicherová ◽  
Martin Jozef Péč ◽  
Igor Rohoň ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Mast Cell ◽  
Interferon Alpha ◽  
Rare Case ◽  
Systemic Treatment ◽  
Systemic Mastocytosis ◽  
Skin Lesions ◽  
Line Treatment ◽  
First Line ◽  
First Line Treatment

The authors present a rare case of a patient with telangiectasia macular eruptiva perstans, with confirmed D816V mutation which later progressed to systemic mastocytosis confirmed by trepanobiopsy. First-line treatment – phototherapy – had to be stopped, and systemic treatment with interferon alpha-2a was initiated. The treatment was successful with regression of skin lesions as well as mast cell infiltrates in the bone marrow. However, the treatment was complicated by the onset of psoriasis lesions.

Efficacy of Rituximab in Hairy Cell Leukemia.

Blood ◽  
2006 ◽  
Vol 108 (11) ◽  
pp. 4719-4719
Author(s):  
Styliani I. Kokoris ◽  
Maria K. Angelopoulou ◽  
Zacharoula I. Galani ◽  
Konstantinos Anargyrou ◽  
Sotirios Sachanas ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Interferon Alpha ◽  
Hairy Cell Leukemia ◽  
Response Rate ◽  
Costal Margin ◽  
Line Treatment ◽  
Hairy Cell ◽  
Cell Leukemia ◽  
First Line ◽  
First Line Treatment

Abstract BACKGROUND: Hairy Cell Leukemia (HCL) is a rare B-chronic lymphoproliferative disorder (BCLD) with an indolent course. First-line treatment modalities include 2-chlorodeoxyadenosine (2-CDA), 2-deoxycoformycin (2-DCF) and interferon-alpha. The efficacy of anti-CD20-Rituximab (R) in other BCLDs, as well as strong CD20 expression by HCL cells, indicate that R could be an alternative treatment of HCL. AIMS: The experience of a single Hematology Unit in the treatment of relapsed HCL with R. PATIENTS AND METHODS: We retrospectively analyzed all HCL patients (pts) who received R as salvage therapy in 1st or subsequent relapse. RESULTS: 13 patients treated with R were located among 110 patients diagnosed with HCL between 1980 and 2005. 11 were males and their median age before R treatment was 46 years (range: 42–88). 5 pts had splenomegaly with a median spleen size of 7cm below left costal margin (range:5–20cm). 3 pts has an absolute neutrophil count<1.5×109/L, 4 a hemoglobin <10g/dL and 5pts a platelet count<100×109/L. All patients displayed a typical immunophenotype from blood and/or bone marrow (CD20 strongly +, CD19+, CD22+, FMC-7+, CD11c+, CD25+, CD103+). Four of them were CD23+ and two CD10+. 8 pts received Rituximab at 1st relapse. Among them, one had received 2-DCF as first-line treatment, one 2-CDA and 6 interferon-alpha as induction and maintenance. 3 pts had received more than one prior treatments. Two pts received R at diagnosis, due to older age. The median time from diagnosis to R initiation was 61 months (range: 4–275). R was administered at 375mg/m2 weekly for 6 cycles. Overall response rate was 67%, with 4 pts showing a negative immunophenotype. One pt discontinued treatment after the first cycle due to the development of thrombocytopenia that was attributed to the drug. 7/8 responders showed a complete restoration of their cytopenias. No other complications were recorded, except of mild infusion-related symptoms. Among the responding pts, none has relapsed so far with a median follow-up of 14 months (range: 4–40+). Among partial responders, one achieved a complete response including a negative bone marrow and immunophenotype after R retreatment. CONCLUSIONS: R is a highly effective and tolerable treatment for HCL in relapse with a response rate of 67%. Retreatment or maintenance with R may be important, since ongoing responses are seen.

scholarly journals Severe aplastic anemia: allogeneic bone marrow transplantation as first-line treatment

2018 ◽  
Vol 2 (15) ◽  
pp. 2020-2028 ◽  
Author(s):  
George E. Georges ◽  
Kris Doney ◽  
Rainer Storb
Keyword(s):  
Bone Marrow ◽  
Aplastic Anemia ◽  
Marrow Transplantation ◽  
Severe Aplastic Anemia ◽  
Unrelated Donor ◽  
Line Treatment ◽  
First Line ◽  
Matched Unrelated Donor ◽  
First Line Treatment

Abstract Treatment of severe aplastic anemia has improved significantly over the past 4 decades. This review will summarize the key areas of progress in the use of allogeneic hematopoietic cell transplantation and nontransplant immunosuppressive therapy (IST) for the treatment of aplastic anemia and then summarize the recommendations for first-line treatment. Based on recent data, we argue that guidelines for the initial treatment of patients with newly diagnosed severe aplastic anemia require revision. At the time of diagnosis, before beginning treatment, HLA typing should be done to identify a marrow donor among family members or in the unrelated donor registries, and a marrow transplant should be considered first-line therapy. The priority order of donor source for bone marrow transplantation is: (1) HLA-identical sibling, (2) HLA-matched unrelated donor, and (3) HLA-haploidentical donor if an HLA-matched unrelated donor is not rapidly available. Each of these donor marrow sources may be preferable to nontransplant IST. We make this recommendation because of the long-term persistent risk for disease relapse and secondary myelodysplastic syndrome or acute myeloid leukemia with the use of nontransplant IST for patients with aplastic anemia. In contrast, marrow transplantation is associated with high cure rates of aplastic anemia and a relatively low risk for graft-versus-host disease, with many patients now living for decades without the risk for disease recurrence or the development of clonal disorders. Implementation of this first-line treatment strategy will provide patients with severe aplastic anemia the best chance of long-term disease-free survival.

Imatinib Mesylate in the Treatment of Systemic Mastocytosis, a Phase I/II Trial.

Blood ◽  
2004 ◽  
Vol 104 (11) ◽  
pp. 1516-1516 ◽  
Author(s):  
H.J. Droogendijk ◽  
J.C. Kluin-Nelemans ◽  
P.L.A van Daele
Keyword(s):  
Bone Marrow ◽  
Cell Proliferation ◽  
Mast Cell ◽  
Mast Cells ◽  
Imatinib Mesylate ◽  
Systemic Mastocytosis ◽  
Skin Lesions ◽  
Tissue Response ◽  
Serum Tryptase ◽  
Kit Receptor

Abstract Introduction: mastocytosis comprimes a group of diseases characterized by abnormal proliferation and accumulation of mast cells in one or more organs. A cutaneous and systemic form of mastocytosis is distinguished. Systemic mastocytosis defines the disease process in which mast cell proliferation exceeds the skin. The clinical manifestations of systemic mastocytosis depend on the tissues involved and the tissue response to the accumulation of mast cells. Although in general the disease progresses slowly, it may develop into a malignant disease. Currently there is no cure for systemic mastocytosis. Mast cells develop from pluripotent bone marrow progenitor cells that express CD34 antigen and are dispersed as precursors which undergo proliferation and maturation in different tissues. Normal mast cell development involves the action of stam cell growth factor and c-kit receptors, which are expressed by mast cells at their different developmental stages. Deregulation and/or abnormalities of the c-kit receptor are assumed to play a causal role in disordered mast-cell proliferation. In most patients a mutation in the gene for c-kit exists. One of the mutations is the D816V mutation. Aim of the study:imatinib mesylate, formerly called ST1571, is a potent inhibitor of c-kit receptor tyrosine kinase activity. In this study, we evaluate whether imatinib mesylate is safe and effective in the treatment of patients with systemic mastocytosis. Primary end-points of study are reduction in urinary N-methylhistamine excretion, serum tryptase activity, skin lesions, number of mast cells in sections of bone marrow, hepato-and/or splenomegaly and symptoms.Adverse effects on therapy are also considered. Results: up to now, 10 patients with systemic mastocytosis are treated with 400 mg of imatinib mesylate orally once daily. During the first 2 weeks of the study the patients also received 30 mg of prednisolone daily. In general imatinib mesylate is well tolerated. The first results show a 38–80% reduction in urinary N-methylhistamine excretion and 30–66% reduction in serum tryptase activity. Skin lesions diminish in two of the six patients with cutaneous mastocytosis,. Number of mast cells in sections of bone marrow are reduced in 63% (5/8) of the patients. Hepato-and/or splenomegaly is slightly decreased in two of the three patients with organomegaly. Finally 60 % of all patients experiences relief of symptoms. In eight patients the D816V mutation was found. In contrast with former studies imatinib mesylate is also effective in these patients. Further results are to be awaited. Conclusion: imatinib mesylate is safe and seems effective in the treatment of patients with systemic mastocytosis (including patients with the D816V mutation).

Outcomes in Children with Severe Aplastic Anemia Receiving Bone Marrow Transplantation from an HLA-Matched Family Donor or Intensive Immunosuppressive Therapy As First-Line Treatment,

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 3421-3421 ◽  
Author(s):  
Nao Yoshida ◽  
Akira Kikuchi ◽  
Ryoji Kobayashi ◽  
Hiromasa Yabe ◽  
Yoshiyuki Kosaka ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Bone Marrow Transplantation ◽  
Aplastic Anemia ◽  
Immunosuppressive Therapy ◽  
Cell Transplantation ◽  
Marrow Transplantation ◽  
Line Treatment ◽  
First Line ◽  
Family Donor ◽  
First Line Treatment

Abstract Abstract 3421 Bone marrow transplantation (BMT) from an HLA-matched family donor (MFD) is the treatment of choice for severe aplastic anemia (SAA) in children. For children without an MFD, immunosuppressive therapy (IST) with a combination of antithymocyte globulin and cyclosporine has been successful. However, this treatment approach is based on the results of comparative studies between these therapies conducted in the 1980s, and the outcomes of both BMT and IST have improved markedly over the past three decades. Therefore, updated evidence for treatment decisions in pediatric SAA is required. In the present study, we compared the outcomes of children with SAA who received IST (subjects enrolled in the prospective multicenter trials of IST conducted by the Japan Childhood Aplastic Anemia Study Group) or BMT from an MFD (subjects registered in the Transplant Registry Unified Management Program conducted by the Japan Society for Hematopoietic Cell Transplantation). The influence of potential risk factors on overall survival (OS) and failure-free survival (FFS) was assessed according to first-line treatment, time period of treatment (1992–1999 and 2000–2009), age and other variables related to each treatment. FFS was defined as survival with treatment response. Death, primary or secondary graft failure, relapse and secondary malignancy were considered treatment failures in patients who received BMT. Death, relapse, disease progression requiring stem cell transplantation from an alternative donor or 2nd IST, clonal evolution and evolution to paroxysmal nocturnal hemoglobinuria were considered treatment failures in patients who received IST. Between 1992 and 2009, 599 children with SAA younger than 17 years received BMT from an MFD (n=213) or IST (n=386) as first-line treatment. While the OS did not differ between patients receiving IST and BMT (88±2% vs. 90±2% at 15 years), FFS was significantly inferior in patients receiving IST as compared to those receiving BMT (54±3% vs. 84±3% at 15 years, P<0.0001). There was no significant improvement in outcomes over the two time periods; OS and FFS at 10 years in 1992–1999 vs. 2000–2009 were 87±2% vs. 93±2% and 66±3% vs. 67±3%, respectively. On multivariate analysis, age <10 years was identified as a favorable factor for OS (P=0.007) and choice of first-line IST was the only unfavorable factor for FFS (P<0.0001). These updated data support the current algorithm for treatment decisions, which recommends BMT when an MFD is available. Disclosures: No relevant conflicts of interest to declare.

Role of an EpCAM auto-antibody in ovarian cancer patients with special regard to disseminated tumor cells in bone marrow

2009 ◽  
Vol 27 (15_suppl) ◽  
pp. e16557-e16557
Author(s):  
M. Heubner ◽  
S. Kasimir-Bauer ◽  
D. Errico ◽  
D. Herlyn ◽  
R. Kimmig ◽  
...  
Keyword(s):  
Ovarian Cancer ◽  
Bone Marrow ◽  
Cancer Patients ◽  
Tumor Cells ◽  
Disseminated Tumor Cells ◽  
Healthy Controls ◽  
Line Treatment ◽  
First Line ◽  
Platinum Based Chemotherapy ◽  
First Line Treatment

e16557 Background: EpCAM is a tumor associated antigen which is frequently expressed in ovarian cancer. Recently, an autoantibody (AAB) against EpCAM has been identified in ovarian cancer patients. Autoantibodies are immunogene factors and might be of prognostic importance. We showed that disseminated tumor cells (DTC) in bone marrow carry the EpCAM antigen on their surface and correlate with poorer progression free survival (PFS). Here, we evaluated whether EpCAM-AABs have an impact on clinical parameters or the presence of DTC in ovarian cancer patients. Methods: EpCAM-AABs were determined in sera of 28 healthy voluntary age matched females and 62 patients with primary epithelial ovarian cancer before and after platinum-based chemotherapy using a recombinant EpCAM-epitope for antibody- detection by ELISA technique. Mean follow up time was 13 months. DTC in BM were detected by immunocytochemistry applying the pan cytokeratin antibody A45-B/B3. All samples exceeding the mean antibody titer of healthy controls plus 2 standard deviations were considered positive. Results: The antibody titer of healthy controls was 0.061 + 0.015. Using a cut-off value of 0.091, we found 9/62 (15%) ovarian cancer patients to be positive for EpCAM-AABs after first-line treatment. Interestingly, no positive AAB-titers were seen before therapy. Using the paired T-Test, we noted a significant posttherapeutic increase of AABs (CI 0.95, p < 0.0001). Analysis of PFS, FIGO stage, resection status, grading, age, sensitivity to platinum based chemotherapy and DTC did not reveal significant associations with positive EpCAM-AAB titers. Conclusions: The clinical course of ovarian cancer patients and the prevalence of DTC were not altered by EpCAM-AABs. Interestingly, we observed an increase of antibody-levels after first-line treatment. For further validation, we intend to extend our patient collective. In future, it might also be interesting to investigate the impact of AABs on response to targeted therapies against EpCAM. No significant financial relationships to disclose.

Predictors of first line systemic therapy for metastatic renal cell carcinoma in IMDC favorable risk patients.

2020 ◽  
Vol 38 (15_suppl) ◽  
pp. e17086-e17086
Author(s):  
Ryuichi Mizuno ◽  
Kimiharu Takamatsu ◽  
Nobuyuki Tanaka ◽  
Nozomi Hayakawa ◽  
Takeo Kosaka ◽  
...  
Keyword(s):  
Systemic Therapy ◽  
Systemic Treatment ◽  
Risk Group ◽  
Risk Category ◽  
Line Treatment ◽  
First Line ◽  
Baseline Serum ◽  
Female Age ◽  
Serum Crp ◽  
First Line Treatment

e17086 Background: With the induction of molecular targeted agents, which interfere with proteins that play critical roles in tumor growth and progression, and immune checkpoint inhibitors (ICI), the scenario of systemic treatment of metastatic renal cell carcinoma (mRCC) have dramatically been changed with improved survival. Prognostic risk assessment is essential for choosing the most appropriate first line treatment option, with selection based on International Metastatic RCC Database Consortium (IMDC) Risk Category. This study was designed to evaluate the actual efficacy of systemic therapy in the IMDC favorable risk group patients with mRCC. Methods: A total of 218 patients with mRCC who received systemic therapy were retrospectively reviewed (Institutional review board approval No 2013-0425). Among them, 55 (25%) patients were classified as favorable risk group based on the IMDC Risk Category. These mRCC patients in the favorable risk were divided into 2 groups by following factors; gender (male or female), age (≥70, or not), baseline serum CRP levels (≥0.6, and < 0.6 mg/dl), metastatic sites (exclusive lung, or lung and others), BMI (≥22, or < 22), respectively. The overall survival (OS) of patients in the favorable risk group were compared between the two cohorts. Results: The median PFS for first line treatment and OS of patients in favorable risk group were 31.6 (95% CI 19.9–33.7)) and 85.8 (56.8-NE) months, respectively. The median OS in patients with BMI≥22 and < 22 was 65.6 (95% CI 56.8–NE) and 56.8 (95% CI 30.8–NE) months, respectively (p = 0.0497). The median OS in patients with exclusive lung metastasis, and lung and other metastases were 115.1 (95% CI NE) and 65.6 (95% CI 43.9–NE) months, respectively (p = 0.0073). No significant difference was found in OS between male and female, age≥70 and < 70, and baseline serum CRP levels ≥0.6 mg/dl and < 0.6, respectively. Conclusions: The first line systemic treatment for mRCC patients in IMDC favorable risk group was feasible and effective in patients with BMI≥22 or exclusive lung metastasis.

A nationwide population-based study comparing survival in unresectable advanced or synchronous metastatic esophageal and gastric adenocarcinoma.

2020 ◽  
Vol 38 (4_suppl) ◽  
pp. 308-308
Author(s):  
Marieke Pape ◽  
Pauline A.J. Vissers ◽  
David Bertwistle ◽  
Laura McDonald ◽  
Hanneke W.M. Van Laarhoven ◽  
...  
Keyword(s):  
Systemic Treatment ◽  
Rank Test ◽  
Tumor Characteristics ◽  
Line Treatment ◽  
First Line ◽  
Treatment Regimens ◽  
Netherlands Cancer Registry ◽  
Number Of Patients ◽  
Significant Difference ◽  
First Line Treatment

308 Background: Similarities between esophageal and gastric adenocarcinomas have been identified in terms of genomic characteristics. There is however no consensus on the combined or stratified inclusion of esophageal adenocarcinoma (EAC) within gastric cancer (GC) clinical trials. The aim of our study was to compare patient and tumor characteristics, first-line treatment regimens and overall survival (OS) of patients with EAC and GC. Methods: We selected patients with unresectable advanced and/or synchronous metastatic EAC (n = 1554) or GC (including junction tumors; n = 2095) diagnosed in the period 2015-2017 from the nationwide Netherlands Cancer Registry. Patients with a positive HER2 test result and/or receiving trastuzumab as a first-line treatment were excluded. Data on OS were analyzed using Kaplan-Meier curves with Log-Rank test. Results: The EAC patient population had significantly more male patients (83% vs 66%), lower median age (68 vs 71 years) and higher median BMI (25.4 vs 24.5). Significant differences in location of metastases were identified, with higher percentages in non-regional lymph nodes (48% vs 28%), liver (50% vs 35%), lung (21% vs 9%) and bone (19% vs 7%) and lower in peritoneum (5% vs 42%), in EAC versus GC patients respectively. EAC patients more often received any type (supportive or active systemic) of treatment (76% vs 60%). Median OS was longer in EAC than GC patients (EAC: 4.8 vs GC: 4.1 months; p < 0.01). The percentages of patients receiving first-line systemic treatment were equal in both groups (43%). The number of patients receiving CapOx or FOLFOX was not significantly different (43% vs 47%). Carboplatin+paclitaxel was more frequently given in EAC versus GC (34% vs 3%), while EOX or ECC was given less frequently (12% vs 30%). No significant difference was observed in median OS between EAC and GC patients receiving first-line active systemic treatment (8.0 vs 7.6 months; p = 0.28). Conclusions: Patient characteristics, tumor characteristics, treatment regimens and OS differ between EAC and GC patients. Despite these differences, in patients receiving first-line active systemic treatment no significant differences in OS were found.

Phase II trial of subcutaneous anti-CD52 monoclonal antibody alemtuzumab (Campath-1H) as first-line treatment for patients with B-cell chronic lymphocytic leukemia (B-CLL)

Blood ◽  
2002 ◽  
Vol 100 (3) ◽  
pp. 768-773 ◽  
Author(s):  
Jeanette Lundin ◽  
Eva Kimby ◽  
Magnus Björkholm ◽  
Per-Anders Broliden ◽  
Fredrik Celsing ◽  
...  
Keyword(s):  
Monoclonal Antibody ◽  
Bone Marrow ◽  
Chronic Lymphocytic Leukemia ◽  
B Cell ◽  
Lymphocytic Leukemia ◽  
Prolonged Treatment ◽  
Line Treatment ◽  
First Line ◽  
Cell Chronic Lymphocytic Leukemia ◽  
First Line Treatment

Abstract This phase II study determined the efficacy and safety of alemtuzumab, a humanized anti-CD52 monoclonal antibody, delivered subcutaneously as first-line therapy, over a prolonged treatment period of 18 weeks in 41 patients with symptomatic B-cell chronic lymphocytic leukemia (B-CLL). Injections were administered subcutaneously 3 times per week, from week 2 to 3 onward. An overall response rate (OR) of 87% (95% CI, 76%-98%; complete remission [CR], 19%; partial remission [PR], 68%) was achieved in 38 evaluable patients (81% of intent-to-treat population). CLL cells were cleared from blood in 95% patients in a median time of 21 days. CR or nodular PR in the bone marrow was achieved in 66% of the patients and most patients achieved this after 18 weeks of treatment. An 87% OR (29% CR) was achieved in the lymph nodes. The median time to treatment failure has not yet been reached (18+ months; range, 8-44+ months). Transient injection site skin reactions were seen in 90% of patients. Rigor, rash, nausea, dyspnea, and hypotension were rare or absent. Transient grade IV neutropenia developed in 21% of the patients. Infections were rare, but 10% patients developed cytomegalovirus (CMV) reactivation. These patients rapidly responded to intravenous ganciclovir. One patient, allergic to cotrimoxazole prophylaxis, developedPneumocystis carinii pneumonia. Alemtuzumab is highly effective as first-line treatment in patients with B-CLL. Prolonged treatment is important for maximal bone marrow response. Subcutaneous administration induced very few “first-dose” flulike symptoms and may reduce health care costs in comparison with the intravenous infusions.

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