Use of high-dose oral bisphosphonate therapy for symptomatic fibrous dysplasia of the skull

2008 ◽  
Vol 109 (5) ◽  
pp. 889-892 ◽  
Author(s):  
Kevin Chao ◽  
Laurence Katznelson
Keyword(s):  
Fibrous Dysplasia ◽  
Therapeutic Option ◽  
Skeletal Development ◽  
Bisphosphonate Therapy ◽  
Fracture Repair ◽  
High Dose ◽  
Oral Bisphosphonate ◽  
Dose Oral ◽  
Insight Into

Fibrous dysplasia of the bone in adults is a rare anomaly of skeletal development caused by a defect in differentiation of osteoblasts. This condition is associated with bone pain, bone deformity, and an increased incidence of fracture. Involvement of the skull is associated with headache along with dysmorphic features. Until recently, the principal treatment has been resection or fracture repair, although the latter is often palliative at best. However, new insight into the molecular mechanism of fibrous dysplasia has led to the use of bisphosphonates to treat this disease. The authors examined the effects of high-dose oral alendronate (40 mg daily) for 6 months on 3 adult patients with intractable headache due to fibrous dysplasia of the skull. Each patient had disease processes not amenable to surgery. The patients underwent clinical follow-up at 1, 3, and 6 months. Their pain levels were documented at each visit by using a visual analog scale. All 3 patients demonstrated a significant decrease in pain levels and became independent of scheduled analgesics. Tumor bulk did not progress during this interval in any patient. Overall, alendronate was tolerated well, although in 1 patient it was discontinued early due to esophagitis. High-dose oral bisphosphonate therapy is an alternative therapeutic option for the palliative treatment of patients with fibrous dysplasia of the skull.

Phase II, randomized, double blind, placebo-controlled study comparing siltuximab plus bortezomib versus bortezomib alone in pts with relapsed/refractory multiple myeloma.

2012 ◽  
Vol 30 (15_suppl) ◽  
pp. 8018-8018 ◽  
Author(s):  
Robert Z. Orlowski ◽  
Liana Gercheva ◽  
Cathy Williams ◽  
Heather J Sutherland ◽  
Tadeusz Robak ◽  
...  
Keyword(s):  
Multiple Myeloma ◽  
Overall Response Rate ◽  
Randomized Study ◽  
Controlled Study ◽  
High Dose ◽  
Double Blind ◽  
Disease Characteristics ◽  
Dose Oral ◽  
Secondary Endpoints

8018^ Background: Preclinical studies of siltuximab (S), a chimeric anti-IL-6 mAb, in combination with bortezomib (B) indicate an additive to synergistic effect in multiple myeloma (MM) cell lines. This randomized study evaluated the safety and efficacy of S+B compared with placebo (plc)+B in pts with relapsed/refractory MM after 1−3 prior tx lines, measurable disease but no prior B exposure. Methods: 286 pts were randomized 1:1 to S+B: B+plc. S 6 mg/kg or plc was given IV q2w. B 1.3 mg/m2 was given IV on d 1, 4, 8, 11, 22, 25, 29, 32 for a max of 4 of 42-d cycles and then reduced to q1w for 35-d cycles. B was stopped for pts with PD/intolerability, and high dose oral dexamethasone (dex) 40 mg could then be started qd on d 1−4, 9−12, 17−20 for a max of 4 of 28-d cycles and on d 1−4 of subsequent cycles until PD, while S/plc continued. Primary endpoint was PFS by EBMT criteria censored at the start of dex/subsequent tx. Secondary endpoints included overall response rate (ORR), OS, and safety before dex. Results: 142 and 144 pts received S+B and B+plc, respectively. Baseline demographics and disease characteristics were well balanced across S+B and B+plc, except for age (median 64 vs. 61 yrs) and myeloma type (IgG 65 vs. 71%, IgA 27 vs. 20%). Median tx duration was 5.1 mo in both grps. Median PFS was 8.1 mo in S+B and 7.6 mo in B+plc (HR 0.869, p = 0.345). ORR (CR+PR) was 55% in pts on S+B and 47% on B+plc (p = 0.213); CR rates were 11 and 7% (p = 0.342), respectively. With 24.5 mo median follow up, median OS was 30.8 mo for S+B and 36.9 mo for B+plc (HR 1.353 for S+B, p = 0.103). Fewer pts on S+B than B+plc moved to dex (23 vs. 31%) and had subsequent SCT (5 vs. 11%). Gr ≥3 AEs occurred in 91% on S+B and 74% on B+plc. Common gr ≥3 AEs in S+B were neutropenia (49%), thrombocytopenia (48%), leukopenia (14%). SAEs occurred in 29% on S+B and 24% on B+plc. Death occurred within 30 d of last study agent administration pre-dex in 8% on S+B and 5% on B+plc. Conclusions: The combination of S+B had higher response rates but did not prolong survival compared with B+plc. A negative survival trend heavily influenced by differences in dex and SCT rescue was noted. S+B appears to be generally well tolerated but had a higher incidence of hematologic AEs.

scholarly journals Interleukin-2 may induce prolonged remissions in advanced acute myelogenous leukemia

Blood ◽  
1994 ◽  
Vol 84 (7) ◽  
pp. 2158-2163 ◽  
Author(s):  
G Meloni ◽  
R Foa ◽  
M Vignetti ◽  
A Guarini ◽  
S Fenu ◽  
...  
Keyword(s):  
Acute Myelogenous Leukemia ◽  
Residual Disease ◽  
Therapeutic Option ◽  
Interleukin 2 ◽  
Myelogenous Leukemia ◽  
High Dose ◽  
Antileukemic Effect ◽  
Acute Myelogenous ◽  
History Of

Abstract The administration of interleukin-2 (IL-2) may induce complete remissions in acute myelogenous leukemia (AML) patients with a low proportion of residual bone marrow (BM) blasts. To confirm this preliminary observation, we treated 14 AML patients with advanced disease and with a residual BM blastosis that ranged between 7% and 24% with repeated 5-day cycles of high-dose recombinant IL-2 administered by daily continuous intravenous infusion. Patients who responded have been subsequently submitted to a monthly maintenance scheme with subcutaneous IL-2 at lower doses. While using this schedule and closely monitoring clinical and laboratory conditions, side effects were acceptable and no toxic deaths recorded. Eight of the 14 patients treated with high-dose IL-2 obtained a complete remission (CR). Five remain in persistent CR (four in third CR and one in fourth CR) after a median follow-up time of 32 months (14, 30, 32, 33, and 68 months, respectively). In all five patients, the IL-2-induced remission is the longest in the natural history of the disease. These findings show that IL-2 displays an antileukemic effect in AML with limited residual disease, and suggest that IL-2 should be considered a therapeutic option for resistant or relapsed AML patients.

Thalidomide Based Regimens for Treatment of Unfit Patients with Relapsed and Refractory Multiple Myeloma: a Monocentric Experience

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 5048-5048
Author(s):  
Alessandro Moscetti ◽  
Francesca Saltarelli ◽  
Maria Paola Bianchi ◽  
Bruno Monarca ◽  
Giacinto La Verde
Keyword(s):  
Multiple Myeloma ◽  
Side Effects ◽  
Response Rate ◽  
Conflicts Of Interest ◽  
Therapeutic Option ◽  
Alkylating Agents ◽  
High Dose ◽  
Refractory Multiple Myeloma ◽  
Unfit Patients

Abstract Abstract 5048 Thalidomide, an immunomodulating drug with antiangiogenic activity, is an efficacious therapeutic option for unfit patients with multiple myeloma. Its efficacy may be increased by the addiction of steroids or other cytotoxic drugs such melphalan or cyclophosphamide. In this study we assessed the efficacy and toxicity of thalidomide based regimens as savage therapy in a series of elderly patients with relapsed/refractory multiple myeloma. Previous treatments included at least one therapy (range 1–4), such as high dose dexamethasone, alkylating agents, anthracyclines, IFN-α and autologous graft. Thalidomide 50–200 mg/die was administered orally in a total of 16 patients (median age 73.8 years, range 59–84) with relapsed/refractory multiple myeloma observed in our Hematology Department between May 2004 and January 2010. Oral dexamethasone or prednisone was added to the treatment. All patients continued therapy until relapse or progression and were prospectively followed-up including accurate monitoring of side effects. Response to thalidomide was assessed according to the European Group for Blood and Marrow Transplantation criteria. The median follow-up time was 25.6 months (range 8 – 68). Overall response rate was 81.2% (13/16 patients) with a median duration of response of 26.7 months (range 7 – 67): 3 patients showed a very good partial remission, 10 partial response, 1 stable disease and 2 progression of disease. During follow-up, 6 patients died (3 due to progression, 2 due to other neoplasm, 1 due to heart failure), 10 patients are still alive (2 VGPR and 7 PR in continuous therapy, 1 PD in third line therapy). No response was observed in 3/16 patients (18.7%). Despite the following side effects, mild to moderate bradycardia (25%, 1 needed PMK positioning and 1 dose reduction), peripheral sensitive polyneuropathy (18.7%) and constipation (6%), no patient discontinued therapy. This study shows that thalidomide based regimens are an effective therapy with a high response rate and manageable side effects when used in patient with multiple myeloma with relapsed/refractory disease. Disclosures: No relevant conflicts of interest to declare.

scholarly journals Globe Subluxation following Long-Term High-Dose Steroid Treatment for Myasthenia Gravis

2020 ◽  
Vol 11 (3) ◽  
pp. 534-539 ◽  
Author(s):  
Jasmien Dam ◽  
Florit Marcuse ◽  
Marc De Baets ◽  
Catherine Cassiman
Keyword(s):  
Side Effects ◽  
Myasthenia Gravis ◽  
Term Treatment ◽  
Steroid Treatment ◽  
Oral Steroid ◽  
High Dose ◽  
Long Term Treatment ◽  
Dose Oral

This case report describes the unusual presentation of a globe subluxation following long-term high-dose oral steroid treatment for myasthenia gravis (MG). The patient presented initially with fluctuating vertical diplopia. Auto-antibodies against the acetylcholine receptor were weakly positive, confirming the diagnosis of MG. After initial treatment with pyridostigmine, the disease evolved to generalized MG. Plasmapheresis and high-dose steroids were started subsequently. As a side effect of this treatment the patient gained about 30 kg in weight and developed steroid myopathy and a prominent cushingoid facies with bilateral exophthalmos. A year after his initial diagnosis he experienced a spontaneous globe subluxation on the left eye. He was able to immediately reposition the globe manually himself. Four months later a new subluxation occurred. Because of these aforementioned severe side effects of the steroid treatment, the methylprednisolone was tapered and replaced by tacrolimus. After about 6 weeks the patient went into remission. We believe, that the spontaneous globe subluxations were caused by a weakness of the extraocular muscles in combination with a significant gain of intraorbital fat tissue, both induced by cumulative, excessive steroids. Steroids are often necessary in the treatment of MG; however, most of the time a high dose of 64 mg is not needed for ocular MG and especially the continuation of a dose of 58 mg or more for a long period is not recommended. Careful follow-up is obligatory to timely recognize side effects. In case of severe side effects or the need for long-term treatment, the use of other immunosuppressive therapies should be considered. Extra care and caution is recommended in patients who are anatomically predisposed with proptosis.

scholarly journals Organized Hematoma of the Sphenoid Sinus With Acute Blindness: Insight Into Pathogenesis of Disease

2020 ◽  
Vol 99 (9) ◽  
pp. 605-609
Author(s):  
Giant C. Lin ◽  
Jade Wells Porrmann ◽  
Melody Paz ◽  
Yaron A. Moshel ◽  
Jeffrey LeBenger ◽  
...  
Keyword(s):  
Maxillary Sinus ◽  
Fibrous Dysplasia ◽  
Sphenoid Sinus ◽  
Polyostotic Fibrous Dysplasia ◽  
Loss Of Vision ◽  
Sphenoid Sinusitis ◽  
Rate Of Growth ◽  
Insight Into

Sinonasal organized hematomas (OHs) are rare lesions that primarily localize to the maxillary sinus. The rate of growth of these masses has not been described in the literature. We present a case of a 59-year-old gentleman with polyostotic fibrous dysplasia who presented with acute loss of vision in the left eye from an expanding OH of the sphenoid sinusitis. After expanded endonasal, transpterygoid approach and debulking, patient experienced significant vision improvement. Close follow-up imaging preoperatively allowed radiologic documentation of the rate of OH growth and this is presented in detail.

scholarly journals Possible Role of Oral Ibandronate Administration in Osteonecrosis of the Jaw: A Case Report

2012 ◽  
Vol 25 (1) ◽  
pp. 311-316 ◽  
Author(s):  
A. Notarnicola ◽  
S. Lisi ◽  
M. Sisto ◽  
A.V. De Marino ◽  
M. D'Amore
Keyword(s):  
Risk Factors ◽  
Osteonecrosis Of The Jaw ◽  
Bisphosphonate Therapy ◽  
Caucasian Woman ◽  
Oral Bisphosphonate ◽  
Antiresorptive Drugs ◽  
Magnetic Resonance Imaging Mri ◽  
Alternative Medications

We describe a case of Osteonecrosis of the Jaw (ONJ) that developed in a 65-year-old Caucasian woman with osteopenia and other risk factors who was receiving low doses of oral bisphosphonate therapy (ibandronate, 150 mg monthly). Computed tomography (CT), panoramic radiographs (OPT), 99mTc-Sn-MDP scintigraphy, and magnetic resonance imaging (MRI) were performed to study the diseased area; cytological examination also revealed the presence of suppurative material around the area of exposed bone. A diagnosis of bisphosphonate-related osteonecrosis of the jaw complicated by osteomyelitis was made. The patient was prescribed a drug protocol consisting of metronidazole 250 mg 2 times daily, chlorhexidine mouthwashes 3 times daily and chewing exercises for two months. Ibandronate was stopped and replaced with strontium ranelate. The symptoms improved and the patient is still under close follow-up. Assessment of the benefits versus risks is particularly necessary in patients with several risk factors to ascertain their eligibility for treatment with antiresorptive drugs and when this is not possible to choose alternative medications.

scholarly journals Stereotactic radiosurgery for hemangiomas and ependymomas of the spinal cord

2003 ◽  
Vol 15 (5) ◽  
pp. 1-5 ◽  
Author(s):  
Stephen I. Ryu ◽  
Daniel H. Kim ◽  
Steven D. Chang
Keyword(s):  
Spinal Cord ◽  
Stereotactic Radiosurgery ◽  
Therapeutic Option ◽  
Spinal Tumors ◽  
High Dose ◽  
Significant Treatment ◽  
Conventional Radiation ◽  
Conformal Treatment ◽  
Dose Radiation

Object The optimal treatment for intramedullary spinal tumors is controversial, because both resection and conventional radiation therapy are associated with potential morbidity. Stereotactic radiosurgery can theoretically deliver highly conformal, high-dose radiation to surgically untreatable lesions while simultaneously mitigating radiation exposure to large portions of the spinal cord. The purpose of this study was to evaluate the authors' initial experience with frameless stereotactic radiosurgery for intramedullary spinal tumors. Methods Between 1998 and 2003, 10 intramedullary spinal tumors were treated with stereotactic radiosurgery at the authors' institution. Seven hemangioblastomas and three ependymomas were treated in four men and three women. These patients either had recurrent tumors, had undergone several previous surgeries, had medical contraindications to surgery, or had declined open resection. Conformal treatment planning delivered a prescribed dose of 1800 to 2500 cGy (mean 2100 cGy) to the lesions in one to three stages. No significant treatment-related complications have been recorded. The mean radiographic and clinical follow-up duration was 12 months (range 1–24 months). One ependymoma and two hemangioblastomas were smaller on follow-up neuroimaging. The remaining tumors were stable at the time of follow-up imaging. Conclusions Stereotactic radiosurgery for intramedullary spinal tumors is feasible and safe in selected cases and may prove to be another therapeutic option for these challenging lesions.

scholarly journals Favorable outcomes with reduced steroid use in juvenile dermatomyositis

2021 ◽  
Vol 19 (1) ◽  
Author(s):  
Amir B. Orandi ◽  
Lampros Fotis ◽  
Jamie Lai ◽  
Hallie Morris ◽  
Andrew J. White ◽  
...  
Keyword(s):  
Disease Control ◽  
Median Duration ◽  
Treatment Guidelines ◽  
Juvenile Dermatomyositis ◽  
Tertiary Care ◽  
Primary Outcome Measure ◽  
High Dose ◽  
Oral Steroids ◽  
Dose Oral

Abstract Background High-intensity glucocorticoid regimens are commonly used to induce and maintain remission in Juvenile Dermatomyositis but are associated with several adverse side-effects. Evidence-based treatment guidelines from North American and European pediatric rheumatology research societies both advocate induction with intravenous pulse steroids followed by high dose oral steroids (2 mg/kg/day), which are then tapered. This study reports the time to disease control with reduced glucocorticoid dosing. Methods We retrospectively reviewed the records at a single tertiary-care children’s hospital of patients diagnosed with Juvenile Dermatomyositis between 2000 and 2014 who had a minimum of 2 years of follow-up. The primary outcome measure was time to control of muscle and skin disease. Additional outcome measures included glucocorticoid dosing, effect of treatment on height, frequency of calcinosis, and complications from treatment. Results Of the 69 patients followed during the study period, 31 fulfilled inclusion criteria. Median length of follow-up was 4.58 years, (IQR 3–7.5). Myositis control was achieved in a median of 7.1 months (IQR 0.9–63.4). Cutaneous disease control was achieved in a median of 16.7 months (IQR 4.3–89.5). The median starting dose of glucocorticoids was 0.85 mg/kg/day, (IQR 0.5–1.74). The median duration of steroid treatment was 9.1 months, (IQR 4.7–17.4), while the median duration of any pharmacotherapy was 29.2 months (IQR 10.4 to 121.3). Sustained disease control off medications was achieved in 21/31 (68%) patients by the end of review. Persistent calcinosis was identified in only one patient (3%). Conclusion Current accepted treatment paradigms for Juvenile Dermatomyositis include oral glucocorticoids beginning at 2 mg/kg/day and reduced over a prolonged time period. However, our results suggest that treatment using reduced doses and duration with early use of steroid-sparing agents is comparably effective in achieving favorable outcomes in Juvenile Dermatomyositis.

Therapeutic response to glucocorticoids, anticoagulation and plasma exchange in a patient with primary antiphospholipid syndrome presenting with purpura fulminans

Lupus ◽  
2018 ◽  
Vol 27 (13) ◽  
pp. 2170-2173
Author(s):  
M Plüß ◽  
M Zeisberg ◽  
G A Müller ◽  
R Vasko ◽  
P Korsten
Keyword(s):  
Plasma Exchange ◽  
Antiphospholipid Syndrome ◽  
Therapeutic Response ◽  
Therapeutic Option ◽  
Purpura Fulminans ◽  
High Dose ◽  
Primary Antiphospholipid Syndrome ◽  
Internal Organs ◽  
Catastrophic Antiphospholipid Syndrome

We report the case of a 25-year-old female patient who presented with purpura fulminans as a manifestation of primary antiphospholipid syndrome. Purpura fulminans is considered a rare cutaneous manifestation of antiphospholipid syndrome. Most frequently, it occurs in the context of catastrophic antiphospholipid syndrome and is associated with significant morbidity and mortality, either due to loss of affected extremities or thromboembolic damage to internal organs. After insufficient efficacy of parenteral anticoagulation and oral glucocorticosteroid treatment, we escalated treatment to high-dose intravenous glucocorticosteroid and five consecutive sessions of plasma exchange with good and sustained clinical response. At follow-up six months after admission, skin manifestations had healed with scarring, and no additional thrombotic events had occurred. Plasma exchange may hold promise as a therapeutic option in refractory or severe cases of antiphospholipid syndrome-related purpura fulminans with extensive cutaneous necrosis, although evidence is limited.

High Dose Chemotherapy with Autologous Stem Cell Support Is Effective in Resistant and Refractory Hodgkin Lymphoma. A Single Center Analysis.

Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 5506-5506
Author(s):  
Emilia Cocorocchio ◽  
Fedro Peccatori ◽  
Simona Bassi ◽  
Federica Gigli ◽  
Davide Radice ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hodgkin Lymphoma ◽  
Progressive Disease ◽  
Therapeutic Option ◽  
High Dose Chemotherapy ◽  
Late Relapse ◽  
High Dose ◽  
Standard Chemotherapy ◽  
Refractory Hodgkin Lymphoma

Abstract We report retrospective data concerning 49 relapsed or refractory Hodgkin lymphoma pts, treated from 1995 in our institution with high dose chemotherapy (HDCT) with autologous peripheral blood stem cell (aPBSC) support. 41/49 had resistant disease, because presenting an early relapse (14/41), a late relapse (12/41) after CR induced by standard chemotherapy (CT) or persistence of disease (15/41) after initial CT. 8/49 had refractory disease, presenting stable or progressive disease within three months after the end of first line CT. Median age at transplantation time of 32 yrs (range 18–58 yrs). Before high dose chemotherapy, all pts received at least a standard chemotherapy (hybrid regimen for 4–6 cycles ± RT, according to the extension of disease). At the time of transplantation, 13 pts were in complete remission, 31 were in partial or very good partial remission and 5 were in stable /progressive disease. We used as myeloablative regimen mainly HDBEAM. Radiotherapy was performed in 9 pts after transplantation. In the subgroup of the 26 relapsed pts, 21 achieved a CR, 2 a PR and 3 a PD/SD; with a median follow up of 23 months from transplant (range 2 – 112 months), expected DFS and OS at 10 years are 90.5% and 76.9%, respectively. Analyzing separately early versus late relapsed patients, DFS expected at 10 years are 81.8% and 100%, respectively, and the OS expected are 71.4 and 83%, respectively. 11/15 pts with persistence of disease after initial CT achieved a CR, 2 PR, 2 presented SD/PD; with a median follow-up of 32 months from transplant (range 2 – 88 months), expected DFS and OS are 90.9% and 75%, respectively. Finally, 5/8 refractory pts achieved a CR (two of them progressed after transplant), 2 pts a PR (both progressed and died after transplant), 1 patient obtained a SD (progressed and died after transplant). No toxic deaths or second cancer or other severe late toxicity were recorded. Autologous transplantation is a safe procedure, considering the manageable early and late toxic effect; the clinical results achieved indicate that HDCT with aPBSC support is to be considered the first therapeutic option for those pts who present an early or late relapse after standard chemotherapy. The low number of refractory pts doesn’t allow any definitive conclusion about its role in this subgroup, but the relative high number of events observed seems to suggest that further analysis of biological features are warranted in order to better define the best therapeutic option.

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