Background: There is limited real-world evidence on hereditary angioedema (HAE) patient character... more Background: There is limited real-world evidence on hereditary angioedema (HAE) patient characteristics and health-careresource utilization (HCRU); in addition, pediatric patients have been described in small cohorts.Objective: To describe patient characteristics, treatment patterns, and HCRU among adult and pediatric patients treatedfor HAE in a large U.S. cohort.Methods: This retrospective cohort study used an administrative claims data base (January 2006 to September 2015).Eligible patients with either greater than or equal to one pharmacy claim for HAE-indicated therapies (C1 inhibitors, ecallantide, icatibant) or gtaer than or equal to two medical claims with codes associated with HAE (per medical billing codes), and greater than or equal to one claim for androgens, fresh frozen plasma, tranexamic acid, or « -aminocaproic acid formed a “treated cohort.” Three nonexclusive treated cohorts were assessed: overall, pediatric, and HCRU (greater than or equal to 2 years of continuous...
Background: Therapeutic modalities in the field of Complementary and Alternative Medicine (CAM) c... more Background: Therapeutic modalities in the field of Complementary and Alternative Medicine (CAM) continue to gain popularity as effective adjuncts to traditional interventions for a variety of medical conditions and developmental/psychological disorders, including autism spectrum disorders (ASD). Music therapy, a form of mind-body therapy, is a CAM technique used increasingly to promote musical and non-musical communicative behaviors, positive peer interactions, compliant behavior, emotional synchronicity, and initiation of engagement in children and young adults who have been diagnosed with autism or related disorder, with promising results. The Children’s Music Fund’s (CMF) mission is to provide music therapy for children and young adults with chronic pain, life altering illnesses, and developmental disorders, with the goal of promoting general well-being for its clients. In addition, CMF seeks to actively improve and increase the available research on music therapy within the fiel...
Allergy and asthma proceedings : the official journal of regional and state allergy societies, 2015
This article presents a current perspective on the characteristics of hereditary angioedema (HAE)... more This article presents a current perspective on the characteristics of hereditary angioedema (HAE) attacks and treatment as captured by a home infusion service. Retrospective data on 158 HAE patients who were enrolled in this acute treatment program were analyzed for factors surrounding an attack. The majority of patients had a high level of disease severity at baseline (88%), with a higher than expected likelihood of having a positive family history (87.8%). The most likely times for patients to call for home treatment were just before and during working hours (6:00 A.M.-5:00 P.M.). Eighty-three percent had more than one alternate mode of medication. Factors associated with a severe attack included an overall severe rating of HAE attacks in the previous year, an abdominal attack alone or a combination of peripheral and abdominal attacks versus a peripheral attack alone, and the use of two doses rather than one for treatment of the current attack. Average time to relief onset was 43....
Good's syndrome is a relatively rare immunodeficiency condition that presents in the fourth o... more Good's syndrome is a relatively rare immunodeficiency condition that presents in the fourth or fifth decade of life and is defined by hypogammaglobulinemia in the setting of a thymoma. The humoral defect may be severe enough to cause an absence in B cells, with a consequent recurrence of sinopulmonary disease, chronic non-infectious diarrhea and opportunistic infections. The prognosis in patients with Good's syndrome appears to be worse than in those with X-linked agammaglobulinemia (XLA) and common variable immune deficiency (CVID). There have only been three cases of Good's syndrome associated with mycobacterium, and only one case with a cavitary lesion in the lungs. We present here a unique case of Good's syndrome with a non-mycobacterial cavitary lesion.
Hereditary angioedema (HAE) due to C1-inhibitor deficiency is a rare autosomal dominant disease t... more Hereditary angioedema (HAE) due to C1-inhibitor deficiency is a rare autosomal dominant disease that manifests as sudden unpredictable attacks of subcutaneous or submucosal edema affecting the skin, intestine, and upper airway. Ecallantide is a plasma kallikrein inhibitor indicated for treatment of HAE attacks in patients aged 16 years and older. This analysis examines safety and efficacy of ecallantide for treatment of HAE attacks in patients <18 years of age. Data for patients aged 9 to 17 years treated subcutaneously with 30 mg ecallantide or placebo were pooled from 4 clinical studies (2 double-blind, placebo-controlled and 2 open-label). Efficacy end points included 2 HAE-specific patient-reported outcome measures: mean symptom complex severity (MSCS) score and treatment outcome score (TOS). Times to initial improvement, sustained improvement, and complete or near-complete symptom resolution were calculated. Treatment-emergent adverse events were examined. Overall, 29 pediatric patients were included; 25 of them received ecallantide for 62 total HAE attacks, and 10 received placebo for 10 total attacks. Ecallantide-treated attacks revealed clinically relevant reduction in symptom severity at 4 hours postdosing based on mean change in MSCS score (-1.4 ± 0.9 ecallantide versus -0.9 ± 0.6 placebo) and TOS (73.9 ± 35.50 ecallantide versus 45.0 ± 43.78 placebo). Patients treated with ecallantide showed rapid improvement in symptoms (median time to complete or near-complete symptom resolution: 181 minutes). No serious adverse events related to treatment were observed. Ecallantide appears effective for HAE attacks in adolescents, with rapid symptom improvement. No unexpected safety issues were identified.
We describe a unique case of urticaria associated with a Salmonella infection of uterine leiomyom... more We describe a unique case of urticaria associated with a Salmonella infection of uterine leiomyomas. A 55-year-old woman with a known history of uterine leiomyomas and a chief complaint of recurrent small and coalescing urticarial lesions confined to the abdomen presented with an 18-year history of recurrent fever and flu-like symptoms associated with the urticaria. After confirming the presence of a leiomyoma containing necrotic tissue on the computed tomography scan, a hysterectomy was performed. A large, 11-cm intramural leiomyoma was removed, and a culture of the purulent content grew a previously untyped Salmonella. The urticaria resolved on removal of the necrotic tissue. Subsequent follow-up for more than 2 years shows no relapse of symptoms. Our patient harbored Salmonella bacteria in a necrotic uterine leiomyoma, where it was difficult to detect until the time of surgery. In patients presenting with localized urticaria of the abdomen, an infection in the pelvic and abdominal tissue should be considered in the differential diagnosis.
Background: There is limited real-world evidence on hereditary angioedema (HAE) patient character... more Background: There is limited real-world evidence on hereditary angioedema (HAE) patient characteristics and health-careresource utilization (HCRU); in addition, pediatric patients have been described in small cohorts.Objective: To describe patient characteristics, treatment patterns, and HCRU among adult and pediatric patients treatedfor HAE in a large U.S. cohort.Methods: This retrospective cohort study used an administrative claims data base (January 2006 to September 2015).Eligible patients with either greater than or equal to one pharmacy claim for HAE-indicated therapies (C1 inhibitors, ecallantide, icatibant) or gtaer than or equal to two medical claims with codes associated with HAE (per medical billing codes), and greater than or equal to one claim for androgens, fresh frozen plasma, tranexamic acid, or « -aminocaproic acid formed a “treated cohort.” Three nonexclusive treated cohorts were assessed: overall, pediatric, and HCRU (greater than or equal to 2 years of continuous...
Background: Therapeutic modalities in the field of Complementary and Alternative Medicine (CAM) c... more Background: Therapeutic modalities in the field of Complementary and Alternative Medicine (CAM) continue to gain popularity as effective adjuncts to traditional interventions for a variety of medical conditions and developmental/psychological disorders, including autism spectrum disorders (ASD). Music therapy, a form of mind-body therapy, is a CAM technique used increasingly to promote musical and non-musical communicative behaviors, positive peer interactions, compliant behavior, emotional synchronicity, and initiation of engagement in children and young adults who have been diagnosed with autism or related disorder, with promising results. The Children’s Music Fund’s (CMF) mission is to provide music therapy for children and young adults with chronic pain, life altering illnesses, and developmental disorders, with the goal of promoting general well-being for its clients. In addition, CMF seeks to actively improve and increase the available research on music therapy within the fiel...
Allergy and asthma proceedings : the official journal of regional and state allergy societies, 2015
This article presents a current perspective on the characteristics of hereditary angioedema (HAE)... more This article presents a current perspective on the characteristics of hereditary angioedema (HAE) attacks and treatment as captured by a home infusion service. Retrospective data on 158 HAE patients who were enrolled in this acute treatment program were analyzed for factors surrounding an attack. The majority of patients had a high level of disease severity at baseline (88%), with a higher than expected likelihood of having a positive family history (87.8%). The most likely times for patients to call for home treatment were just before and during working hours (6:00 A.M.-5:00 P.M.). Eighty-three percent had more than one alternate mode of medication. Factors associated with a severe attack included an overall severe rating of HAE attacks in the previous year, an abdominal attack alone or a combination of peripheral and abdominal attacks versus a peripheral attack alone, and the use of two doses rather than one for treatment of the current attack. Average time to relief onset was 43....
Good's syndrome is a relatively rare immunodeficiency condition that presents in the fourth o... more Good's syndrome is a relatively rare immunodeficiency condition that presents in the fourth or fifth decade of life and is defined by hypogammaglobulinemia in the setting of a thymoma. The humoral defect may be severe enough to cause an absence in B cells, with a consequent recurrence of sinopulmonary disease, chronic non-infectious diarrhea and opportunistic infections. The prognosis in patients with Good's syndrome appears to be worse than in those with X-linked agammaglobulinemia (XLA) and common variable immune deficiency (CVID). There have only been three cases of Good's syndrome associated with mycobacterium, and only one case with a cavitary lesion in the lungs. We present here a unique case of Good's syndrome with a non-mycobacterial cavitary lesion.
Hereditary angioedema (HAE) due to C1-inhibitor deficiency is a rare autosomal dominant disease t... more Hereditary angioedema (HAE) due to C1-inhibitor deficiency is a rare autosomal dominant disease that manifests as sudden unpredictable attacks of subcutaneous or submucosal edema affecting the skin, intestine, and upper airway. Ecallantide is a plasma kallikrein inhibitor indicated for treatment of HAE attacks in patients aged 16 years and older. This analysis examines safety and efficacy of ecallantide for treatment of HAE attacks in patients <18 years of age. Data for patients aged 9 to 17 years treated subcutaneously with 30 mg ecallantide or placebo were pooled from 4 clinical studies (2 double-blind, placebo-controlled and 2 open-label). Efficacy end points included 2 HAE-specific patient-reported outcome measures: mean symptom complex severity (MSCS) score and treatment outcome score (TOS). Times to initial improvement, sustained improvement, and complete or near-complete symptom resolution were calculated. Treatment-emergent adverse events were examined. Overall, 29 pediatric patients were included; 25 of them received ecallantide for 62 total HAE attacks, and 10 received placebo for 10 total attacks. Ecallantide-treated attacks revealed clinically relevant reduction in symptom severity at 4 hours postdosing based on mean change in MSCS score (-1.4 ± 0.9 ecallantide versus -0.9 ± 0.6 placebo) and TOS (73.9 ± 35.50 ecallantide versus 45.0 ± 43.78 placebo). Patients treated with ecallantide showed rapid improvement in symptoms (median time to complete or near-complete symptom resolution: 181 minutes). No serious adverse events related to treatment were observed. Ecallantide appears effective for HAE attacks in adolescents, with rapid symptom improvement. No unexpected safety issues were identified.
We describe a unique case of urticaria associated with a Salmonella infection of uterine leiomyom... more We describe a unique case of urticaria associated with a Salmonella infection of uterine leiomyomas. A 55-year-old woman with a known history of uterine leiomyomas and a chief complaint of recurrent small and coalescing urticarial lesions confined to the abdomen presented with an 18-year history of recurrent fever and flu-like symptoms associated with the urticaria. After confirming the presence of a leiomyoma containing necrotic tissue on the computed tomography scan, a hysterectomy was performed. A large, 11-cm intramural leiomyoma was removed, and a culture of the purulent content grew a previously untyped Salmonella. The urticaria resolved on removal of the necrotic tissue. Subsequent follow-up for more than 2 years shows no relapse of symptoms. Our patient harbored Salmonella bacteria in a necrotic uterine leiomyoma, where it was difficult to detect until the time of surgery. In patients presenting with localized urticaria of the abdomen, an infection in the pelvic and abdominal tissue should be considered in the differential diagnosis.
Uploads
Papers by Raffi Tachdjian