First study of extended half-life rFVIIIFc in previously untreated patients with hemophilia A: PUPs A-LONG final results.

PUPs A-LONG evaluated the safety and efficacy of recombinant factor VIII Fc fusion protein (rFVIIIFc) in previously untreated patients (PUPs) with hemophilia A. This open-label, phase 3 study enrolled male PUPs (<6 years) with severe hemophilia A to receive rFVIIIFc. The primary endpoint was the occurrence of inhibitor development. Secondary endpoints included annualized bleed rate (ABR). Of 103 subjects receiving ≥1 dose of rFVIIIFc, 80 (78%) were aged <1 year at the study start, 20 (19%) had a family history of inhibitors, and 82 (80%) had high-risk F8 mutations. Twenty subjects began on prophylaxis, while 81 began an on-demand regimen (69 later switched to prophylaxis). Eighty-seven (81%) subjects completed the study. Inhibitor incidence was 31.1% (95% confidence interval [CI], 21.8% to 41.7%) in subjects with ≥10 exposure days (or inhibitor); high-titer inhibitor incidence was 15.6% (95% CI, 8.8% to 24.7%). The median (range) time to high-titer inhibitor development was 9 (4-14) exposure days. Twenty-eight (27%) subjects experienced 32 rFVIIIFc treatment-related adverse events; most were inhibitor development. There was 1 nontreatment-related death due to intracranial hemorrhage (onset before the first rFVIIIFc dose). The overall median (interquartile range [IQR]) ABR was 1.49 (0.00-4.40) for subjects on variable prophylaxis dosing regimens. In this study of rFVIIIFc in pediatric PUPs with severe hemophilia A, overall inhibitor development was within the expected range, although high-titer inhibitor development was on the low end of the range reported in the literature. rFVIIIFc was well-tolerated and effective for prophylaxis and treatment of bleeds. This trial is registered at www.clinicaltrials.gov (NCT02234323).

Daratumumab, bortezomib, cyclophosphamide and dexamethasone in newly diagnosed and relapsed multiple myeloma: LYRA study.

This United States community study evaluated the combination of daratumumab, bortezomib, cyclophosphamide and dexamethasone (D-VCd) in newly diagnosed multiple myeloma (NDMM) and relapsed multiple myeloma (RMM). Patients received 4-8 induction cycles of bortezomib 1·5 mg/m2 , cyclophosphamide 300 mg/m2 and dexamethasone 40 mg weekly. Intravenous daratumumab 16 mg/kg was administered as approved except for a split-first dose in Cycle 1. Eligible patients underwent autologous stem cell transplantation. All patients received ≤12 daratumumab maintenance doses monthly. Eighty-six NDMM and 14 RMM patients received ≥1 treatment dose. In NDMM patients, very good partial response or better (≥VGPR) and overall response rates after 4 induction cycles were 44% (primary endpoint) and 79%, respectively, and 56% and 81% at end of induction. The 12-month progression-free survival (PFS) rate was 87%. Efficacy was also observed in RMM patients. Fatigue (59%) and neutropenia (13%) were the most frequent treatment-emergent adverse event (TEAE) and grade 3/4 TEAE, respectively. Infusion reactions occurred in 54% of patients, primarily during the first dose, and were mild (2% grade 3). The first 2 daratumumab infusions were 4·5 and 3·8 h (median). Overall, D-VCd was well tolerated, split-first daratumumab dosing was feasible, the ≥VGPR rate after 4 cycles was 44% and the 1-year PFS rate was 87%.

Validation Study of the Composite Score to Identify Von Willebrand Disease in Children.

BACKGROUND: The diagnosis of type 1 von Willebrand disease (VWD) presents a diagnostic challenge in children. In fact, 25% or more of children with VWD may be diagnosed only after they experience postoperative bleeding. We previously described a 4-variable composite score that has 92.5% sensitivity and 95% specificity for diagnosing VWD in children with known VWD when 2 of 4 criteria are positive: (1) Tosetto bleeding score ≥ 1; (2) family history of VWD; (3) personal history of iron deficiency anemia; and/or (4) positive James early bleeding score. The purpose of this study was to prospectively validate a composite score of ≥ 2 for identifying children with VWD. PROCEDURE: Children without a previously diagnosed bleeding disorder presenting for hematology evaluation were enrolled. Sensitivity, specificity, positive, and negative predictive value of the composite score was determined. RESULTS: A total of 193 subjects were enrolled from 12 participating centers were included in the analysis. Forty-seven children had type 1 VWD, including 11 with von Willebrand Ristocetin Cofactor (VWF):RCo < 30 IU/dL, 14 subjects with a VWF:RCo 30 to 39 IU/dL, and 22 with a VWF:RCo 40 to 49 IU/dL. Including all 4 variables, a composite score of ≥ 2 had a sensitivity of 63.6% to 76.0%, specificity of 33.5% to 35.1%, negative predictive value of 76.9% to 93.8%, and positive predictive value of 5.5% to 25%. CONCLUSIONS: The negative predictive value of the composite score was robust, especially at lower VWF:RCo suggesting that VWD testing could be eliminated in nearly a third of children referred for VWD testing.

Impact of individualized pain plan on the emergency management of children with sickle cell disease.

BACKGROUND: Vaso-occlusive crisis (VOC) the hallmark of sickle cell disease (SCD) is often treated inadequately in the emergency department (ED). We hypothesized that pain management plans individualized for each patient can improve pain management and lead to high levels of patient satisfaction. PROCEDURE: Starting in 2002, we treated all patients with SCD reporting to Children's Hospital of Pittsburgh (CHP) ED with VOC using a structured algorithm. We recorded regimens used successfully for each patient as an "individualized pain plan" and implemented it during subsequent VOC visits and adjusted it to patient response. We compared rates of hospitalization following an ED visit with VOC and readmission within 1 week after discharge for CHP with that of four comparable hospitals from Pediatric Health Information (PHIS) database. Patients and parents completed surveys of satisfaction with pain management and with care. RESULTS: Between 2002 and 2008 there was a greater decline in the rate of admission of patients presenting to the ED at CHP (78% to 52%) as compared to PHIS (71% to 68%), (P < 0.05) and readmission rates at CHP (7.3% to 3.2%) as compared to PHIS (6.5% to 5.1%) (P < 0.05). Improvement in pain score during ED management was 2.0 or more on a Wong Baker scale of 0-5 (P < 0.01). Participants on average, rated quality of pain management as very good or higher. CONCLUSION: Individualized pain management plans in the ED are effective in delivering high quality management of VOC and are associated with a high level of patient satisfaction and decreased avoidable hospitalizations.

Predictors of von Willebrand disease in children.

BACKGROUND: Bleeding and bruising are common symptoms for which children are referred to a pediatric hematologist. Although bleeding scores have been validated to quantify bleeding risk in adults, there are no similar definitive data in children. The aim was to describe presenting bleeding symptoms in children and evaluate if these bleeding symptoms were predictive of von Willebrand disease (VWD) or low von Willebrand factor (VWF). PROCEDURE: We performed a retrospective chart review of consecutive pediatric patients referred for perceived bleeding symptoms to the coagulation clinic at The Children's Hospital of Pittsburgh of UPMC. All underwent a uniform bleeding symptom inventory and hemostatic testing. Single and multiple variable logistic regression models were created to predict either VWD, low VWF, or nonspecific defective platelet aggregation, using the predictor variables of family bleeding history, mucocutaneous bleeding, cutaneous bleeding, and surgical bleeding, based on The International Society on Thrombosis and Hemostasis (ISTH) bleeding definitions. RESULTS: Of 298 pediatric patients evaluated, 8% had VWD (VWF:RCo and VWF:Ag ≤ 30 IU/dl) and 34% had low VWF (VWF:RCo and VWF:Ag 30-50 IU/dl). Further, 16% had a nonspecific platelet aggregation disorder, 41% had normal hemostatic testing, and 1% had factor VIII or IX deficiency. In single and multiple variable logistic regression analysis, neither a personal or family bleeding history at presentation, nor the presence of two or more bleeding symptoms were predictive of VWD, low VWF, or nonspecific defective platelet aggregation. CONCLUSIONS: These findings suggest that qualitative assessment of bleeding symptoms alone is not useful in children.

Oocyte cryopreservation in a patient with sickle cell disease prior to hematopoietic stem cell transplantation: first report.

PURPOSE: To report the first occurrence of successful ovarian stimulation, oocyte retrieval and oocyte cryopreservation for fertility preservation in an adolescent with severe sickle cell disease scheduled to undergo a hematopoietic stem cell transplant METHODS: Case report. RESULTS: A 19 year old female with severe sickle cell disease presented for fertility preservation counseling prior to hematopoietic stem cell transplantation. She ultimately underwent ovarian stimulation using a minimal stimulation GnRH antagonist protocol resulting in the successful banking of oocytes prior to transplant. The unique hazards associated with ovarian stimulation in patients with sickle cell disease, such as thrombosis and vaso-occlusive events, are discussed and the methods undertaken to minimize these risks are described. CONCLUSIONS: Controlled ovarian hyperstimulation and oocyte banking for fertility preservation is feasible in young women with sickle cell disease requiring hematopoietic stem cell transplant and deserves further investigation. Given the elevated risk of thrombosis and predisposition to painful vaso-occlusive events, controlled ovarian hyperstimulation in patients with sickle cell disease is not straightforward and requires a multi-disciplinary team approach to adequately address and minimize the risks in this unique patient population.

Long-term follow-up of patients treated with caplacizumab and safety and efficacy of repeat caplacizumab use: Post-HERCULES study.

INTRODUCTION: Caplacizumab demonstrated efficacy and safety in patients with immune-mediated thrombotic thrombocytopenic purpura (iTTP) in the phase 3 HERCULES trial. However, data on long-term outcomes following caplacizumab treatment are limited. OBJECTIVES: The post-HERCULES trial (NCT02878603) evaluated long-term outcomes of patients with iTTP treated with caplacizumab in HERCULES and safety and efficacy of repeated caplacizumab use. PATIENTS/METHODS: Over 3 years of follow-up, patients could receive open-label caplacizumab with therapeutic plasma exchange (TPE) and immunosuppressive therapy (IST) in case of recurrence. Adverse events (AEs) were assessed during the overall study period (intention-to-observe [ITO] population) and during recurrences (recurrence population). TTP-related events (TTP-related death, recurrence, major thromboembolic events) were assessed in the efficacy ITO population (patients without recurrence during HERCULES or before post-HERCULES). RESULTS: Among 104 enrolled patients, incidences of AEs and serious AEs were similar between patients who had received caplacizumab + TPE + IST during HERCULES (n = 75) and those treated with placebo + TPE + IST (placebo; n = 29). TTP-related events occurred in 8% of patients (4/49) randomized to caplacizumab during HERCULES versus 38% (11/29) randomized to placebo. Nineteen patients had ≥1 recurrence; 13 of these were treated with caplacizumab. The first recurrence episode was resolved or resolving for all patients treated with caplacizumab, including nine patients with repeat caplacizumab use. All second recurrences (6/6) were resolved. Safety profile of caplacizumab for treatment of recurrence was consistent with HERCULES; most bleeding events were nonserious. No major cases of organ dysfunction were observed. CONCLUSIONS: Long-term follow-up supports the safety and efficacy of caplacizumab for iTTP and its repeated use for recurrences.

Final results of the PUPs B-LONG study: evaluating safety and efficacy of rFIXFc in previously untreated patients with hemophilia B.

PUPs B-LONG evaluated the safety and efficacy of recombinant factor IX Fc fusion protein (rFIXFc) in previously untreated patients (PUPs) with hemophilia B. In this open-label, phase 3 study, male PUPs (age <18 years) with hemophilia B (≤2 IU/dL of endogenous factor IX [FIX]) were to receive treatment with rFIXFc. Primary end point was occurrence of inhibitor development, with a secondary end point of annualized bleed rate (ABR). Of 33 patients who received ≥1 dose of rFIXFc, 26 (79%) were age <1 year at study entry and 6 (18%) had a family history of inhibitors. Twenty-eight patients (85%) received prophylaxis; median dosing interval was 7 days, with an average weekly dose of 58 IU/kg. Twenty-seven patients (82%) completed the study. Twenty-one (64%), 26 (79%), and 28 patients (85%) had ≥50, ≥20, and ≥10 exposure days (EDs) to rFIXFc, respectively. One patient (3.03%; 95% confidence interval, 0.08% to 15.76%) developed a low-titer inhibitor after 11 EDs; no high-titer inhibitors were detected. Twenty-three patients (70%) had 58 treatment-emergent serious adverse events; 2 were assessed as related (FIX inhibition and hypersensitivity in 1 patient, resulting in withdrawal). Median ABR was 1.24 (interquartile range, 0.00-2.49) for patients receiving prophylaxis. Most (>85%) bleeding episodes required only 1 infusion for bleed resolution. In this first study reporting results with rFIXFc in pediatric PUPs with hemophilia B, rFIXFc was well tolerated, with the adverse event profile as expected in a pediatric hemophilia population. rFIXFc was effective, both as prophylaxis and in the treatment of bleeding episodes. This trial was registered at www.clinicaltrials.gov as #NCT02234310.

Do neonates, infants and young children need a higher dose of enoxaparin in the cardiac intensive care unit?

BACKGROUND: Thromboembolic events are a serious complication occurring in critically ill children admitted to the cardiac intensive care unit. Although enoxaparin is one of the current anticoagulants of choice, dosages in children are extrapolated from adult guidelines. Recent data suggest that this population may need a higher dose than what is currently recommended to achieve target anti-factor Xa levels. The purpose of this study was to evaluate whether children less than 2 years old admitted to the cardiac intensive care unit require a higher enoxaparin dose than that currently recommended to achieve target anti-factor Xa levels. METHODS: Retrospective chart review including patients who received enoxaparin for the treatment or prophylaxis of venous thrombosis between January, 2005 and October, 2007. Patients were classified as younger and older as well as prophylactic and therapeutic on the basis of age and enoxaparin dose, respectively. Younger patients were those 2 month old or less and older patients were those older than 2 months of age. RESULTS: A total of 31 patients were identified; 13 (42%) were 2 months or younger and 25 (81%) were postoperative patients. Ten (32%) received prophylactic and 21 (68%) received therapeutic enoxaparin doses. To achieve optimal anti-factor Xa levels, enoxaparin dose was increased in all groups and reached statistical significance in all patients except those older than 2 months who received prophylactic enoxaparin. An average of 2.8 dosage adjustments was needed. No bleeding complications were reported. CONCLUSIONS: Young children, infants, and neonates admitted to the cardiac intensive care unit required a significantly higher enoxaparin dose than that currently recommended to achieve target anti-factor Xa levels.

Phenotypical variability in congenital FVII deficiency follows the ISTH-SSC severity classification guidelines: a review with illustrative examples from the clinic.

BACKGROUND: One of the most common rare inherited bleeding disorders, congenital factor VII (FVII) deficiency typically has a milder bleeding phenotype than other rare bleeding disorders. Categorizing severity in terms of factor activity associated with hemophilia (severe <1%, moderate 1%-5%, mild 6%-40%) has led to the observation that bleeding phenotype does not follow closely with FVII activity. Over the past decade, large-scale global registries have investigated bleeding phenotype more thoroughly. The International Society on Thrombosis and Haemostasis has reclassified FVII deficiency as follows: severe, FVII <10%, risk of spontaneous major bleeding; moderate, FVII 10%-20%, risk of mild spontaneous or triggered bleeding; mild, FVII 20%-50%, mostly asymptomatic disease. CASE REPORTS: Eleven illustrative cases of congenital FVII deficiency adapted from clinical practice are described to demonstrate the variability in presentation and in relation to FVII activity levels. Severe FVII deficiency usually presents at a young age and carries the risk of intracranial hemorrhage, hemarthrosis, and other major bleeds. Moderate FVII deficiency tends to present later, often in adolescence and particularly in girls as they reach menarche. Milder disease may not be apparent until found incidentally on preoperative testing, during pregnancy/childbirth, or following unexplained bleeding when faced with hemostatic challenges. CONCLUSION: It is important for health care professionals to be aware of the new definitions of severity and typical presentations of congenital FVII deficiency. Failure to appreciate the risks of major bleeding, including intracerebral hemorrhage in those with FVII activity <10%, may put particularly young children at risk.

Epistaxis as a Common Presenting Symptom of Glanzmann's Thrombasthenia, a Rare Qualitative Platelet Disorder: Illustrative Case Examples.

Children often present to emergency departments (EDs) with uncontrollable nose bleeding. Although usually due to benign etiologies, epistaxis may be the presenting symptom of an inherited bleeding disorder. Whereas most bleeding disorders are detected through standard hematologic assessments, diagnosing rare platelet function disorders may be challenging. Here we present two case reports and review diagnostic and management challenges of platelet function disorders with a focus on Glanzmann's thrombasthenia (GT). Patient 1 was a 4-year-old boy with uncontrolled epistaxis. His medical history included frequent and easy bruising. Previous laboratory evaluation revealed only mild microcytic anemia. An otolaryngologist stopped the bleeding, and referral to a pediatric hematologist led to the definitive diagnosis of GT. Patient 2 was a 2.5-year-old girl with severe epistaxis and a history of milder recurrent epistaxis. She had a bruise on her abdomen with a palpable hematoma and many scattered petechiae. Previous assessments revealed no demonstrable hemostatic anomalies. Platelet aggregation studies were performed following referral to a pediatric hematologist, leading to the diagnosis of GT. As evidenced by these cases, the ED physician may often be the first to evaluate severe or recurrent epistaxis and should recognize indications for coagulation testing and hematology consultation/referral for advanced hematologic assessments.

Recognition and management of platelet-refractory bleeding in patients with Glanzmann's thrombasthenia and other severe platelet function disorders.

Patients with rare qualitative platelet disorders or platelet function disorders (PFDs) may present to the hospital physician with severe bleeding episodes or excessive surgical bleeding. Although standard treatment consists of platelet transfusions, repeated transfusions may result in the development of antiplatelet antibodies (APA) or clinical refractoriness, rendering further platelet therapy ineffective. In such settings, an approved treatment option for patients with Glanzmann's thrombasthenia (GT), one of the well-known rare PFDs, is recombinant activated coagulation factor VII (rFVIIa). Data regarding the efficacy of rFVIIa in patients with GT and platelet refractoriness are available from a large patient registry, an international survey, and multiple case reports and demonstrate efficacy in patients with and without refractoriness or APA. This article reviews the rFVIIa clinical data in patients with GT and platelet refractoriness and discusses clinical implications relevant to the hospital-based physician. Because uncontrolled bleeding can be life-threatening, hospital physicians should be alert to the signs of platelet refractoriness, be able to recognize continued internal or external bleeding, and know how to adapt treatment regimens for the effective management of bleeding. The management of patients who receive rFVIIa should occur in consultation with a hematologist with experience in PFDs, and patients with suspected platelet refractoriness should be referred to such a hematologist as early as possible. A critical unmet need is the development of a definition of an adequate response to platelet transfusion, which would facilitate early recognition of platelet refractoriness in patients with PFDs who exhibit a normal platelet count.

Heavy Menstrual Bleeding as a Common Presenting Symptom of Rare Platelet Disorders: Illustrative Case Examples.

Heavy menstrual bleeding (HMB) is a common symptom in patients who present to the obstetrician-gynecologist or adolescent medicine specialist and might result from an underlying inherited bleeding disorder. Whereas relatively common bleeding disorders such as von Willebrand disease are often included in standard laboratory assessments, rarer platelet function disorders can be challenging to diagnose. Additionally, HMB can be a particularly difficult symptom to manage in adolescents with platelet function disorders, and it is associated with decreased quality of life. We review the diagnostic and management issues of patients with platelet function disorders through the presentation of 2 patient case reports, with a focus on a diagnosis of Glanzmann thrombasthenia, an inherited qualitative disorder that affects platelet function. Whereas the first patient presented to the emergency department before the diagnosis of a bleeding disorder and required a hematologic referral and extensive laboratory assessments, the second patient had been diagnosed with Glanzmann thrombasthenia as a child but experienced severe management challenges at the onset of menarche. In both patients, collaboration between the obstetrician-gynecologist or adolescent medicine specialist and the hematologist was critical for achieving acute management of the bleeding symptoms and for ensuring optimal long-term disease management. Together, these cases highlight the importance of properly identifying females with HMB who might have an undiagnosed bleeding disorder and of consulting with a hematologist to determine an appropriate management plan throughout all life stages.

Malignant pleural mesothelioma in a child with ataxia-telangiectasia.

Ataxia-telangiectasia (AT) is a hereditary disorder characterized by progressive neurological dysfunction, oculocutaneous telangiectasia, immunodeficiency, cancer susceptibility, and radiation sensitivity. Pleural neoplasms are extremely rare in the pediatric population, even in patients with AT. We describe the case of a 16-year-old male with AT who developed a malignant pleural mesothelioma (MPM). Benign or infectious lung and pleural diseases are common in those with AT. Hence, delayed diagnosis of respiratory neoplasms can occur in these patients. This report highlights the need of heightened vigilance in patients with AT with recurrent or persistent pleuropulmonary disease. To our knowledge, no other cases of MPM in children with AT have been reported.

The prevalence of bleeding disorders among healthy pediatric patients with abnormal preprocedural coagulation studies.

BACKGROUND: We evaluated the prevalence of hemostatic disorders among pediatric patients with abnormal screening coagulation tests. PROCEDURE: We analyzed 48 consecutive referrals for abnormal prothrombin times, partial thromboplastin times, or closure times obtained as preprocedural screens. Patients were evaluated by uniform diagnostic testing. RESULTS: Seventeen patients (35%) had an isolated nonspecific inhibitor (NSI). Six patients (12.5%) presented with mildly low factor activity with a concomitant NSI. These deficiencies were of unclear clinical significance. One patient (2%) had a lupus anticoagulant. Only 9 patients (19%) had a possible or true mild bleeding disorder: 5 patients (10%) had isolated low von Willebrand factor levels, 2 patients (4%) had possible type I von Willebrand disease, and 2 (4%) had platelet aggregation disorders. In all patients, personal and family bleeding history had a positive predictive value of 45% for hemostatic disorders. CONCLUSIONS: The most common diagnosis among the patients referred to us for abnormal preoperative coagulation tests was a NSI, which is not associated with an increased risk of operative bleeding complications. Less than 20% had a possible or true mild bleeding disorder. Although certain bleeding disorders can be occult in children and are associated with perioperative bleeding risks, our study demonstrates the inherent limitations in making a laboratory diagnosis of a bleeding disorder in pediatric patients preoperatively. Our findings contribute to existing doubt about the usefulness of prothrombin times, partial thromboplastin times, and closure times to identify occult bleeding disorders in this population.

Caring Connection: Internet resources for family caregivers of children with cancer.

This poster will describe the evaluation of a family-focused web-based resource, the "Caring Connection", designed to provide individualized clinical information, patient-centric information resources, and access to online family-to-provider and family-to-family communication for caregivers of children with cancer. Data from structured interviews with family caregivers describe their Internet use and information needs. The interview data further provide information regarding the information and communication needs of family caregivers of children with cancer. Website server logs provide data to corroborate the interview findings.

Elevation of tricuspid regurgitant jet velocity, a marker for pulmonary hypertension in children with sickle cell disease.

BACKGROUND: Pulmonary hypertension (PHTN) is a potentially life-threatening complication, detected by echocardiographic evidence of elevated tricuspid regurgitant velocity (TRV). This condition has been described in adults with sickle cell disease (SCD) and other hemolytic disorders; however, there is little information on the occurrence of this condition in pediatric patients. METHODS: Records for pediatric SCD patients were retrospectively reviewed to determine clinical characteristics and co-morbidities of patients with elevated TRV on echocardiograms obtained under steady state conditions as an outpatient. Correlation of TRV > or =2.5 m/sec with age, sex, type of SCD, number of outpatient echocardiograms per patient, episodes of vasoocclusive crisis (VOC) and acute chest syndrome (ACS), mean hemoglobin and reticulocyte count, asthma, obstructive sleep apnea, cerebrovascular disease (CVD), and hydroxyurea therapy was determined. RESULTS: Of 224 SCD patients, 44 had outpatient echocardiographic measurement of TRV. Patients (11 of 44) (26.2%) with TRV > or =2.5 m/sec were compared to 31 patients without elevated TRV. Significant differences were noted for percent with HbSS disease (P = 0.041), CVD (P = 0.021), hemoglobin (P = 0.003), % reticulocytes (P = 0.037), and number of echocardiograms performed (P < 0.001). No significant differences were observed for gender, age, asthma, or frequency of VOC and ACS. CONCLUSIONS: Elevated TRV, a surrogate marker for PHTN, occurs in children with SCD and is associated with low hemoglobin, elevated reticulocyte count, and cerebral vasculopathy. Appropriate screening by echocardiography can lead to detection and treatment that may reduce TRV and potentially reverse the disease process, prevent the increased morbidity and mortality associated with PHTN.

Caring connection: developing an Internet resource for family caregivers of children with cancer.

This article describes the development of a family-focused Internet resource, the Caring Connection Web site, designed to provide individualized clinical information, patient-centered information, and access to online communication for caregivers of children with cancer. Data from surveys with family caregivers described the scope and characteristics of their current Internet use. Web site prototype development and user testing provided additional insights that inform the ongoing design of the Caring Connection Web site. The family caregivers and oncology healthcare providers who participated are active users of Internet health information. The preliminary data they provided support the feasibility and usability of the Caring Connection prototype. Ongoing development of the Caring Connection Web site will provide an innovative approach to assist these family caregivers in meeting currently unmet information and communication needs, and will provide evidence regarding "best practice" in design and development of Internet resources to support communication and information sharing.

Caring Connection: developing an Internet resource for family caregivers of children with cancer.

This poster will describe the development of a family-focused, web-based resource, the "Caring Connection", designed to provide individualized clinical information, patient-centered information resources, and access to online family-provider and family-to-family communication for caregivers of children with cancer. Data from structured interviews with family caregivers and healthcare providers describe their current Internet use and information needs. Website prototype development and user testing provide additional insights that are informing the ongoing design of the "Caring Connection" website.