Melodee Nugent

Peripheral blood stem cell donation: an analysis from the International Bone Marrow Transplant Registry (IBMTR) and European Group for Blood and Marrow Transplant (EBMT) databases

Donation-related data for 1488 allogeneic peripheral blood stem cell (PBSC) transplants reported to the International Bone Marrow Transplant Registry (IBMTR) or the European Blood and Marrow Transplant Group (EBMT) by 152 teams worldwide between 1994 and 1998 were reviewed. In 1998, 26% of allografts registered with the IBMTR were collected from blood. Median age of PBSC donors was 38 years (range <1–76), and 55% were male. Of 1486 donor–recipient pairs evaluable for HLA compatibility, 1322 (89%) were HLA-identical siblings. Recombinant human granulocyte colony-stimulating factor (G-CSF) was employed to mobilize PBSCs in almost all (99%) cases. One hundred and seventy (20%) of 828 evaluable PBSC donors had a central catheter placed for leukapheresis. Eighty-five percent of 1321 evaluable PBSC grafts were collected with one or two leukaphereses. There were 15 reported donation-related adverse events (1% of evaluable donors). Complications were catheter-related in five. No donation-related fatalities were reported. These data suggest that PBSC donation is becoming more prevalent worldwide. It appears to have a safety profile comparable to marrow harvesting, although experience with the latter is much more extensive. Bone Marrow Transplantation (2001) 27, 689–692.

Fontan Palliation in the Modern Era: Factors Impacting Mortality and Morbidity

BACKGROUND Advances in management of the Fontan patient include interval superior cavopulmonary shunt, total cavopulmonary connection, either lateral tunnel or extracardiac conduit, and the use of a fenestration. Coincident with these improvements, Fontan palliation has been applied to a wider ranger of anatomic subgroups. METHODS A cross-sectional analysis of 256 consecutive patients undergoing a total cavopulmonary connection Fontan after superior cavopulmonary shunt between January 1, 1994, and June 30, 2007 were studied. Fenestration was used selectively. Fontan failure was defined as death, transplant, or takedown. Event-free survival was defined as freedom from death, transplant, Fontan takedown, functional class III to IV, pacemaker, antiarrhythmic medication, protein-losing enteropathy, stroke, or thrombus. RESULTS Survival was 97% +/- 1%, 96% +/- 1%, and 94% +/- 2%, respectively, at 1, 5, and 10 years. Event-free survival was 96% +/- 1%, 87% +/- 3%, and 64% +/- 6%, respectively, at 1, 5, and 10 years. Factors predicting worse event-free survival included longer cross-clamp time (p = 0.003), fenestration (p = 0.014), and longer hospital length of stay (p = 0.016). Ventricular morphology did not predict outcome. Left ventricle (n = 113, 44%) versus right ventricle (n = 142, 56%) failure-free survival (death, transplant, or Fontan takedown) at 10 years was 92% +/- 4% versus 91% +/- 3%, respectively (p = 0.19). Left ventricle versus right ventricle event-free survival at 10 years was 75% +/- 7% versus 67% +/- 9%, respectively (p > 0.1). CONCLUSIONS Survival for patients undergoing a completion Fontan in the current era is excellent, but patients remain at risk for morbid events. In the intermediate follow-up period, we could not identify a difference in outcome between dominant left and right ventricle morphology.

Bone marrow transplants for paroxysmal nocturnal haemoglobinuria

Paroxysmal nocturnal haemoglobinuria (PNH) is a rare clonal haematological disorder characterized by intravascular haemolysis and increased risk of thrombosis. PNH is associated with bone marrow failure syndromes including aplastic anaemia, myelodysplasia and leukaemia. Bone marrow transplants are sometimes used to treat PNH, but small series and reporting biases make assessment of transplant outcome difficult. The outcome of 57 consecutive allogeneic bone marrow transplants for PNH reported to the International Bone Marrow Transplant Registry (IBMTR) between 1978 and 1995 was analysed. The 2‐year probability of survival in 48 recipients of HLA‐identical sibling transplants was 56% (95% confidence interval 49–63%). Two recipients of identical twin transplants remain alive 8 and 12 years after treatment. One of seven recipients of alternative donor allogeneic transplants is alive 5 years after transplant. The most common causes of treatment failure were graft failure and infections. Our results indicate that bone marrow transplantion can restore normal bone marrow function in about 50% of PNH patients.

Sentinel Injuries in Infants Evaluated for Child Physical Abuse

OBJECTIVE: Relatively minor abusive injuries can precede severe physical abuse in infants. Our objective was to determine how often abused infants have a previous history of “sentinel” injuries, compared with infants who were not abused. METHODS: Case-control, retrospective study of 401, <12-month-old infants evaluated for abuse in a hospital-based setting and found to have definite, intermediate concern for, or no abuse after evaluation by the hospital-based Child Protection Team. A sentinel injury was defined as a previous injury reported in the medical history that was suspicious for abuse because the infant could not cruise, or the explanation was implausible. RESULTS: Of the 200 definitely abused infants, 27.5% had a previous sentinel injury compared with 8% of the 100 infants with intermediate concern for abuse (odds ratio: 4.4, 95% confidence interval: 2.0–9.6; P < .001). None of the 101 nonabused infants (controls) had a previous sentinel injury (P < .001). The type of sentinel injury in the definitely abused cohort was bruising (80%), intraoral injury (11%), and other injury (7%). Sentinel injuries occurred in early infancy: 66% at <3 months of age and 95% at or before the age of 7 months. Medical providers were reportedly aware of the sentinel injury in 41.9% of cases. CONCLUSIONS: Previous sentinel injuries are common in infants with severe physical abuse and rare in infants evaluated for abuse and found to not be abused. Detection of sentinel injuries with appropriate interventions could prevent many cases of abuse.

Assessment of regional right ventricular velocities, strain, and displacement in normal children using velocity vector imaging.

Background: Velocity vector imaging (VVI) is a novel technique to assess regional myocardial mechanics. We evaluated the utility of VVI in the assessment of right ventricular (RV) function in normal children. Methods: RV images (apical 4‐chamber view, high frame rate capture) from 30 normal children (mean 8.7 years; range 1.1–18) were selected. Longitudinal myocardial velocities, peak systolic strain (PSS), maximal longitudinal displacement (LD), and times to peak for these parameters were obtained from six RV segments. Simultaneous Doppler tissue imaging (DTI) derived peak systolic and early diastolic velocities were obtained from the free tricuspid annulus. Measurements were made independently by two observers. Results: Qualitative analysis shows dominant longitudinal systolic and diastolic myocardial motion, particularly at the basal segments. Velocities and LD were greatest at the basal segments and decreased from base to apex. PSS values were less consistent between segments and observers, with the greatest PSS generally found in the apical segments. LD and time to peak displacement (TPD) were the most reproducible. TPD in the basal segments were strikingly similar in each patient and correlated with R‐R intervals. Peak diastolic velocities in the right base did correlate with corresponding annular DTI velocities; DTI velocities were consistently higher than VVI velocities. Conclusions: RV segmental velocities and LD are reliably obtained by VVI. PSS is less reproducible. LD is a reproducible measure of systolic function and TPD appears to be useful in the assessment of synchrony. Correlation of these findings with children who have known RV pathology is an important next step.

Incidence, Clinical Characteristics, and Natural History of Pediatric IBD in Wisconsin: a Population-based Epidemiological Study

Background:Epidemiological studies of pediatric inflammatory bowel diseases (IBD) are needed to generate etiological hypotheses and inform public policy; yet, rigorous population-based studies of the incidence and natural history of Crohns disease (CD) and ulcerative colitis (UC) in the United States are limited. Methods:We developed a field-tested prospective system for identifying all new cases of IBD among Wisconsin children over an 8-year period (2000–2007). Subsequently, at the end of the study period, we retrospectively reconfirmed each case and characterized the clinical course of this incident cohort. Results:The annual incidence of IBD among Wisconsin children was 9.5 per 100,000 (6.6 per 100,000 for CD and 2.4 per 100,000 for UC). Approximately 19% of incident cases occurred in the first decade of life. Over the 8-year study period, the incidence of both CD and UC remained relatively stable. Additionally, (1) childhood IBD affected all racial groups equally, (2) over a follow-up of 4 years, 17% of patients with CD and 13% of patients with patients with UC required surgery, and (3) 85% and 40% of children with CD were treated with immunosuppressives and biologics, respectively, compared with 62% and 30% of patients with UC. Conclusions:As in other North American populations, these data confirm a high incidence of pediatric-onset IBD. Importantly, in this Midwestern U.S. population, the incidence of CD and UC seems to be relatively stable over the last decade. The proportions of children requiring surgery and undergoing treatment with immunosuppressive and biological medications underscore the burden of these conditions.

Medical providers' understanding of sex trafficking and their experience with at-risk patients.

BACKGROUND AND OBJECTIVES: Sex trafficking (ST) victims have unique medical and mental health needs and are often difficult to identify. Our objectives were to evaluate knowledge gaps and training needs of medical providers, to demonstrate the importance of provider training to meet the pediatric ST victim’s specific needs, and to highlight barriers to the identification of and response to victims. METHODS: A survey was sent to providers in specialties that would be most likely to encounter victims of ST. Participants included physicians, nurses, physician assistants, social workers, and patient and family advocates at multiple hospitals and medical clinics in urban, suburban, and rural locations. RESULTS: Of ∼500 survey recipients, 168 participants responded. In 2 clinical vignettes, 48% correctly classified a minor as an ST victim, and 42% correctly distinguished an ST victim from a child abuse victim. In all, 63% of respondents said that they had never received training on how to identify ST victims. Those with training were more likely to report ST as a major problem locally (P ≤ .001), to have encountered a victim in their practice (P ≤ .001), and to have greater confidence in their ability to identify victims (P ≤ .001). The greatest barriers to identification of victims reported were a lack of training (34%) and awareness (22%) of ST. CONCLUSIONS: Health care providers demonstrate gaps in knowledge and awareness of ST, specifically of pediatric victims, that correlate with their limited experience and training. Training is crucial to improve identification of these victims and provide appropriate care for their specific needs.

Pulmonary Valve Replacement Improves But Does Not Normalize Right Ventricular Mechanics in Repaired Congenital Heart Disease: A Comparative Assessment Using Velocity Vector Imaging

BACKGROUND This study evaluated regional right ventricular (RV) mechanics before and after pulmonary valve replacement (PVR) by ultrasonic speckle tracking technology using velocity vector imaging (VVI). METHODS Fifty-eight patients who underwent PVR (May 1999 to August 2007) were included. Two-dimensional Doppler indices included qualitative pulmonary incompetence, RV outflow tract peak gradient, RV systolic pressure estimate, indexed RV area, and fractional area change; VVI regional indices included peak systolic and diastolic velocities, peak systolic strain, maximal longitudinal displacement, and times to peak measure from 6 RV segments. RESULTS PVR was performed at a median age of 12.1 years with echocardiographic analysis at median intervals of 2.8 months before and 30 months after PVR. Peak velocities and displacement increased in all 6 RV segments after PVR; peak systolic strain did not improve consistently. All indices remained significantly lower compared with normal values. There was a significant decrease in the degree of pulmonary incompetence, RV outflow tract gradient, RV systolic pressure, and indexed RV area (23.5 vs. 17.8 cm(2)/m(2)), but no significant change in the percentage of fractional area change (28.8% vs. 29.6%). CONCLUSION Regional RV mechanics using VVI in a relatively young cohort shows mild improvement after PVR, but the RV is not normalized despite physiologic improvement in loading conditions. This suggests intrinsic dysfunction or chronic myocardial injury that is nonmodifiable or requires earlier intervention to optimize physiology. VVI appears to be a potentially useful quantitative tool for follow-up evaluation of RV performance after congenital heart disease surgery.

Thromboelastography in the assessment of bleeding following surgery for congenital heart disease.

Background: Perioperative bleeding is common in pediatric cardiac surgery patients. Traditional laboratory tests do not adequately characterize coagulation derangements in patients with bleeding. We sought to establish preoperative thromboelastography parameters in children prior to cardiopulmonary bypass, to compare thromboelastography assessment with standard coagulation parameters postoperatively, and to assess thromboelastography in children with significant hemorrhage. Methods: Sixty patients requiring cardiopulmonary bypass were enrolled in a prospective observational study of perioperative thromboelastography. Thromboelastography measures were obtained preoperatively, intraoperatively after protamine administration, upon admit to the intensive care unit, and when patients were treated for bleeding. Thromboelastography measures were not used for clinical care. Postoperative thromboelastography measurements were compared with the standard coagulation parameters. Intraoperative thromboelastography, postoperative thromboelastography, and clinical outcomes were compared among patients who did and did not have significant postoperative bleeding. Results: Preoperative thromboelastography parameters were similar to other published normal values for pediatric patients. Transfusion recommendations based on thromboelastography measurements were significantly different from those based on the standard coagulation testing. Thromboelastography measures after initial protamine administration were significantly different in patients with postoperative bleeding. This difference was not present upon arrival to the intensive care unit. Patients with significant bleeding tended to cease bleeding when clinical interventions were in agreement with recommendations based on thromboelastography. Conclusions: Pediatric patients with significant postoperative bleeding after surgery are more likely to have abnormal thromboelastography early after cessation of cardiopulmonary bypass. Thromboelastography illustrates derangements in the coagulation system and may aid in the treatment of postoperative bleeding.

Pediatric Solid Organ Transplant Recipients: Transition to Home and Chronic Illness Care

Pediatric SOT recipients are medically fragile and present with complex care issues requiring high‐level management at home. Parents of hospitalized children have reported inadequate preparation for discharge, resulting in problems transitioning from hospital to home and independently self‐managing their childs complex care needs. The aim of this study was to investigate factors associated with the transition from hospital to home and chronic illness care for parents of heart, kidney, liver, lung, or multivisceral recipients. Fifty‐one parents from five pediatric transplant centers completed questionnaires on the day of hospital discharge and telephone interviews at three wk, three months, and six months following discharge from the hospital. Care coordination (p = 0.02) and quality of discharge teaching (p < 0.01) was significantly associated with parent readiness for discharge. Readiness for hospital discharge was subsequently significantly associated with post‐discharge coping difficulty (p = 0.02) at three wk, adherence with medication administration (p = 0.03) at three months, and post‐discharge coping difficulty (p = 0.04) and family management (p = 0.02) at six months post‐discharge. The results underscore the important aspect of education and care coordination in preparing patients and families to successfully self‐manage after hospital discharge. Assessing parental readiness for hospital discharge is another critical component for identifying risk of difficulties in managing post‐discharge care.