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Biomedical subjects

Leslie E Lehmann

Publications and source records attributed to Leslie E Lehmann.

6 recordsLinked to original sources

Transverse myelitis after therapy for primitive neuroectodermal tumors.

Traditional therapy for malignant primitive neuroectodermal tumors in children includes surgery, multi-agent chemotherapy, and radiation. Given the poor prognosis with conventional therapy alone, newer treatment approaches have incorporated high-dose chemotherapy followed by autologous stem cell rescue. Treatment with chemotherapy and radiation is not without unanticipated and unwanted side effects. Specifically, radiation-induced damage to the central nervous system can occur, though the frequency is thought to be acceptably low. This report describes two cases of treatment-related transverse myelitis in patients who received induction chemotherapy and craniospinal irradiation followed by high-dose chemotherapy with autologous stem cell rescue. Other patients treated with a similar strategy but different sequence and timing of treatment did not experience symptoms of myelitis, suggesting that the specific timing of radiation in relationship to the chemotherapy may be of critical importance.

Adolescent↗

A novel pattern of transaminase elevation associated with autologous transplant for neuroblastoma.

To determine the pattern and degree of hepatic transaminitis experienced by children undergoing autologous transplantation for neuroblastoma. Sixty-four children with high-risk neuroblastoma received an autologous transplant with cyclophosphamide, etoposide, and carboplatin conditioning. Forty-eight went on to receive a second transplant with M and TBI conditioning. Charts were reviewed for evidence of hepatic regimen-related toxicity. A high rate of transaminitis was observed after both regimens. In each transplant, there was an early period of transaminitis during conditioning, from which patients recovered, followed by a second period of transaminase elevation. The degree of elevation was not associated with age, whether the administered dose was calculated based on a per kg or per M(2) basis or the presence of regimen-related severe mucositis. Elevated transaminases at admission were not associated with maximal hepatotoxicity during the first transplant although there was an association in the second transplant. However, the magnitude of transaminase elevation was less in the second transplant. VOD occurred in one and three patients in transplants 1 and 2, respectively. Both conditioning regimens were associated with an early and late elevation of transaminases without significant cholestasis. This biphasic pattern of transaminitis has not been reported previously. The high prevalence of transaminase elevation at time of both transplants was not associated with an increased incidence of VOD. We conclude that elevated transaminases should not preclude proceeding to a first or second autologous transplant with these regimens.

Antineoplastic Combined Chemotherapy Protocols↗

Oral chronic graft-versus-host disease in pediatric patients after hematopoietic stem cell transplantation.

Chronic graft-versus-host disease (cGVHD) is a serious and potentially life-threatening complication of hematopoietic stem cell transplantation. This study, which is the largest single-center series of oral disease in pediatric patients with cGVHD, describes the oral findings in 49 consecutive patients seen in a pediatric multidisciplinary cGVHD clinic. All consecutive patients seen at the multidisciplinary pediatric hematopoietic stem cell transplantation/cGVHD clinic at the Dana-Farber Cancer Institute (Boston, MA) from July 2001 through October 2003 were included in this study. Subjective and objective assessments of mucosal, salivary gland, and sclerotic pathology were performed for each patient, and specific therapy was initiated when indicated. Oral mucosal cGVHD was identified in 22 (45%) of 49 patients. Only 4 (8%) of 49 patients reported mouth pain, and all patients reported being able to eat well. All patients who required specific therapy for their oral mucosal cGVHD (45%) were already taking at least 1 immunomodulatory agent; however, efficacy of treatment was difficult to assess because of inconsistent follow-up periods. Subjective and objective salivary gland and sclerotic disease were observed far less often. Oral mucosal pathology is common in these patients, and appropriate diagnosis and management of oral lesions is critical to reduce patient morbidity and to improve quality of life. The apparent lack of salivary gland involvement was notable. Developing validated age-appropriate evaluation strategies and identifying effective treatment guidelines will be invaluable in the future management of these patients.

Adolescent↗

A multiagent strategy to decrease regimen-related toxicity in children undergoing allogeneic hematopoietic stem cell transplantation.

Regimen-related toxicity (RRT) is a frequent cause of morbidity and mortality after allogeneic hematopoietic stem cell transplantation (HSCT). In this pilot study, we examined the feasibility and potential efficacy of administering a fixed combination of agents as a novel approach to reducing RRT in children undergoing HSCT. Thirty-seven patients were treated with ursodeoxycholic acid, folinic acid, vitamin E, and parenteral nutrition titrated to measured energy expenditure in the peritransplantation period. Outcomes were compared with those in historical controls (n = 131). Compliance with oral ursodeoxycholic acid and vitamin E of at least 90% was achieved in a mean of 86% (95% confidence interval, 75%-97%) of patients. In the study group, we observed (1) reduced prevalence and severity of mucositis (P = .008 and.004, respectively); (2) less severe hepatic toxicity (P = .007); and (3) shorter time to engraftment (P = .02) compared with the control group. These benefits appeared most pronounced among high-risk patients. The administration of this regimen, including oral medications, is feasible during the peritransplantation period, and it is well tolerated. The decreased RRT observed in comparison to historical controls suggests that combination approaches deserve exploration as a means of reducing the morbidity of HSCT.

Adolescent↗

Dyskeratosis congenita vs. chronic graft versus host disease: report of a case and a review of the literature.

Dyskeratosis congenita (DC) is a rare inherited bone marrow failure syndrome characterized by the triad of reticular pigmentation of the skin, nail dystrophy, and mucosal leukoplakia. DC is often associated with severe pancytopenia, and bone marrow failure is the principal cause of early mortality. Malignant transformation of mucosal leukoplakias may also occur. Rarely, aplastic anemia precedes the other clinical manifestations of the disease. We present a case of a 13-year-old boy who was diagnosed at age four with idopathic aplastic anemia, was treated successfully with an allogeneic bone marrow transplant, then subsequently developed skin, nail, and tongue lesions. While the initial impression was chronic graft-versus-host disease, additional work-up confirmed the diagnosis of DC.

Adolescent↗