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11. |
Early Bone Marrow Transplantation in an Infant with Wiskott‐Aldrich Syndrome |
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American Journal of Pediatric Hematology/Oncology,
Volume 13,
Issue 3,
1991,
Page 310-314
Lorraine Beard,
Ian Toogood,
Christopher Pearson,
Antonio Ferrante,
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摘要:
The Wiskott-Aldrich Syndrome (WAS) is a rare X-linked immunohematological disorder characterized by eczema, profound thrombocytopenia, and progressive immunodeficiency. Severe hemorrhage, overwhelming sepsis, or lymphoreticular malignancy usually cause death in childhood. Recently, bone marrow transplantation (BMT) has been curative in some well-established cases, but there is no general agreement about the place of BMT in infants with WAS before the development of significant immunological abnormalities. We describe the successful use of early histocompatible BMT in a 10-month-old infant in whom WAS was diagnosed on the basis of eczema, thrombocytopenia, small platelets, and raised serum immunoglobulin A (Ig) and IgE, but before the development of immunodeficiency as evidenced clinically by recurrent infections, or immunologically by low serum IgM or consistently abnormal lymphocyte responses to mitogens. After an unstable period for several weeks post-transplantation when he developed marked hepatomegaly and severe interstitial pneumonitis, he made a good recovery. His eczema and thrombocytopenia resolved and he has shown no clinical or laboratory evidence of immunodeficiency. It is now over 2 years since his BMT. Because of the poor prognosis of WAS, where a histocompatible donor is available, BMT at the earliest opportunity, despite the inherent risks of such a procedure, may be the best option for an infant with WAS.
ISSN:0192-8562
出版商:OVID
年代:1991
数据来源: OVID
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12. |
Recurrent Disseminated Retinoblastoma Treated by High‐dose Chemotherapy, Total Body Irradiation, and Autologous Bone Marrow Rescue |
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American Journal of Pediatric Hematology/Oncology,
Volume 13,
Issue 3,
1991,
Page 315-319
Ulla Saarinen,
Hannu Sariola,
Liisa Hovi,
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摘要:
A case of recurrent, disseminated retinoblastoma is presented. The primary intraocular tumor, a metastatic mass at recurrence, and the tumor cells infiltrating bone marrow were all positive for the anti-GD2 monoclonal antibody (Mab) 3A7. Indirect immunofluorescence using the monoclonal antibody 3 A7 was an effective method of detecting residual disease in the marrow. After remission was achieved by conventional therapy, the patient underwent autologous bone marrow transplantation (ABMT). The preparative regimen consisted of VP-16, cisplatinum, high-dose melphalan, and total body irradiation. The autologous marrow inoculum was clean of tumor cells at the detection level of 1:10,000. The transplant course was uneventful, and the patient is well and disease-free 17 months after ABMT. We conclude that high-dose chemotherapy and total body irradiation in an ABMT setting is feasible and a potentially curative approach to disseminated retinoblastoma.
ISSN:0192-8562
出版商:OVID
年代:1991
数据来源: OVID
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13. |
Erythroblastopenia in Two Patients after Splenectomy and Polychemotherapy |
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American Journal of Pediatric Hematology/Oncology,
Volume 13,
Issue 3,
1991,
Page 320-325
Cornelia Domeyer,
Ursula Schulte-Overberg,
Irene Boll,
Eckard Baumgarten,
Christoph Bührer,
Rüdiger Fengler,
Günter Henze,
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ISSN:0192-8562
出版商:OVID
年代:1991
数据来源: OVID
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14. |
Danazol Therapy for Chronic Immune‐Mediated Thrombocytopenic Purpura in a Patient with Common Variable Immunodeficiency |
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American Journal of Pediatric Hematology/Oncology,
Volume 13,
Issue 3,
1991,
Page 326-329
Frank Saulsbury,
Karen Bringelsen,
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摘要:
We describe a patient with common variable immunodeficiency (CVI) and chronic immune-mediated thrombocytopenic purpura (ITP), refractory to treatment with corticosteroids, splenectomy, and intravenous immunoglobulin. He had a prompt response to danazol, and the platelet counts have been maintained in the normal range with low-dose, alternate-day, danazol therapy. This case provides evidence that danazol is effective in the treatment of chronic ITP in patients with immunodeficiency and indicates that some patients can be maintained on an alternate-day regimen.
ISSN:0192-8562
出版商:OVID
年代:1991
数据来源: OVID
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15. |
Deep Venous Thrombosis in a Child with Nephrotic Syndrome Associated with a Circulating Anticoagulant and Acquired Protein S Deficiency |
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American Journal of Pediatric Hematology/Oncology,
Volume 13,
Issue 3,
1991,
Page 330-333
Frederick Garbrecht,
Sharon Gardner,
Valerie Johnson,
Eric Grabowski,
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摘要:
Thromboembolic events occur with a frequency of 3–5% in children with nephrotic syndrome (NS). Although numerous abnormalities in all phases of coagulation have been described in NS, the pathogenesis of clotting abnormalities remains poorly understood in this group of patients. We describe a child with long-standing NS in whom a severe deep venous thrombosis and pulmonary embolism secondary to acquired protein S deficiency and a strong lupus-type circulating anticoagulant developed. In addition, this patient had a markedly decreased plasma level of C4b binding protein. Although acquired protein S deficiency has been described in various clinical disorders including NS, our patient is unusual in having C4bBP deficiency, and his is the only reported pediatric case of NS complicated by thromboembolism in which a circulating anticoagulant has been implicated, to our knowledge.
ISSN:0192-8562
出版商:OVID
年代:1991
数据来源: OVID
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16. |
Childhood Ki‐1 LymphomaPresentation as a Buttock Mass |
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American Journal of Pediatric Hematology/Oncology,
Volume 13,
Issue 3,
1991,
Page 334-337
Stuart Winter,
Marilyn Duncan,
Elliott Foucar,
Thomas McConnell,
Karen Cartwright,
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摘要:
Ki-1 lymphoma is a rare, large-cell anaplastic non-Hodgkin's lymphoma that most commonly affects older children and young adults. Presentation usually occurs as a localized infiltration of the skin and lymph nodes. We report an unusual case of childhood Ki-1 lymphoma that presented as a buttock mass in an eight-year-old girl. Pathologic evaluation revealed the characteristic lymphoma cells expressing Ki-1 antigen (CD-30), HLA-DR, interleukin 2 (CD-25), T-cell gene rearrangement, and the cytogenetic karyotype t(2;5). The patient is in complete remission following treatment with combination chemotherapy. This report broadens the clinical spectrum associated with Ki-1 lymphomas and illustrates the importance of combining routine pathologic examination with other specialized diagnostic techniques in the evaluation of childhood soft-tissue masses.
ISSN:0192-8562
出版商:OVID
年代:1991
数据来源: OVID
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17. |
Monosomy 7 Syndrome in an Infant with Neurofibromatosis |
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American Journal of Pediatric Hematology/Oncology,
Volume 13,
Issue 3,
1991,
Page 338-341
J. Kelleher,
T. Carbone,
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摘要:
A 9-month-old boy with known familial neurofibromatosis type I (NF-1) presented with a clinical and laboratory picture suggestive of juvenile chronic myelomonocytic leukemia (JCMMoL). Chromosomal studies obtained from the bone marrow indicated, however, that he had monosomy 7 syndrome. We believe this is the first reported case of monosomy 7 syndrome in a child with NF in the United States, and that this case complements a recent report of two cases of NF, JCMMoL, and monosomy 7 in Japanese children. Since monosomy 7 syndrome is very difficult to differentiate from JCMMoL or acute nonlymphocytic leukemia (ANLL) unless appropriate chromosomal studies are obtained, we believe it is possible that monosomy 7 may occur with increased frequency in patients with NF-1. Monosomy 7 syndrome might therefore be a significant cause of the known association between NF-1 and nonlymphoid leukemia.
ISSN:0192-8562
出版商:OVID
年代:1991
数据来源: OVID
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18. |
The Potential for Renal Salvage in Nonmetastatic Unilateral Wilms' Tumor |
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American Journal of Pediatric Hematology/Oncology,
Volume 13,
Issue 3,
1991,
Page 342-344
Judith Wilimas,
Lynn Magill,
David Parham,
Mahesh Kumar,
Edwin Douglass,
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摘要:
The potential for resection with renal salvage, and the ability of computed tomographic (CT) criteria to predict this potential, were evaluated retrospectively in 43 children with nonmetastatic unilateral Wilms' tumor (stage I, II, or III at diagnosis). CT criteria for renal salvage were (a) tumor involving only one pole and occupying less than one third of the kidney; (b) functioning kidney; (c) no invasion of collecting system or renal vein; and (d) clear margins between tumor, kidney, and surrounding structures. Preoperative CT scans met these criteria in only two of the 43 cases. Surgical and/or pathologic reports confirmed CT-based findings in all cases. Most of the tumors were large (median vertical and transverse diameters both = 10 cm), and only three patients were estimated to have more than 50% functioning renal tissue. A prospective trial would be necessary to evaluate the long-term benefits and possible complications of renal salvage procedures in patients with Wilms' tumor. Given the small proportion of patients likely to be eligible for such procedures, and the currently excellent cure rates in low-stage disease, such as trial would be difficult to implement.
ISSN:0192-8562
出版商:OVID
年代:1991
数据来源: OVID
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19. |
Sideroblastic Anemia Showing Unique Response to Pyridoxine |
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American Journal of Pediatric Hematology/Oncology,
Volume 13,
Issue 3,
1991,
Page 345-350
Ryusuke Murakami,
Touru Takumi,
Junko Gouji,
Hajime Nakamura,
Masao Kondou,
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摘要:
We treated and followed up for 6 years a patient with pyridoxine-responsive sideroblastic anemia. The patient was a boy age 1 year and 9 months, who was diagnosed on the basis of peripheral red cell morphology and an increased number of sideroblasts in the bone marrow. Bone marrow erythroblasts showed a marked reduction of delta-aminolevulinic acid synthase (ALA-S) activity. The response of the patient to pyridoxine and its active form, pyridoxal phosphate, was unique. After the first course of pyridoxal phosphate therapy (300 to 500 mg/day i.v. for 4 days), all hematological data were restored to normal and remained normal for 29 months without the further administration of pyridoxal phosphate. The second course of pyridoxal phosphate therapy (500 mg/day i.v. for 2 days) was effective for 6 months. The third, fourth, and fifth courses of the therapy consisted of daily oral pyridoxine hydrochloride at a dose of 180 mg/day for 4 to 6 weeks, and the respective periods of hematological remission were 7, 12, and >18 months. These observations suggest the presence of a complicated ALA-S activating or inactivating system, or both, in our patient.
ISSN:0192-8562
出版商:OVID
年代:1991
数据来源: OVID
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20. |
Clinical Research UpdateUse of Recombinant Erythropoietin in Treatment of the Anemia of Prematurity |
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American Journal of Pediatric Hematology/Oncology,
Volume 13,
Issue 3,
1991,
Page 351-363
Daniel Halperin,
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摘要:
The anemia of prematurity is defined by a progressive decline in hemoglobin level occurring over the first 2 months of life. Unlike term newborns whose “physiologic anemia” rarely if ever necessitates any treatment, preterm infants may become anemic enough to have clinical symptoms that indicate a need for red blood cell transfusions. Various factors contribute to the development of this anemia. Some of these factors, such as the short life span of erythrocytes in preterm infants, increased sensitivity of the erythrocytes to oxidative injury, and the blood losses caused by repeated phlebotomies, would normally be expected to induce corrective reticulocytosis. Characteristically, however, this anemia is hyporegenerative. Thus, it is associated with relative reticulocytopenia, low serum erythropoietin levels, and bone marrow erythroid hypoplasia. The recent availability of recombinant human erythropoietin has opened new perspectives in the management of a variety of anemias. Based on current knowledge of the regulation and patho-physiology of fetal and neonatal erythropoiesis, recombinant erythropoietin may represent a logical and efficient alternative to giving red blood cell transfusions in the treatment of the anemia of prematurity. Clinical trials have been initiated in several countries using different approaches and methodology. At this early stage these trials do not yet fully affirm that recombinant erythropoietin can be used as the first-line therapy in infants with the anemia of prematurity. Our own observations, however, suggest that this agent is well tolerated by preterm infants and may exert a corrective effect on the anemia of prematurity.
ISSN:0192-8562
出版商:OVID
年代:1991
数据来源: OVID
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