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C Vermylen

Publications and source records attributed to C Vermylen.

At least 19 recordsLinked to original sources
PubMed2006

[Medical treatment of juvenile hemangiomas].

Infantile hemangioma always involute in 5 to 7 years. However, 10% of proliferating hemangiomas will necessitate a therapeutic approach, often medical, in order to avoid life or organ threatening, fonctional or esthetic sequelae. "Which hemangioma need to be treated, when and how" are important questions for the optimal management of infantile hemangiomas. Corticotherapy is still the treatment of choice for these lesions. Other anti-angiogenic molecules have also been successfully used such as interferon alfa-2a and vincristine. This chapter tries to answer these questions and detail the different medical modalities for the treatment of infantile hemangioma.

Adrenal Cortex Hormones↗
PubMed2006

Inadvertent anticoagulation of a haemophiliac child with routine line flushing.

We report the case of a 3-year-old boy with severe haemophilia A presenting with recurrent haemarthroses despite daily infusions of factor VIII delivered through a central venous access device (CVAD). Regular rinsing of the CVAD with heparin, according to a standard protocol, resulted in systemic anticoagulation, as demonstrated by prolonged thrombin time and therapeutic anti-Xa levels. The bleeding symptoms resolved after replacing heparin with a normal saline solution. This case illustrates that heparin administered to maintain CVAD patency should be used with caution in young haemophiliacs. Prolonged thrombin time should alert the physician to this possible CVAD complication.

Anticoagulants↗
PubMed2005

Multi-centre pilot study of 2-chlorodeoxyadenosine and cytosine arabinoside combined chemotherapy in refractory Langerhans cell histiocytosis with haematological dysfunction.

The aim of this study was to assess the efficacy and adverse effects of 2-chlorodeoxyadenosine (2-CdA) and cytosine arabinoside (Ara-C) in children with refractory Langerhans cell histiocytosis (LCH) and haematopoietic dysfunction. Ten patients, with a median age at diagnosis of 0.5 years, were enrolled in this study. Treatment comprised at least two courses of Ara-C (1000 mg/m(2)/d) and 2-CdA (9 mg/m(2)/d) administered for 5d every 4 weeks; subsequent median follow-up was 2.8 years (range 0.03-6.4 years). Among the 7 patients who received at least two courses of therapy, disease activity decreased in 6 patients, and control of disease was achieved in all patients after a median delay of 5.5 months. All patients suffered World Health Organisation (WHO) grade 4 haematological toxicity. Two septic deaths occurred shortly after administration of the first course of 2-CdA/Ara-C; a third patient was withdrawn from the trial after the first course and subsequently died following haematopoietic stem cell transplantation. This series is small, but we conclude that 2-CdA and Ara-C combined chemotherapy probably has major activity in childhood refractory Langerhans cell histiocytosis.

Antineoplastic Combined Chemotherapy Protocols↗
PubMed2005

[A particular hereditary anemia in a two-month-old infant: elliptocytosis].

We report the case of a 2.5-month-old infant with severe anaemia discovered fortuitously during an acute febrile illness. The patient was admitted because of a septic arthritis of the knee. Initial biology showed a 3.5 g/dl haemoglobin concentration. The anaemia was microcytic and hypochromic, with obvious haemolysis and reticulocytosis. Standard analysis was not contributive. Further investigations allowed the diagnosis of elliptocytosis. The patient was treated by antibiotics, orthopaedic measures and iterative transfusions. Now, 18 months from the initial episode, she is in good health. With this history, we discuss the clinical process facing severe anaemia during infancy and review the particularities of such uncommon congenital anaemia. Elliptocytosis is a haemolytic anaemia caused by congenital anomalies of the erythrocyte membrane. Diagnosis requires morphological studies of the red blood cells on peripheral blood smear. The disease is often overlooked by membrane protein electrophoresis. The condition is heterogeneous concerning clinical, biochemical and genetic aspects. Most of the cases are linked to mutations of the alpha-spectrin gene, in autoassociation regions. Search of spectrin and protein 4.1 genes mutations can confirm the diagnosis but is not routinely performed.

Acute Disease↗
PubMed2001

Five years of experience with hydroxyurea in children and young adults with sickle cell disease.

The short-term beneficial effect of hydroxyurea (HU) in sickle cell disease (SCD) has been proven by randomized studies in children and adults. The Belgian registry of HU-treated SCD patients was created to evaluate its long-term efficacy and toxicity. The median follow-up of the 93 patients registered is 3.5 years; clinical and laboratory data have been obtained for 82 patients at 1 year, 61 at 2 years, 44 at 3 years, 33 at 4 years, and 22 after 5 years. On HU, the number of hospitalizations and days hospitalized dropped significantly. Analysis of the 22 patients with a minimum of 5 years of follow-up confirm a significant difference in the number of hospitalizations (P =.0002) and days in the hospital (P <.01), throughout the treatment when compared to prior to HU therapy. The probabilities of not experiencing any event or any vaso-occlusive crisis requiring hospitalization during the 5 years of treatment were, respectively, 47% and 55%. On HU, the rate per 100 patient-years of severe events was estimated to be 3.5% for acute chest syndrome, 1.2% for aplastic crisis, 0.4% for splenic sequestration; it was 0% for the 9 patients with a history of stroke or transient ischemic attack followed for an average of 4 years. No important adverse effect occurred. Long-term chronic treatment with HU for patients with SCD appears feasible, effective, and devoid of any major toxicity; in patients with a history of stroke, HU may be a valid alternative to chronic transfusion support. (Blood. 2001;97:3628-3632)

Acute Disease↗
PubMed2001

[Fanconi's anemia and molecular biology research].

Fanconi's anemia is a rare autosomal recessive disease characterized by congenital abnormalities, a progressive pancytopenia and a predisposition to cancer. The diagnosis is based on an abnormal increase of spontaneous chromosome breakage, more specifically on a clear-cut increase of chromosome breakage in the presence of bifunctional alkylating agents. Eight complementation groups (A to H) have been defined, and the genes corresponding to four of these groups have been cloned (FANCA, FANCC, FANCF and FANCG). The function of the proteins encoded by the genes of Fanconi's anemia remains unknown. Numerous studies indicate that different cellular processes are probably involved, including DNA repair pathways, apoptosis, cell cycle regulation and oxygen metabolism. Nevertheless, the exact cellular and molecular mechanisms implicated in Fanconi's anemia remain a challenge for fundamental research. The treatment of Fanconi's anemia is also the subject of intense research, bearing principally upon bone marrow transplantation, which is successful in the case of HLA-identical sibling donors, and gene therapy, which is still at a preliminary stage on the clinical level.

Apoptosis↗
PubMed2001

[Bone marrow transplantation in Fanconi's anemia: report of seven cases].

OBJECTS: Follow-up of patients with Fanconi's anemia treated in our unit and review of the literature concerning bone marrow transplantation in Fanconi's anemia. PATIENTS AND METHODS: Ten patients were followed in our unit for 20 years. We summarize their clinical features, treatment and clinical course. RESULTS: Among the ten patients, seven received allogeneic marrow transplantation. Only two patients are still alive. Two transplanted patients died from complications shortly after the transplantation. Three other patients died later after the transplantation, two of them from oropharyngeal carcinomas. DISCUSSION: The 5-year survival is about 70% in the transplantation with an HLA-identical sibling donor; it is only about 30% if the donor is an HLA-matched unrelated or mismatched related patient. Furthermore, retrospective studies have shown that the long-term outcome of carcinoma is a major complication after the transplantation. CONCLUSION: Our series of patients with Fanconi's anemia reflects fairly faithfully the complications encountered in this disease. Although the improvement of the graft technique may decrease the rate of death due to transplantation, the long-term development of solid tumors remains a problem for which no solution has been suggested up to now.

Bone Marrow Transplantation↗
PubMed2001

Unusual evolution of an Epstein-Barr virus-associated leiomyosarcoma occurring after liver transplantation.

We report the case of a child who developed, 2 yr after orthotopic liver transplantation (OLTx) for biliary atresia, a multi-focal hepatic tumor with lymphonodular metastases, identified as an Epstein-Barr virus (EBV)-associated leiomyosarcoma. Chemotherapy was given without tumor response. Subsequently, slow growth of the tumor was observed. Immunosuppression was tapered and stopped 9 yr after transplantation. At the present time, 12 yr after the discovery of the first hepatic lesions, the patient is alive and completely symptom-free, the abdominal masses are stable, and liver function tests are completely normal. Smooth muscle tumors are increasingly recognized in children with various immunodeficiencies occurring after organ transplantation. This unusual evolution of a clinically aggressive tumor into a stable disease after restoration of immunity confirms that the immune status of the patient is a crucial factor.

Biliary Atresia↗
PubMed2001

Smooth muscle tumor developing in an immunocompromised child after therapy for leukemia.

We report a 5.5-year-old boy who underwent autologous peripheral blood stem cell transplantation for high-risk acute lymphoblastic leukemia and who had two abdominal masses develop 6 months later. Macroscopically complete resection of the abdominal tumors was performed and revealed a well-differentiated leiomyosarcoma. Smooth muscle tumors, benign or malignant, are increasingly recognized in children with various immunodeficiencies; the association with acute lymphoblastic leukemia is rarely described.

Abdominal Neoplasms↗
PubMed2001

[Immune deficiencies: diagnosis, management, some perspectives].

Severe primary immunodeficiencies (PID) are rare; their global incidence is comparable to that of childhood leukemia; they include more than 100 different entities. Clinical manifestations are: unusually severe or frequent infections or infections that do not respond to adequate treatment; an increased risk of certain malignancies; sometimes auto-immune manifestations. Delayed diagnosis and management of PID can lead to severe and irreversible complications or to death. PID can become manifest only in the adult; in common variable immune deficiency, the median age at diagnosis is between the 2nd and the 3rd decade of life. PID are often transmitted genetically; recent progresses in molecular biology have allowed more precise and earlier, including antenatal, diagnosis. Molecular treatment of 3 infants with a severe immunodeficiency has recently been achieved in April 2000. Those progresses were mostly based on the study of immunodeficiency databases. We present here the work of a Belgian group specialized in PID; meetings have started in June 1997. This group establishes guidelines for the diagnosis and treatment of PID, adapted to the local situation. The elaboration of a national register of PID is also underway; this has to provide all guaranties of anonymity to patients and families. Such a register already exists at the European level; it has provided the basis for new diagnostic and therapeutic possibilities. The inclusion of Belgian data in this register should allow essential progresses essential for our patients.

Adolescent↗
PubMed2000

Stem cell transplantation in patients with severe congenital neutropenia without evidence of leukemic transformation.

Severe congenital neutropenia (CN) (Kostmann syndrome) is a hematologic disorder characterized by a maturation arrest of myelopoiesis at the promyelocyte/myelocyte stage of development. This arrest results in severe neutropenia leading to absolute neutrophil counts (ANC) below 0.2 x 10(9)/L associated with severe bacterial infections from early infancy. Data on over 300 patients with CN collected by the Severe Chronic Neutropenia International Registry (SCNIR) beginning in 1994 indicate that more than 90% of these patients respond to recombinant human granulocyte-colony stimulating factor (r-HuG-CSF) treatment with an ANC greater than 1. 0 x 10(9)/L. For patients who are refractory to r-HuG-CSF treatment and continue to have severe and often life-threatening bacterial infections, hematopoietic stem cell transplantation is the only currently available treatment. We report on a total of 11 patients with CN reported to the SCNIR who underwent transplantation for reasons other than malignant transformation between 1976 and 1998. Of these patients, 8 were nonresponders or showed only partial response to r-HuG-CSF treatment with ongoing infections. Results from these patients suggest that transplantation of stem cells from an HLA-identical sibling is beneficial for patients refractory to r-HuG-CSF. (Blood. 2000;95:1195-1198)

Adolescent↗
PubMed2000

Outcome of 69 allogeneic stem cell transplantations for Fanconi anemia using HLA-matched unrelated donors: a study on behalf of the European Group for Blood and Marrow Transplantation.

Allogeneic stem cell transplantation is the only treatment that can restore a normal hematopoiesis in Fanconi anemia (FA). In this retrospective multicenter study, we analyzed the results of this approach using HLA-matched unrelated bone marrow donors, and tried to identify covariates predicting the outcome of the transplant. From January 1985 to June 1998, 69 FA patients were transplanted with unrelated HLA-matched donors. Patients' characteristics before and after transplant were provided by the European group blood and marrow transplant registry and were analyzed in collaboration with the European Fanconi Anemia Registry. The 3-year probability of survival was 33%. Extensive malformations, a positive recipient cytomegalovirus serology, the use of androgens before transplant, and female donors were associated with a worse outcome. Primary graft failures were observed more frequently when female donors were used, mainly because the grafts contained lower nucleated cell doses per kilogram of recipient body weight compared with grafts coming from male donors. The probability of grade III-IV acute graft-versus-host disease (GVHD) was 34%. Elevated serum alanine/aspartate transaminases before transplantation; limb, urogenital tract, or nephrologic malformations; and non-T-cell-depleted grafts were predictors of severe acute GVHD. This study shows the dramatic impact of preexisting congenital malformations on the outcome of FA patients transplanted with HLA-matched unrelated donors. If the use of T-cell depletion has led to a dramatic reduction of acute GVHD incidence, no significant outcome improvement was observed with this approach, mainly because of an increased risk of graft failure. (Blood. 2000;95:422-429)

Adolescent↗
PubMed2000

[Perinatal thrombocytopenia of maternal origin].

Maternal causes of perinatal thrombocytopenia include neonatal alloimmune thrombocytopenia, autoimmune disorders, intrauterine infections and hypertensive diseases. The diagnosis of these pathologies is difficult because they can occur in sick neonates, but also in healthy babies without a history suggesting illness. The treatment has to be quickly established in order to decrease eventual hemorrhagic complications. These latter can be amplified by several clinical circumstances and especially by the platelet immaturity of this time of life. The risk of recurrence for the next pregnancies has to be determined and can lead to diagnostic or therapeutic measures during the antenatal period.

Adult↗
PubMed2000

Clonal monosomy 7 and 5q--in a child with myelodysplastic syndrome.

The authors report the case of a 5-year-old boy referred for thrombocytopenia and neutropenia. Bone marrow examination showed a myelodysplasia with clonal monosomy 7. The acceleration of the disease was marked by the appearance of an additional cytogenetic abnormality, i.e., the deletion of the long arm of chromosome 5 in the clonal cells. RAS gene mutation was not detected. Chemotherapy was started to achieve complete remission before a bone marrow transplantation. This treatment was complicated by a prolonged aplasia and the patient died of systemic mycotic infection.

Child, Preschool↗
PubMed1999

Ferritin-associated iron induces neutrophil dysfunction in hemosiderosis.

Neutrophils (PMNs) from patients with secondary iron overload have an increased iron and ferritin content as well as a phagocytosis defect. Several serum components might be incriminated in the cellular iron accumulation. We therefore compared the effects on the PMN phagocytosis of total serum as well as the ferritin and transferrin fractions of serum derived from patients with thalassemia major and healthy control subjects. An incubation system of PMNs was developed. PMN phagocytosis was measured before and after incubation. Total serum from patients with thalassemia induced a defect that was prevented by co-incubation with deferoxamine (DFO). Gel-filtration chromatography was performed to separate the serum fraction containing transferrin and albumin from that containing ferritin. The transferrin-albumin fraction had no effect on PMN phagocytosis. On the contrary, the ferritin fraction of normal serum was deleterious to PMN phagocytosis, and the same fraction from thalassemic serum decreased PMN phagocytosis even more. Co-incubation with DFO or catalase improved this defect. Moreover, a cellular increase in the L-type subunit of ferritin was observed after the incubation of PMNs with the ferritin-containing fraction from thalassemic serum. In conclusion, serum from patients with thalassemia is toxic to PMNs, and this toxicity is due to ferritin-associated iron.

Adolescent↗
PubMed1999

The belgian experience in unrelated donor bone marrow transplantation: identification of center experience as an important prognostic factor.

BACKGROUND AND OBJECTIVE: We reviewed all unrelated donor bone marrow transplants (UDBMT) performed in Belgium up to December 1995 to identify prognostic factors for relapse, transplant-related mortality and survival. DESIGN AND METHODS: A total of 163 UDBMT were performed in 92 males and 71 females aged 1-55 (median 26) years. Patients were transplanted for ALL (n=35), AML (n=34), CML (n=51), other myeloid malignancies (n=14), SAA (n=21) or miscellaneous other diseases (n=8). Most patients had advanced disease; a few patients were in CR1 (n=10) or early chronic phase (CP) of CML (n=5). RESULTS: Overall survival at 5 yrs was 17% (95% confidence interval: 8-32%), but survival was significantly better for patients with non-malignant disorders (55% at 4 yrs). The relapse rate +/-SE was projected to be 40 (28-54)% at 5 yrs, 36 (20-56)% for standard-risk and 68 (43-85)% for high-risk malignancies (p=0.0029). There was no relapse in CML patients transplanted in 1st CP compared to 68% at 4 yrs with more advanced CML (p=0.0033). Grade II-IV acute graft-versus-host disease (aGVHD) occurred in 55% by day 100 and was strongly modulated by age, ranging from 41% in <20-yr-old to 80% in >40-yr-old patients (p=0. 0021). Transplant-related mortality (TRM) was projected to be 72 (52-87)% at 5 yrs including 2 very late deaths from lung fibrosis and secondary cancer. Main causes of death were original disease in 27, secondary malignancy in 2, GVHD in 28, interstitial pneumonia in 21, other infections in 19, and miscellaneous toxic causes in 21 patients. In multivariate analysis, the relapse rate was strongly dependent on the disease status (p=0.0029), TRM being significantly worse with older age (p=0.0049), and overall survival being significantly worse in more advanced disease (p=0.0006), after a second transplant (p=0.0166), in centers of smaller size (p=0.0316) and in older patients (NS). INTERPRETATION AND CONCLUSIONS: Although results have improved somewhat in recent years, UDBMT remains a procedure with a high TRM. UDBMT should be performed in patients with less advanced diseases and in centers with more experience, particularly in the treatment of adult patients.

Adolescent↗