Allogeneic stem cell transplantation in patients with ß-thalassemia: King Faisal specialist hospital and research centre experience.

From January 1998-July 2006, 62 stem cell transplantation (SCT) were performed on 60 patients with beta-thalassemia from HLA-related match donors. The overall survival (OS) and event free survival (EFS) for all patients were 94 and 77%. The outcome of allogeneic SCT in our experience is satisfactory with OS 92% and EFS 77%. Transplantation at a young age and when the disease is mild offers the best outcome. More advanced disease is associated with higher rate of rejection and severe graft versus host disease.

Failure to attend appointments and loss to follow-up: a prospective study of patients with malignant lymphoma in Riyadh, Saudi Arabia.

Failure to attend medical appointments (No Show) and loss to follow-up (LTFU) among patients with cancer can adversely affect their treatment and eventual outcome. In a 3-year prospective study of 199 patients with malignant lymphoma, all of those with No Shows were contacted, and reasons given for No Shows were categorized. Of the 340 No Shows, 34.1% were due to hospital-based communication problems, 17.6% to errors in patient communication with the hospital, 7.4% to transportation problems and 16.5% to other personal reasons. Almost one quarter (24.4%) of the patients were not contactable. Reasons for No Show in all categories were instructive as to patients' attitudes to treatment. Nineteen (12.2%) of the 156 patients who had not died in the 3-year follow-up period were identified as LTFU. These 19 LTFU patients accounted for 77 (22.6%) of all No Shows. The data indicate that LTFU in this cohort is significantly less frequent than in a prior cohort followed up for 3 years from 1997 to 1998. These findings suggest that some causes of No Show can be addressed, and individuals are identified as at particular risk for No Show and ultimately LTFU. This study points out that pre-emptive strategies to reduce No Shows may be feasible and efficacious.

Loss to follow-up of patients with malignant lymphoma.

Loss to follow-up (LTFU) in cancer patients is a serious problem, yet there is little data on this and on the underlying reasons. Of 144 paediatric and 431 adult patients with lymphoma diagnosed in 1997/1998 at King Faisal Specialist Hospital and Research Center, Riyadh (KFSHRC), 30% and 48.5%, respectively, were LTFU after 4 years (excluding patients known to have died). In 2001-2002, 196 paediatric and adult lymphoma patients at KFSHRC were enrolled in a prospective study in which explanations were obtained in detail for non-attendance at follow-up appointments (No Show). Sixteen months after commencement of the study, 49 patients were No Show, because of patient-based communication problems (20), transportation problems (8), patient not contactable (18), and personal reasons (3). In addition, patients were recorded incorrectly as No Show through hospital/patient communication problems. The No Show patients, especially the 23 who failed to keep a second appointment, are identifiable as potential LTFU during the 3 years in which this cohort will be followed. This study and, we suggest, other studies on LTFU should stimulate interest in this issue, in the predisposing factors, and in strategies to address them.

Assessment and comparison of intraoperative and postoperative pain associated with NovaSure and ThermaChoice endometrial ablation systems.

STUDY OBJECTIVE: To assess and compare intraoperative and postoperative pain associated with NovaSure impedance-controlled endometrial ablation system and ThermaChoice system. DESIGN: Prospective, international, multicenter, double-arm study (Canadian Task Force classification II-1). Setting. Academic medical centers and private offices. PATIENTS: Sixty-seven premenopausal women with menorrhagia. INTERVENTION: Endometrial ablation with either the NovaSure (37 women) or ThermaChoice (30) system. NovaSure-treated patients received no endometrial pretreatment; those treated with ThermaChoice received the recommended 3-minute suction dilatation and curettage. MEASUREMENTS AND MAIN RESULTS: Standard pain measurement instruments (visual analog scale, numeric rating scale) were used to assess intraoperative and postoperative pain. Serum levels of prostaglandin-F(2alpha) were measured before and 5, 30, and 60 minutes after the procedure. Patients treated with the NovaSure system reported statistically significantly lower intraoperative and postoperative pain than those treated with the ThermaChoice system (p <0.0001). Procedure time was statistically significantly shorter with the NovaSure system (p <0.0001). Prostaglandin-F(2alpha) values did not differ statistically between groups. CONCLUSION: The NovaSure system is associated with statistically significantly lower intraoperative and postoperative pain than ThermaChoice system, and endometrial ablation with NovaSure could become an office-based procedure.

Primary T-cell lymphoma of the brain in children: a case report and literature review.

Described here are the clinical features and results of treatment in a 10-year-old Saudi Arabian girl with primary T-cell lymphoma of the central nervous system. At presentation the patient had nystagmus and ataxia. The diagnosis was established by tissue biopsy obtained from the cerebellum. Therapy included cranio-spinal irradiation and combination chemotherapy of a systemic high dose of methotrexate, cytosine, arabinoside, and L-asparaginase. Remission was obtained easily but was interrupted by a local intracranial relapse 57 months after diagnosis (37 months after cessation of therapy; at present the patient is still alive and receiving palliative treatment). This report is warranted because of the rarity of this condition in children.

Extramedullary myeloid tumors in children: the limited value of local treatment.

PURPOSE: To determine the incidence of extramedullary tumors (EMT) in Saudi Arabian children with acute myeloid leukemia, the factors associated with these tumors and the impact of local treatment on local tumor control, complete remission and survival rates. PATIENTS AND METHODS: One hundred children, median age 6 years, who received their primary treatment for acute myeloid leukemia at King Faisal Specialist Hospital and Research Center, from 1983 to 1997 were studied. EMT at diagnosis occurred in 18 (18%) patients at 25 sites. Meningeal leukemia, hepatosplenomegaly, lymph node enlargement, gingival hypertrophy, and cutaneous infiltration were not included in the definition of EMT. With these exclusions, children with EMT were younger than those without EMT (median age, 3.5 v. 7.5 years) and were more likely to have meningeal leukemia at diagnosis (33% v. 10%). The t(8;21) translocation was associated with a 47% EMT incidence compared with 23% without the translocation. Local radiation treatment was given to 16 of 25 (64%) EMT sites. RESULTS: The overall 5-year survival rate for all patients was 28%, and this was not significantly influenced by the drug regimen used, meningeal leukemia at diagnosis, the presence of the (8;21) translocation, M4 and M5 morphology combined, or EMT at diagnosis. Significant differences were observed in the 5-year survival rates for patients who underwent allogeneic bone marrow transplantation (52%; N = 37) and those who attained complete remission (CR) but did not undergo transplantation (21%; N = 44) and those who did not achieve complete remission with initial therapy (5%; N = 19). Systemic and local EMT CR was achieved in 17 of 18 patients with EMT, including 12 patients who underwent radiation treatment and 5 of 6 of those who did not. Isolated relapse was not seen at an EMT site and was not noted at any later stage of the disease. CONCLUSIONS: Permanent local control at sites of EMT was achieved in all patients who attained a bone marrow CR, whether or not the site was irradiated. Local radiation treatment of an EMT site did not appear to contribute to overall CR and survival rates. The use of radiation treatment should be conservative and limited to patients in whom there is a real and immediate threat to vision or renal function or when the spinal cord is compromised.

Primary unilateral renal lymphoblastic lymphoma.

A 4-year-old boy presented with a right-sided mass. A lobulated tumour in the right flank was felt on palpation, and radiological investigation revealed a solid, multinodular intrarenal tumour. On biopsy the tumour was found to be a lymphoblastic lymphoma.

Pediatric cancer: The King Faisal Specialist Hospital and Research Centre experience.

Between January 1976 and December 1993, a total of 3291 children with cancer were treated at King Faisal Specialist Hospital and Research Centre (KFSH&RC). Males accounted for 60.7% and females 39.3%, with a ratio of 1.5:1. The peak age was two to five. The three most common malignancies were leukemias (26.2%), lymphomas (21.3%), and central nervous system (CNS) tumors (15.3%). This report presents some of the epidemiologic data related to the largest number of children with malignancy treated in a single institution in Saudi Arabia.

Small round cell tumor with divergent differentiation: cytologic, histologic, and ultrastructural findings.

A case study of desmoplastic small round cell tumor with divergent differentiation in a 13-yr-old boy is presented. Clinical, cytologic, histologic, immunohistochemical, and ultrastructural findings are reported. There was a close correlation between the various histologic and cytologic patterns. Immunohistochemical studies revealed strong positivity of many of the tumor cells for cytokeratin, vimentin, and desmin. Ultrastructurally, some of the tumor cells contained aggregates of cytoplasmic intermediate filaments. Fine-needle aspiration biopsy may suggest the diagnosis of desmoplastic small round cell tumor. Confirmation of the diagnosis may be provided by immunohistochemistry and electromicroscopy.

Fine-needle aspiration biopsy of Langerhans histiocytosis (histiocytosis-X).

A series of 14 fine-needle aspiration biopsies (FNAB) from histologically proven cases of histiocytosis-x (Hx) were reviewed. The smears revealed a variable mixture of Langerhans cells, eosinophils, macrophages, polymorphonuclear cells, and giant cells. Based on the predominant cells present, the cases were further categorized as Langerhans cell predominant (nine cases), eosinophil predominant (two cases), and macrophage predominant (three cases). Langerhans cells were usually polygonal without significant evidence of phagocytosis and frequently contained indented nuclei. In three cases, several Langerhans cells showed prominent dendritic processes. Electron microscopy in 10 cases revealed Langerhans cells with varying degrees of morphologic differentiation. All cases studied revealed Birbeck granules. These findings indicate that FNAB may be an effective technique for diagnosing Hx.

Chediak-Higashi syndrome: Report on five Saudi Arab children and review of the literature.

Five cases of the Chediak-Higashi Syndrome (CHS) among Saudi Arab children were diagnosed between June 1978 and December 1990. All patients were males, ages 18 months to ten years, born to first degree consanguineous parents. All showed the typical somatic and laboratory characteristics of this syndrome with characteristic hyperpigmented irides in four patients. Three patients were in the accelerated phase, two of whom responded to a combination chemotherapy (vincristine and prednisone) and one improved after splenectomy. All patients died before they passed the first decade of their lives. This report is the first detailed description of CHS among Arab children in the Arabian peninsula.

Partial albinism, immunodeficiency, and progressive white matter disease: a new primary immunodeficiency.

We studied 12 children who presented with a recently recognized syndrome. The salient features of this new syndrome were recurrent fever; hepatosplenomegaly; pancytopenia; blond, golden to silvery gray hair; hypopigmented skin, progressive white matter demyelination; and early death. Seven patients died, four with severe central nervous system (CNS) involvement, and three with bone marrow failure and sepsis. Cutaneous anergy to recall antigens was present in all patients. Other immunological abnormalities were poor antibody responses; deficient T-cell responses to phytohemagglutinin (PHA), concanavalin A (Con-A), and allogeneic lymphocytes; hyperresponsiveness of B lymphocytes to pokeweed mitogen; and variable phagocytic defects. Histopathologic examination of the hair and skin biopsies showed characteristic distribution of melanin with melanocytes present in normal numbers but with fewer short dendritic processes. Langerhans' cells were present in normal numbers in some patients and sparse in others. This syndrome seems to cluster into two tribes from two different geographical areas in the Arabian Peninsula. In the eight families studied, 12 other siblings and close relatives were found to be affected. The mode of inheritance in this syndrome is that of an autosomal recessive pattern. We propose the term "PAID syndrome" to identify patients with the above features.

Histiocytosis-X in Saudi children.

A retrospective analysis of 21 Saudi children with Histiocytosis-X were reviewed. 13 were males, 8 were females, with a ratio of 1.62:1. Five had unifocal disease while 16 were with multifocal disease. Bone involvement was seen in 19 patients (90.4%), and three patients had organ dysfunction. One patient had family history of Histiocytosis-X. Follow-up was from 1-10 years (Median 3 years). Three patients died, three had recurrences and eight patients had various disabilities (38%). Overall disease free survival was 84.2 percent.

Aspiration cytology of Wilms' tumor: correlation of cytologic and histologic features.

We examined the cytomorphologic features of fine-needle aspiration biopsy (FNAB) specimens from 23 Wilms' tumor patients. The findings were correlated with histopathologic patterns from these tumors. The study revealed a close resemblance between the cytologic and histopathologic appearance of various cellular elements in Wilms' tumors. The major cellular patterns seen in Wilms' tumor include blastemal cells, blastemal cells with epithelial differentiation, blastemal cells with tubular differentiation, and stromal elements. It is hoped that recognition of these cellular components in aspiration smears will be helpful in establishing an FNAB diagnosis of Wilms' tumor.

Endodermal sinus (yolk sac) tumor in infants and children. A clinical and pathologic study: an 11 year review.

We reviewed the clinical features, treatment, and results of children with gonadal and extragonadal yolk sac (endodermal sinus) tumors seen in the King Faisal Specialist Hospital and Research Centre between 1976 and 1987. There were nine children (seven girls and two boys) with ages ranging from 7 months to 12 years (median of 3.5 years). Sites of origin included the vagina (two cases), face (two cases), sacrum (two cases), mediastinum (one case), ovary (one case), and testicle (1 case). All children had elevated alpha-fetoprotein (AFP) at diagnosis. One girl had complete surgical excision of an ovarian tumor at the time of diagnosis, and one boy had surgical excision of the testis. In the remaining seven children, the tumor was unresectable. Surgery was limited to a biopsy in six children. All patients received different combinations of chemotherapy, including vincristine (VCR), actinomycin D (Act-D), cyclophosphamide (Cyclo), adriamycin (Adria), bleomycin (Bleo), cis-platinum (CDDP), vinblastine (VBL), and VP-16. Of the nine patients, one was lost to follow-up while in remission, five died, one was lost to follow-up, and three are alive and disease-free at 15, 55, and 67 months from diagnosis. This review demonstrates an unusual preponderance of the extragonadal form of endodermal sinus tumor among our patients.

Aspiration cytology of retinoblastoma: light and electron microscopic correlations.

A series of 16 cases of retinoblastoma diagnosed by fine-needle aspiration biopsy (FNAB) and confirmed by histologic examination is reviewed, and the salient cytomorphologic features are described. Two types of cells were encountered in the aspiration smears; type I cells were undifferentiated while type II cells showed more differentiation and frequently revealed cytoplasmic processes that are probably indicative of early photoreceptor differentiation. Flexner-Wintersteiner rosettes characteristic of retinoblastoma were found in 10 of 16 cases. These findings were further correlated with ultrastructural examination of the tumors in nine cases. It is concluded that the presence of rosettes and type II cells with cytoplasmic processes are the two features that are most helpful in the FNAB diagnosis of retinoblastoma.

Aspiration cytology of neuroblastoma: light microscopy with transmission and scanning electron microscopic correlations.

Fine-needle aspiration biopsies from three patients with neuroblastoma were studied by light microscopy, and the morphologic findings were correlated with those from transmission and scanning electron microscopy. Light microscopic examination of the aspiration smears from all three cases revealed small and large round cells with variable numbers of intertwining cytoplasmic processes. Transmission electron microscopy confirmed the light microscopic finding of cytoplasmic processes; in addition, it revealed the presence of other diagnostic morphologic features, including neurosecretory granules, microtubules, and synaptic cell junctions. Scanning electron microscopy demonstrated that the tumors were composed of a mixture of undifferentiated round cells and more differentiated cells with long cytoplasmic processes. The morphologic spectrum of these processes and their interrelationships with one another and with other cells could be studied in detail. These findings indicate that scanning electron microscopy may be used effectively in the morphologic evaluation and pathologic diagnosis of neuroblastoma.

Surgical treatment of bilateral synchronous Wilms' tumors.

Definite progress has been made in the treatment of bilateral Wilms' tumors with marked improvement in the prognosis. This is confirmed in our series of 6 consecutive patients with synchronous tumors. The recent trend toward more conservative surgery, double or triple drug chemotherapy, and avoidance of high-dose radiation therapy has yielded good results.