Successful treatment of neonatal "autoimmune" thrombocytopenia with high-dose intravenous immunoglobulins.
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Adult↗
Biomedical subjects
F Rodeghiero
Publications and source records attributed to F Rodeghiero.
Treatment of thrombotic thrombocytopenic purpura with high-dose immunoglobulins. Results in 17 patients. Italian Cooperative Group for TTP.
BACKGROUND: The experimental observation that plasma from TTP patients sometimes exhibits a protein which can cause platelet agglutination, and that such agglutination can be inhibited in vitro by the use of IgG led some authors to treat plasma exchange-resistant TTP patients with high-dose IgG (HDIgG). METHODS: We report the results obtained with HDIgG treatment in 17 patients retrospectively examined by the Italian Cooperative Group for the study of TTP: 6 males and 11 females, mean age was 31.7 years for the women (range: 20-65) and 44.6 for the men (range: 26-66). In all cases HDIgG administration was combined with other treatment modalities. RESULTS: Of the 17 patients, 7 died from disease progression (41.1%), 2 achieved partial remission (11.7%) and the remaining 8 achieved complete remission (47%). Of the 10 cases (58.8%) with a positive response, only in 4 did the addition of HDIgG seem to produce significant improvement. All efforts made to characterize the subgroup of patients who responded to HDIgG and compare them with the non responders failed. CONCLUSIONS: Although our results do not unquestionably demonstrate the role of HDIgG in the treatment of TTP, they suggest a possible role for HDIgG in the treatment of those rare plasma exchange-resistant TTP cases.
Adult↗
Polycythemia vera and essential thrombocythemia in young patients.
BACKGROUND AND METHODS: Polycythemia vera (PV) and essential thrombocythemia (ET) in young patients are rarely reported. Their natural histories seem to differ from those of older patients and the best treatment is still uncertain. In this follow-up study we have evaluated a cohort of 64 consecutive patients younger than 40 to determine the incidence of thrombohemorrhagic events and the long-term outcome. RESULTS: Twenty-eight patients (20 M; 8 F) had PV, and 36 ET (21 F, 15 M). Mean follow-up was 8.2 years (range 4 months-16.7 years) in PV and 6.5 years (range 5 months-15 years) in ET. Thrombohemorrhagic symptoms were present at diagnosis in 10/28 patients (35%) with PV and in 12/36 patients (33%) with ET; during follow-up in 15/28 PV patients (53%) and in 13/36 ET patients (36%). Thrombotic events were the most frequent symptoms, both at diagnosis (52% in PV, 65% in ET) and during follow-up (43% in PV, 52% in ET). A total of 19/28 PV patients (67%) and 17/36 ET patients (47%) had thrombotic complications. Hemorrhagic complications at diagnosis were 4% and during follow-up 13% in PV, and 15% and 13% in ET. A total of 5/28 (18%) PV and 6/36 (17%) ET patients had hemorrhagic events. No laboratory parameter, including platelet count, was predictive of these events. Five PV patients had major thrombotic complications (18%). Four PV patients died (14%), 2 because of ANLL (7%), 2 because of thrombotic events (7%). Four ET patients experienced major complications, in three cases thrombotic (8.3%), in one hemorrhagic. No leukemic transformation occurred in ET and no ET patient died. CONCLUSIONS: In our experience, severe thrombohemorrhagic complications are present in young patients with PV and ET, which excludes young age as a favorable prognostic factor. Treatment also seems advisable for young patients and myelosuppressive treatment might be required. Prospective studies are urgently needed to assess the best treatment for this particular subset of patients.
Actuarial Analysis↗
Is ticlopidine a safe alternative to aspirin for management of myeloproliferative disorders?
BACKGROUND: Bleeding and thrombosis are frequent complications in patients with chronic myeloproliferative disorders (cMPD). Antiplatelet therapy is extensively used by many physicians for primary prophylaxis of thrombotic events, even though there have been no prospective trials that demonstrate clinical benefit. The use of aspirin has been associated with a heavy incidence of serious hemorrhages, particularly of gastrointestinal origin. This evidence is mainly based on data from patients treated with dosages far higher than those presently recommended. Moreover, patients with bleeding symptoms or prolonged bleeding time (BT) had not usually been excluded from treatment. METHODS: In this study 58 patients with cMPD and thrombocytosis were treated with aspirin (325-500 mg/day, 31 patients) or with ticlopidine (500 mg/day 27 patients). Only patients with negative bleeding histories and normal BT were considered. Ticlopidine, a drug not extensively investigated in cMPD, was reserved only for patients with histories of gastritis, gastric discomfort, peptic ulcer and/or intolerance to aspirin. All other patients were given aspirin combined with antacids in a buffered preparation. RESULTS AND CONCLUSIONS: Average follow-up was 2 years. Aspirin was associated with a high incidence of gastrointestinal hemorrhages (5/31). Ticlopidine was tolerated better and no bleeding complications were recorded. Both drugs were similarly effective in relieving erythromelalgia and painful paresthesia in almost all cases with these symptoms within 24-48 hours.
Adult↗
Twenty years experience with treatment of idiopathic thrombocytopenic purpura in a single department: results in 490 cases.
BACKGROUND: All patients with idiopathic thrombocytopenic purpura (ITP), diagnosed in our Department between June 1970 and December 1989, have been analyzed to evaluate the efficacy of treatment, incidence of spontaneous remission and see whether or not there are some parameters that may be indicative of the clinical outcome of these patients. PATIENTS AND METHODS: A total of 93 children and 397 adults have been evaluated. Response to treatment was any increase in platelet count to above 30 x 10(9)/1, when the platelet count was < 20 x 10(9)/l, or any doubling of the basal platelet count otherwise. Remission was defined as any platelet count higher than 100 x 10(9)/l lasting for 3 months or longer without treatment. For duration of the response and remission, the last check-up with a favorable platelet count was considered or the midpoint between the last favorable check-up and relapse. RESULTS AND CONCLUSIONS: In children, short-term responses to prednisone were obtained in 78% of acute patients and 67% of chronic patients, while stable remissions or long-term responses were found in 49% and 17% (p = 0.01). In adults, a short term response to prednisone was obtained in 71% of acute and in 72% of chronic patients, while stable remissions or long-term responses in 30% and 5% (p = 0.000003). In 40/45 children (89%), splenectomy induced a stable remission or a long-term response, while in adults, remissions or a long-term responses were achieved in 100/133 (75%) (p = 0.003). Age below 40 was found to be a significant determinant of a better response (p = 0.0013). Of the 92 evaluable adult patients with borderline thrombocytopenia, 8 developed a severe thrombocytopenia (9%). Spontaneous remissions or spontaneous rises to safe levels of platelet counts were recorded in 9% of adult cases presenting with severe thrombocytopenia: among refractory patients, spontaneous remission occurred significantly more frequently in younger patients (p = 0.003). An overall rate of 19% of adult patients continue to need therapy or have become refractory to any treatment. Among these cases, 7 died from cerebral hemorrhage, 6/7 were older than 50 years. Thus, the overall crude rate of thrombocytopenia-related death for our series of patients was 1.8%.
Adolescent↗
Treatment of idiopathic thrombocytopenic purpura (ITP) in patients with refractoriness to or with contraindication for corticosteroids and/or splenectomy with immunosuppressive therapy and danazol.
BACKGROUND: The best treatment for patients with idiopathic thrombocytopenic purpura (ITP) who are refractory to or have contraindications for splenectomy and corticosteroid remains uncertain. We report here our experience with vinca alkaloids (VA), azathioprine (Azp) and danazol in 33 such patients (6 M/27 F), median age 66 (23-83). PATIENTS: Group A (n = 19), Group B (n = 11), Group C (n = 17) patients were treated with VA, Azp and danazol. Fourteen patients were given more than one immunosuppressor agent. Sixteen patients were given 2 mg/week bolus infusions of vincristine (Vcr), while weekly slow infusions of vinblastine (Vnb, 0.1 mg/kg), for 2-4 weeks, were administered to the remaining 3 cases of Group A. Azp was administered at a daily dose of 150 mg for a median duration of 6 months. Danazol was administered at a median daily dose of 400 mg (400-800 mg), for a median length of 5 months. Response was defined as any increase of platelet count to higher than 30 x 10(9)/l, when platelet count was < 20 x 10(9)/l or any doubling of the basal platelet count otherwise. Remission, any increase of platelet count to higher than 100 x 10(9)/l lasting for 3 months or longer without therapy. RESULTS AND CONCLUSIONS: In Group A, there was a response rate of 63%, with 2 remissions (10%). All responses were observed after the first infusion. Two additional patients, who responded transitorily to VA, went into spontaneous remission 19 and 51 months after the last infusion of VA. In Group B, the response rate was 45%, with 1 remission (9%). The response was never observed before one month. One additional patient went into spontaneous remission 60 months after stopping Azp. In Group C, the response rate was 56% with 2 remissions (12%); 2 patients relapsed while on therapy, 4 continue to require therapy and 1 died from a stroke while on therapy. Four patients in Group A and two in Group B discontinued the therapy because of severe side effects. Danazol was generally well tolerated but for one patient was interrupted after only 5 days because of severe dyspepsia. In conclusion, the clinical usefulness of VA and Azp is very limited and burdened by severe side-effects. Danazol seems to be safer but no more effective and its long-term toxicity is not known. There were two hemorrhagic deaths in this series of patients.
Adrenal Cortex Hormones↗
Adverse reactions after high-dose intravenous immunoglobulin: incidence in 83 patients treated for idiopathic thrombocytopenic purpura (ITP) and review of the literature.
BACKGROUND: High dose intravenous immunoglobulin (h.d. Ig) is increasingly used in several hematological diseases. There are few data on the safety and no review of side effects after this treatment is available. We review here the literature on reported adverse reactions after h.d. Ig and report our own experience in the management of 83 patients with idiopathic thrombocytopenic purpura (ITP). PATIENTS AND METHODS: Computer assisted (Medline) and manual searches of the cumulative Index Medicus were undertaken to retrieve articles reporting side effects after infusion of h.d. Ig, published between 1981 and August 1993. Our own experience is based on clinical records of 83 patients, 56 females, 27 male, with a median age of 61 years (range 8-84), treated with h.d. Ig for acute or chronic ITP between 1985 and June 1993. RESULTS AND DISCUSSION: Major adverse reactions included aseptic meningitis (14 cases), hemolytic anemia (8 cases) and renal dysfunction (12 cases). The most frequently reported mild side effect was headache. We have had 2 cases of severe skin reactions, 1 case of acute renal failure, 3 cases of severe headache and 1 case of chills and fever. For other 3 cases, an anaphylactic/oid reaction, caused by a too rapid infusion rate, occurred. Thus, 10/83 (12%) patients, 7 female and 3 male, developed side effects, but they were unpredictable and severe in only 3 cases (4%). Side effects developed only in 21/480 (4%) infusions.
Acute Kidney Injury↗
The clinical significance of the antiplatelet antibody test based on results for 265 thrombocytopenic patients.
BACKGROUND: The usefulness of measuring antiplatelet antibodies by ELISA or cytofluorimetric techniques for the diagnosis of idiopathic thrombocytopenic purpura is still uncertain. METHODS: We evaluated the clinical significance of two widely applicable antiplatelet antibody tests for a series of 265 patients evaluated consecutively in our Department for the diagnosis or follow-up of thrombocytopenia. Flow cytofluorimetry was used to measure platelet-associated immunoglobulins (PAIg) and the enzyme-linked immunosorbent assay (ELISA) was used to detect antiplatelet antibodies in patients sera (S-PBIg). The significance of antiplatelet antibody testing was addressed by studying the factors that influence test positivity, the diagnostic value of a positive test and the prognostic implication of a positive test. RESULTS: The platelet count was found to be strongly associated with positive PAIg or S-PBIg (P < 0.001), while neither splenectomy nor corticosteroid treatment affected PAIg or S-PBIg positivity. Both PAIg and S-PBIg were limited diagnostic value for the differential diagnosis of idiopathic thrombocytopenic purpura (ITP) from secondary thrombocytopenia for patients with platelet counts between 25,000 and 100,000 platelets/microliters, but the percentages of misclassified patients based on only the PAIg or S-PBIg test were 32 and 54%. We found no relationship between PAIg and/or S-PBIg at time of diagnosis and the patient's clinical response to corticosteroid therapy. CONCLUSIONS: We conclude that antiplatelet antibodies are strongly correlated with platelet counts, discriminate poorly between ITP and secondary thrombocytopenia and have negligible prognostic value. Therefore, we do not recommend performing antiplatelet antibody tests as a routine laboratory test in the diagnostic workup of thrombocytopenia.
Adolescent↗
Adult patients with the nephrotic syndrome: really at high risk for deep venous thromboembolism? Report of a series and review of the literature.
BACKGROUND: The reported incidence of thromboembolic episodes in people with nephrotic syndrome (NS) ranges from 6% to 44% and it has been ascribed to the presence of a hypercoagulable state, as suggested by abnormalities of several hemostatic parameters in these patients. However, the results of the studies are often contradictory and fragmented and are rarely based on prospective studies. To assess the incidence of venous thrombosis and the pattern of abnormalities of the hemostatic parameters in NS, we planned a prospective study of a group of patients with NS. PATIENTS AND METHODS: Thirty-six consecutive patients with NS were enrolled during a 9-month period. Every 4-6 months, clinical history was collected and physical examination was carried out. Blood samples for laboratory investigation were taken at entry into the study and 24 months later. A critical review of the literature was also carried out (Medline database and Current Contents). RESULTS: During the follow-up (mean 45.2 months), no thrombotic symptoms were recorded, PTT, PT, TT and mean AT-III levels were within the normal range, whereas fibrinogen, plasminogen, protein C and S, heparin cofactor II levels were significantly higher than normal both at entry into the study and 24 months later. No patient was positive for antiphospholipid antibodies. Slightly decreased levels of AT-III and heparin cofactor II were found in only two cases. CONCLUSIONS: Our study confirms neither the high incidence of thrombotic complications in NS nor the presence of abnormalities of hemostatic parameters commonly associated with venous thromboembolism.
Adolescent↗
Acquired plasma factor XIII deficiencies.
Coagulation factor XIII (FXIII) is of paramount importance in the process of fibrin stabilization, which is the final step of the coagulation cascade. The clinical significance of defective fibrin stabilization is highlighted by the severe hemorrhagic manifestations of congenital FXIII deficiency. In this paper we review the pathophysiology, clinical presentation and therapy of acquired plasma FXIII deficiencies, caused by specific inhibitors or associated with other clinical conditions. For acquired severe FXIII deficiency caused by factor-specific inhibitors, the need for prompt diagnosis and treatment is emphasized by the high hemorrhagic risk and mortality. For moderate reduction of FXIII secondary to other conditions, we discuss the relative importance of FXIII reduction in the development of clinical symptoms and the role of substitution treatment.
Acute Disease↗
Fibrinogen survival and fibrinopeptide A in acute leukemia.
BACKGROUND. Hypofibrinogenemia and increased fibrin(ogen) degradation products in acute leukemia have been attributed to intravascular thrombin generation triggered by leukemic cells. However, the strict relationship between fibrinogen catabolism and turnover of fibrinopeptide A (FPA), which is a sensitive and specific marker of thrombin activity, has not been evaluated in acute leukemia (AL) with or without disseminated intravascular coagulation (DIC) to see whether mechanisms other than thrombin activity could be responsible for fibrinogen consumption. We report here the 125I-fibrinogen kinetics and FPA measurements in 19 patients with AL, 6 of them with DIC. METHODS AND RESULTS. Radiolabelled fibrinogen kinetics were studied in all the patients concomitantly with the start of chemotherapy. Fibrinopeptide A was measured by a radioimmunoassay at time of diagnosis and during chemotherapy. The kinetics of disappearance of radiolabelled fibrinogen where biphasic, with a rapid phase in the first 1-3 days of chemotherapy and a subsequent slow phase associated with the reduction or disappearance of blast cells. Patients with DIC had a significantly shorter half-life and turnover than patients without DIC. The latter group had significant shortening of these parameters in comparison to normal subjects. The thrombin-dependent catabolic rate of fibrinogen, calculated from the mean level of FPA during the first phase of disappearance curve and by assuming 2 moles of FPA generated per mole of fibrinogen, was similar in patients without DIC and in normal subjects, whereas patients with DIC had a significantly higher catabolic rate, even though the increase was not sufficient to account for all the turnover of fibrinogen. No relationship was observed between fibrinogen turnover and FPA turnover.
Acute Disease↗
Erwinia- and E. coli-derived L-asparaginase have similar effects on hemostasis. Pilot study in 10 patients with acute lymphoblastic leukemia.
BACKGROUND: L-asparaginase (L-ase) affects the synthesis of several hemostatic factors, including the naturally occurring clotting inhibitors antithrombin III, protein C and S, and thromboembolic events are a well-recognized complication of the hypercoagulable state resulting from this treatment. Recently, in addition to the widely used E. Coli-derived L-ase, a new L-ase derived from Erwinia Carotovora has been introduced into clinical studies. This formulation seems to have lesser side-effects, but data on its effects on the hemostatic system are very limited. We report here the hemostatic changes observed in 10 adult patients with acute lymphoblastic leukemia (ALL) treated with Erwinia- or with E. Coli-derived L-ase. METHODS AND RESULTS: The decreases (percentages of basal level) of antithrombin III, protein C and plasminogen (functional assays) were similar in both groups of patients (about 50% of the basal value 6 or 8 days after starting L-ase) and so were the number of instances in which the level of these proteins fell below 70% of normal. On the contrary, the fibrinogen level (PT-derived method) was less severely decreased by Erwinia L-ase (34% of basal value for E. Coli L-ase versus 58% for Erwinia L-ase; P = 0.048) and there were more instances in which the fibrinogen level was less than 100 mg/dL than with E. Coli L-ase (40% versus 16%; P = 0.051). Thrombin-AT-III complexes (ELISA) showed similar pattern during both treatments. Two patients treated with Erwinia L-ase had moderate hyperglycemia, requiring insulin treatment but not L-ase discontinuation. No allergic reaction or pancreatitis was observed. A patient in the Erwinia L-ase-treated group developed deep venous thrombosis on day 7 after the start of L-ase and was treated with subcutaneous heparin (12,500 U x 2/day), with prompt improvement. CONCLUSION: This study shows that the two L-ase formulations have similar effects on hemostasis. Patients treated with Erwinia L-ase may develop thrombotic symptoms, as already reported for E. Coli-L-ase.
Adolescent↗
Hematologic and cytogenetic analyses of 99 cases of de novo myelodysplastic syndromes.
BACKGROUND. The myelodysplastic syndromes (MDS) are a heterogeneous group of clonal hematopoietic stem cell disorders characterized by peripheral pancytopenia, abnormal bone marrow differentiation and a high risk of leukemic transformation. The role of karyotypic abnormalities in the pathogenesis of MDS is still unclear. In this paper we present the hematologic and cytogenetic findings in 99 patients with de novo MDS. RESULTS AND CONCLUSIONS. Fifty-three cases had chromosomal abnormalities nor complex karyotypes were associated with the morphologic subsets described by the FAB criteria. Nevertheless, the karyotypic profile is prognostically important, as indicated by the fact that patients with complex karyotype abnormalities have very short survival.
Adult↗
Single step immunophenotyping of acute leukemias not classifiable by standard morphology and cytochemistry: a practical approach.
BACKGROUND: Immunophenotyping is at present a useful aid and often an essential step for correct diagnosis of acute leukemia and for this purpose some investigators have proposed several immunomarker panels. This approach is particularly important in the differential diagnosis of acute leukemias not classifiable by standard morphology and cytochemistry. METHODS: We have tested peripheral blood and/or bone marrow samples from 125 patients not classifiable by FAB criteria. In all the cases, the reactivities of the same panel of 17 monoclonal antibodies were analyzed by flow cytometry, using both single and double fluorescent labeling. RESULTS: Of the 125 patients investigated, 75 (60%) were classifiable as ALL, 58 as B-lineage ALL and 17 as T-lineage ALL; 33 (26.4%) as AML, of which 2 M7; 6 (4.8%) as biphenotypic and 11 (8.8%) as immunophenotypically undifferentiated. CONCLUSIONS: From a critical analysis of our cases and a review of the literature, we suggest that a panel of 9 monoclonal antibodies (CD2, CD5, CD7, CD10, CD19, CD20, CD13, CD33, CD41), is sufficient for reliable, rapid and reasonably low cost typing of acute leukemia, useful for an immediate therapeutic decision.
Acute Disease↗
Fulminant sepsis in adults splenectomized for Hodgkin's disease.
BACKGROUND AND METHODS: Laparotomy with splenectomy still remains important for staging Hodgkin's disease (HD). The risk of fulminant sepsis (FS) after splenectomy is well known, but the incidence of FS in splenectomized HD adult patients has not been accurately assessed. In this study we have tried to assess this risk and its duration and to evaluate the role of HD "per se" in causing FS. RESULTS: Six cases of FS were traced in a group of 226 splenectomized adults, with a crude incidence of 2.65%. Age at the time of the event ranged from 23 to 41 years and time after splenectomy from 46 to 98 months. Four patients were disease-free when sepsis occurred. In 4 cases the causative agent was isolated (3 Streptococcus Pneumoniae, 1 Streptococcus alpha Haemolyticus). The mortality rate was 66%, while net probability of death (life table) at 10 years was 2.6%. M/F rate was 0/6 (P = 0.01). The incidence of FS was 0.33 cases per 100 patient-years (I.C. 95% = 0.12-0.72). There seems to be no relationship to histological type, clinical stage or age at splenectomy. No case of sepsis occurred in a control group of 281 non-splenectomized HD adults (P = < 0.01), despite the more advanced disease present in these cases on the average. CONCLUSIONS: The frequency of FS, the causative agents, the mortality rate, the duration of risk are similar to those previously reported. Prompt treatment of any febrile disease in HD splenectomized patients and a policy of antipneumococcal (and possibly of anti-meningococcal) vaccination seem advisable.
Adolescent↗
Acute renal failure after high-dose intravenous immune globulin in a patient with idiopathic thrombocytopenic purpura.
High-dose intravenous immunoglobulin (IVIG) is considered a safe and efficacious treatment for patients with a variety of hematological and non hematological diseases, including patients with idiopathic thrombocytopenic purpura (ITP) not responsive or with contraindications to corticosteroids and/or splenectomy. Side-effects are usually minor, but severe reactions have been rarely reported. We describe the case of a patient with ITP, without pre-existing renal disease, who developed a severe transitory acute renal failure following administration of IVIG, promptly reversed after hemodialysis.
Acute Kidney Injury↗
Therapeutic impact of adult-type acute lymphoblastic leukemia regimens in B-cell/L3 acute leukemia and advanced-stage Burkitt's lymphoma.
BACKGROUND: Adult B/L3-ALL is a rare disease subset characterized by an aggressive clinical course and a poor response to conventional adult ALL-type chemotherapy. Recent data from the GMALL Group showed that prognosis can be improved with an innovative treatment regimen. In the current retrospective survey we focus on therapeutic results obtained at our Institutions during a 15-year period with ALL-type regimens in 34 adults with either B/L3-ALL or advanced-stage Burkitt's lymphoma. METHODS: Five successive ALL treatment programs were developed. They included a homogeneous induction phase with early intrathecal chemoprophylaxis, multidrug postremission consolidation followed by cranial irradiation (4 trials), high-dose chemotherapy plus autografting (2 trials), late consolidation (2 trials), and variable-length maintenance (4 trials). Early response and prolonged disease-free survival rates were analyzed according to selected clinical and therapeutic variables. RESULTS: Overall, a complete remission was achieved in 62%, with a median duration of 1.6 years and a 10-year remission rate of 49%. A diagnosis of B/L3-ALL (p = 0.007), the use of idarubicin instead of adriamycin during induction (p = 0.018), a serum creatinine < 1.6 mg/dL, and an uninvolved central nervous system were associated with higher response rates. As regards long-term disease-free survival, results were significantly better in patients with < 1 x 10(9)/L L3/blast cells in the peripheral blood (p = 0.0029) and/or aged < 50 years (p = 0.04), and in those consolidated with the most recent rotational high-dose plus peripheral blood stem cell autotransplant regimen. CONCLUSIONS: According to the results presented, ALL-like regimens may still represent a worthwhile therapeutic choice. The use of idarubicin during induction, the prognostic subclassification of patients, a careful control of dysmetabolic complications, the selection of the proper chemo-radioprevention for meningeal disease and perhaps the introduction of high-dose chemotherapy supported by autologous stem cell rescue appear to be the mainstay of further improvements.
Adolescent↗
Mobilization and collection of PBSC in healthy donors: a retrospective analysis of the Italian Bone Marrow Transplantation Group (GITMO).
BACKGROUND AND OBJECTIVE: The number of allogeneic transplants of peripheral blood stem cells (PBSC) is rapidly increasing. Collection of PBSC in healthy subjects currently implies the administration of G-CSF or GM-CSF and, of course, the use of apheretic devices. These procedures involve potential risks, in particular the risk of leukemia secondary to growth-factor treatment. To evaluate the current practice of PBSC mobilization and collection, and initially assess the short-term side effects and efficiency of procedures, the GITMO (Gruppo Italiano Trapianti di Midollo Osseo) promoted a retrospective cooperative study among the Italian centers. METHODS: Seventy-six healthy individuals donating to their HLA-identical or partially matched sibling recipients in seven Italian centers form the basis of the present analysis. The data were retrospectively collected by proper forms, pooled and analyzed by means of a commercially available statistical soft package. RESULTS: All donors received G-CSF as mobilizing agent with different schedules according to each single center policy. A median of 2.5 (range 1-4) aphereses per donor were run. The most frequent side effect was bone pain. In no case did the medium term follow-up reveal subjective complaints or laboratory modifications. After G-CSF mobilization, WBC and lymphocytes counts increased to a maximum of (mean +/- SD) 48.1 +/- 15.6 x 10(9)/L and 4.2 +/- 1.5 x 10(9)/L, respectively. The peak was reached on day 5 in both cases. Platelets decreased after the apheretic procedures, reaching a minimum of (mean +/- SD) 77 +/- 26 x 10(9)/L on day 8 and returning to normal values on day 11. Overall, the apheretic collection yielded (mean +/- SD) 18.6 +/- 19.2 x 10(8)/kg donor body weight MNC; 10.4 +/- 5.7 x 10(6)/kg CD34+ cells; 90.6 +/- 75.9 x 10(4)/kg CFU-GM and 4.3 +/- 1.8 x 10(8)/kg CD3+ cells. The target dose of 4 x 10(6)/kg CD34+ cells was harvested in 51.3% donors after a single apheresis, in 85.5% after the second, and in nearly 100% after a maximum of 3 aphereses. INTERPRETATION AND CONCLUSIONS: These data demonstrate that collection of adequate numbers of circulating progenitors is feasible and well tolerated in healthy donors. However, only careful monitoring of donors and international cooperation will help to definitively assess the long-term safety of G-CSF for mobilization of PBSC.
Adolescent↗