Alpha-interferon therapy induces improvement of platelet counts in children with chronic idiopathic thrombocytopenic purpura.

PURPOSE: To investigate alpha-interferon (IFN) therapy for children with chronic idiopathic thrombocytopenic purpura (ITP). PATIENTS AND METHODS: Patients with refractory ITP lasting more than 12 months from diagnosis were included if they had platelet counts <50 x 10(9)/L and had received no treatment during the past month. Patients received IFN (3 x 10(6) U/m2 per dose), three times per week for 4 weeks; if partial (<150 x 10(9)/L) or no response was obtained, the same dose was continued for another 8 weeks. In patients with favorable response and subsequent decrease to pre-treatment values, an additional 4 weeks of treatment could be administered. RESULTS: Fourteen patients (ages 4-20 y) receiving 17 IFN courses were included. Mean initial platelet count was 29 +/- 15 x 10(9)/L. A significant increase was achieved during 14 of 17 courses (82.4%). All but two responses were transitory, and platelets returned to initial values after IFN discontinuation (mean 44 +/- 26 days). Considering the best response achieved by each patient, we observed: 1) 10 patients who achieved a sustained improvement of platelet count throughout the treatment period, decreasing to initial values after therapy was stopped; 2) one patient who achieved platelet count >150 x 10(9)/L, remaining with normal platelets at 18 months; 3) one patient who achieved platelet count >150 x 10(9)/L, remaining with platelets between 100 and 140 x 10(9)/L at 48 months; 4) one patient who had no response; and 5) one patient in whom therapy worsened the thrombocytopenia. A mild to moderate flu-like syndrome and a moderate decrease of the absolute neutrophil count were the only side effects observed. CONCLUSION: Interferon therapy induces a significant increase of platelet count and seems to be a valid alternative therapy to attempt the achievement of prolonged remission in refractory ITP, to defer splenectomy in younger children, or to improve platelet count before planned splenectomy.

Low levels of serum erythropoietin in children with endemic hemolytic uremic syndrome.

Serum erythropoietin (EPO) levels were measured in ten previously non-transfused children with hemolytic uremic syndrome (HUS). Complete blood cell count, serum EPO, and renal function tests were carried out upon admission and weekly thereafter. Blood samples were obtained: (1) prior to the first transfusion; (2) after the first transfusion but before recovery from renal failure; (3) during the recovery stage. All patients required transfusions (mean 1.8+/-0.8 per child). Absolute values of EPO correlated positively with the hematocrit during the three stages (r = 0.53, 0.36, and 0.12, respectively) which is opposite to expected results. The observed EPO logarithm/predicted EPO logarithm upon admission was low (0.70+/-0.08), falling further during stage 2 (0.57+/-0.03), but increasing thereafter (0.78+/-0.07) without reaching normal values. The reticulocyte production rate followed a parallel course (0.74+/-0.14, 0.54+/-0.11, and 0.60+/-0.10, respectively). On comparing the observed serum EPO levels with those expected, 9 of 11 pre-transfusion samples showed low values; in stage 2, all samples were below normal; in the recovery phase most (77.8%) were still low. Our results show an inadequate EPO synthesis in children with HUS, which could play an important pathogenic role, since it aggravates the severity of the existing hemolytic anemia; the secondary inhibitory effect of repeated transfusions exacerbates this inadequate synthesis.

Correction of iron deficiency with an iron-fortified fluid whole cow's milk in children: results of a pilot study.

PURPOSE: This study assesses the efficacy of an iron-fortified (15 mg Fe, as stabilized ferrous sulfate (SFE-171), per liter) fluid whole cow's milk (IFFWCM) for the treatment of mild iron deficiency in children. Previous studies in healthy adult volunteers showed a mean 10.2 +/- 4.7% iron absorption. PATIENTS AND METHODS: Seventeen children (12 to 48 months old) with iron deficiency (serum iron (SI) < 60 micrograms/dl, transferrin saturation (TS) < 15%, serum ferritin (SF) < 15 ng/ml) were included in this study; 11 of them were anemic. As treatment, they received IFFWCM, instead of the customary whole cow's milk, for at least 4 months; medicinal iron was not administered. Hematocrit (Hct), hemoglobin (Hb), SI, TS, and SF were determined monthly. RESULTS: The Hb increased from 10.3 +/- 0.8 to 12.7 +/- 0.6 g/dl in the group with anemia (delta F-B: 2.4 +/- 1.0 g/dl) and from 12.6 +/- 0.7 to 13.5 +/- 0.3 g/dl in the group without anemia (delta F-B: 0.9 +/- 0.5 g/dl); the difference between both groups was significant (p < 0.01); the rate for Hct values showed a similar pattern. In the whole group, the SI increased to 84.8 +/- 37.4 micrograms/dl, with no difference between children with anemia and children without anemia; TS showed a similar pattern (delta F-B: 19.0 +/- 11.0%). The mean SF increased from 12.1 +/- 2.7 ng/ml to 27.9 +/- 25.4 ng/ml. Normal values for Hct, Hb, SI, and TS were reached by 100% of children; the rate for SF was 56.3%. Time required to reach normal Hct in the children with anemia was 59.4 +/- 33.0 days. Acceptance and tolerance were excellent; no treatment had to be discontinued. The group of patients with anemia was compared with an historical group composed of 55 children matched for age, basal Hct, and achieved Hct increase, treated with medicinal FS (4-6 mg/kg/day): time required to reach normal Hct was shorter in the FS-treated group (39.0 +/- 14.5 days) (p = 0.050). CONCLUSION: The use of IFFWCM alone could be an effective, relatively inexpensive, and well-tolerated treatment of iron deficiency in children.