Treatment of Pediatric Idiopathic Pulmonary Hemosiderosis with Low-Dose Cyclophosphamide

2003 ◽  
Vol 37 (11) ◽  
pp. 1618-1621 ◽  
Author(s):  
Shiou-Huei Huang ◽  
Ping-Yu Lee ◽  
Chen-Kuang Niu
Keyword(s):  
Platelet Count ◽  
Immunosuppressive Therapy ◽  
Low Dose ◽  
Dosing Regimen ◽  
Safety And Efficacy ◽  
Cyclophosphamide Therapy ◽  
Pulmonary Hemosiderosis ◽  
Long Term Treatment ◽  
Idiopathic Pulmonary Hemosiderosis

OBJECTIVE: To report the safety and efficacy of long-term, low-dose cyclophosphamide therapy in a child with idiopathic pulmonary hemosiderosis (IPH). CASE SUMMARY: A 7-year-old boy diagnosed with IPH 4 years previously was initially prescribed prednisolone. Because he only had a transient response to prednisolone, oral cyclophosphamide 2 mg/kg/d was later added. A dramatic improvement was noted during the subsequent follow-up. One year after cyclophosphamide therapy, the patient suddenly developed thrombocytopenia (platelet count 75 times 103/mm3), with the platelet count decreasing to 10 times 103/mm3 over the following 10 months. Cyclophosphamide was tapered to an alternating daily dosage of 1 mg/kg. The tapering resulted in a subsequent increase in the platelet count, which was maintained between 20 and 50 times 103/mm3 without occurrence of petechiae or spontaneous bleeding. Under this reduced dosing regimen, the disease has remained in remission for >1 year. DISCUSSION: Due to the low prevalence of IPH, only limited data document the safety and efficacy of immunosuppressive therapy in treating this disease. Although our patient showed a good response to low-dose cyclophosphamide, he developed thrombocytopenia with its use. The mechanism is unclear, but it may be similar to that of high-dose cyclophosphamide-induced myelosuppression. Due to the development of thrombocytopenia, the use of cyclophosphamide was maintained under a reduced dosing regimen. The benefit of long-term immunosuppressive therapy is controversial, and more clinical evidence is required to support its continued usage. CONCLUSIONS: Long-term, low-dose cyclophosphamide is effective in treating childhood IPH, but caution should be exercised due to the possible development of thrombocytopenia. Periodic monitoring of the platelet count in long-term treatment is recommended.

scholarly journals Successful Long-term Treatment with Once-daily Injection of Low-dose Octreotide in an Aged Patient with Insulinoma

2005 ◽  
Vol 52 (5) ◽  
pp. 629-634 ◽  
Author(s):  
Takuyuki KATABAMI ◽  
Hiroyuki KATO ◽  
Naoko SHIRAI ◽  
Satoru NAITO ◽  
Nobuhiko SAITO
Keyword(s):  
Low Dose ◽  
Term Treatment ◽  
Daily Injection ◽  
Aged Patient ◽  
Once Daily ◽  
Long Term Treatment

Immunosuppressive therapy is more effective than interferon in neuromyelitis optica

2007 ◽  
Vol 13 (2) ◽  
pp. 256-259 ◽  
Author(s):  
C Papeix ◽  
J-S Vidal ◽  
J De Seze ◽  
C Pierrot-Deseilligny ◽  
A Tourbah ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Immunosuppressive Therapy ◽  
Neuromyelitis Optica ◽  
Interferon Beta ◽  
Term Treatment ◽  
Therapeutic Options ◽  
Long Term Treatment

To determine long-term treatment (LTT) of neuromyelitis optica (NMO), we retrospectively reviewed therapies of 26 patients with NMO followed in five French neurological departments. To assess LTT efficacy, the probability of relapse free after LTT was analysed. Patients were divided into two groups according to the first treatment receiving interferon beta (IFN Group, seven patients) or immunosuppressants (IS Group, 19 patients). The probability of relapse was significantly lower in the IS Group (P = 0.0007). From our results, interferon beta is not recommended, and one of the best current therapeutic options for NMO appears to be immunosuppressants. Multiple Sclerosis 2007; 13: 256–259. http://msj.sagepub.com

Long-Term Treatment with Romiplostim in Patients with Chronic Immune Thrombocytopenic Purpura (ITP): 3-Year Update from An Open-Label Extension Study

Blood ◽  
2008 ◽  
Vol 112 (11) ◽  
pp. 402-402 ◽  
Author(s):  
David J Kuter ◽  
James B Bussel ◽  
Adrian Newland ◽  
Joost TM de Wolf ◽  
Troy Guthrie ◽  
...  
Keyword(s):  
Platelet Count ◽  
Term Treatment ◽  
Duration Of Treatment ◽  
Open Label ◽  
Platelet Counts ◽  
Chronic Itp ◽  
Long Term Treatment ◽  
Open Label Extension ◽  
Grade 3

Abstract Chronic ITP is characterized by increased platelet destruction and suboptimal platelet production. Romiplostim is an investigational Fc-peptide fusion protein (peptibody) being studied for its ability to increase platelet counts in patients with chronic ITP. We report data from an open-label extension study of romiplostim in adult patients with chronic ITP. Collection of safety and efficacy data from long-term treatment of these patients is ongoing. Eligible patients had completed a prior romiplostim study and had platelet counts □50×109/L. Romiplostim was administered subcutaneously once weekly with dose adjustments to maintain a platelet count of 50–250×109/L. As of July 13 2007, 142 patients had been treated with romiplostim. Their median time since diagnosis was 6.4 years (range 0.6–46.4 years). Most were female (67%) and had previously undergone a splenectomy (60%). The median baseline platelet count was 17×109/L (range 1–50×109/L). The median duration of treatment was 65 weeks (range 1–156 weeks). Twenty-nine (20%) patients discontinued the study, 10 (7%) due to adverse events (AEs) [2 each of bone marrow reticulin and thrombosis; 1 each of bleeding, pain, cardiac arrest, pneumonia, hepatic and renal failure, and monoclonal gammopathy of undetermined significance]. Different measures of platelet count response were analyzed; any platelet counts within 8 weeks of receiving rescue medications were excluded from these analyses. Platelet counts were increased from baseline by ≥20×109/L more than 80% of the time in 54% of patients and more than 50% of the time in 73% of patients. Platelet counts remained above 20×109/L more than 90% of the time in 67% of patients and more than 50% of the time in 94% of patients. A platelet count >50×109/L and double baseline was achieved by 30% (42/138) of patients after the first dose, by 51% (71/138) of patients after the third dose, and by 87% (124/142) of patients overall. The durability of platelet count increases was analyzed: platelet counts >50×109/L were sustained for ≥10, ≥25, and ≥52 consecutive weeks in 78% (102/131), 54% (66/122), and 35% (29/84) of patients, respectively. The patient incidence of bleeding events both of any severity and of clinical significance (≥Grade 3) declined over time (Table). AEs were reported in 95% of patients, with most mild to moderate in severity. The most common were headache (37%); nasopharyngitis (32%); and contusion, fatigue and epistaxis (each 30%). AE frequency did not increase with time on study (Table). Bone marrow reticulin was present or increased in 8 patients with no evidence of progression to collagen fibrosis or chronic idiopathic myelofibrosis. Thrombotic events were reported in 7 (5%) patients; 6 had pre-existing risk factors for thrombosis. In conclusion, romiplostim increased platelet counts in most patients for most of the time, and clinically relevant bleeding was reduced over time. Romiplostim was well-tolerated and AEs did not increase with longer duration of treatment. Table. Summary of patient incidence of AEs by study period <24 wks (N=142) n (%) 24 to <48 wks (N=126) n (%) 48 to <72 wks (N=97) n (%) 72 to <96 wks (N=65) n (%) 96 to <120 wks (N=29) n (%) 120 to <144 wks (N=25) n (%) AEs 129 (91) 110 (87) 64 (66) 36 (55) 23 (79) 21 (84) Serious AEs 25 (18) 13 (10) 7 (7) 4 (6) 4 (14) 1 (4) Treatment-related AEs 48 (34) 14 (11) 12 (12) 7 (11) 4 (14) 3 (12) Treatment-related serious AEs 6 (4) 3 (2) 1 (1) 2 (3) 1 (3) 1 (4) Study withdrawals due to AEs 4 (3) 5 (4) 0 (0) 0 (0) 0 (0) 1 (4) Bleeding any grade 60 (42) 37 (29) 22 (23) 13 (20) 11 (38) 8 (32) Bleeding ≥ Grade 2 (moderate) 25 (18) 12 (10) 8 (8) 4 (6) 3 (10) 2 (8) Bleeding ≥ Grade 3 difference in thisresponsebetween refractory (severe) 9 (6) 1 (1) 1 (1) 1 (2) 0 (0) 0 (0)

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