Management of membranoproliferative glomerulonephritis: Evidence-based recommendations A L
Transcription
Management of membranoproliferative glomerulonephritis: Evidence-based recommendations A L
Kidney International, Vol. 55, Suppl. 70 (1999), pp. S-41–S-46 Management of membranoproliferative glomerulonephritis: Evidence-based recommendations ADEERA LEVIN Division of Nephrology, University of British Columbia, St. Paul’s Hospital, Vancouver, British Columbia, Canada Management of membranoproliferative glomerulonephritis: Evidence-based recommendations. Idiopathic membranoproliferative glomerulonephritis (MPGN) is one of the least common types of GN. This article critically evaluates the literature and generates evidence-based recommendations for the management of idiopathic MPGN. For all age groups, for idiopathic MPGN with normal renal function and asymptomatic nonnephrotic range proteinuria, no specific therapy is necessary (grades B and C). Close follow-up every three to four months, with specific attention to renal function, proteinuria, and blood pressure control, is recommended. In children with MPGN and nephrotic syndrome and/or impaired renal function, a trial of steroids is warranted (grade A). The best data suggest highdose, alternate-day steroids for a period of 6 to 12 months (40 mg/m2 on alternate days). If no benefit is seen, discontinuation with close follow-up and attention to conservative treatment (that is, blood pressure control, use of agents to reduce proteinuria, and correction of metabolic abnormalities) is recommended. In adults with MPGN, impaired renal function, and/ or nephrotic-range proteinuria, a trial of aspirin (325 mg daily), dipyridamole (75 to 100 mg tid), or a combination of the two for 12 months is reasonable (grade B). Again, if no benefits are seen, the treatment should be stopped. Attention to factors known to delay the progression of renal decline and close followup should be part of the treatment plan (grades B and C). Among all of the glomerulonephritides, membranoproliferative glomerulonephritis (MPGN) is one of the least common. It primarily affects children and young adults, with the mean age of onset ranging between 8 and 30 years. Conventionally, it has been classified as idiopathic or secondary, with idiopathic MPGN further subdivided into types I through III based on morphological pattern. This disease has had many different names over the years, including lobular, hypocomplementemic, and mesangiocapillary GN. There has been a decline in the incidence of primary MPGN over the two decades [1]. The diagnosis of MPGN requires the exclusion of all secondary causes such as hepatitis B or C, HIV, other infections, or collagen vascular disease (grade B). HepaKey words: idiopathic MPGN, secondary MPGN, hepatitis C, nephrotic syndrome, interstitial disease. 1999 by the International Society of Nephrology titis C has been increasingly implicated as a cause of MPGN over the last five years [2–4], and the association of MPGN with HIV disease has also been described [5]. Given the rising prevalence of these diseases, appropriate serology must be obtained in patients presenting with apparently idiopathic MPGN. Because there are a number of other noninfectious secondary causes with different treatment regimens and outcomes [2–8], it is important to conduct a thorough and focused search for secondary causes. The pathogenesis of MPGN has been well studied, and improved understanding has led to the search for agents that will modify the intense cellular proliferation and immune deposition that occurs. Immune-modulating therapy and therapy that interferes with the biological processes (for example, platelet activation) proposed to be central in the pathogenesis of the MPGN lesion have been evaluated in human trials [2]. The focus of this article is to review the clinical studies or trials in treatment of patients with idiopathic MPGN and to critically evaluate the quality of that data in order to make evidence-based recommendations for treatment. METHODS Human clinical trials, including adults and children, were reviewed from the English language literature from 1927 to 1997. Twenty-one studies were selected for review after an extensive MEDLINE search using the key words mesangiocapillary, membranoproliferative and lobular GN, clinical trials, and therapies. The articles selected include over 1100 patients with MPGN (all subtypes and age groups). Studies were evaluated based on the levels of evidence, graded 1 through 6 using the study design, size, power, and end points as criteria for evaluation. Recommendations were then graded based on the level of evidence of the supporting studies. Grades A through D are based on the number, type, and strength of studies available [9–12]. S-41 S-42 Levin: Membranoproliferative GN Table 1. Immune modulating therapy: Steroids, cyclophosphamide Level of evidence Author [Ref] Design N(Rx/C) Rx (dose) Duration 2 1 Tarshish [18] RCT 8(47/33) Prednisone (40 mg/m ) vs. placebo 130 months (mean) 1 Cattran [20] RCT 59(27/ 32) Cyclophosphamide, dypridamole and ASA vs. no therapy 18 months 3 Strife [26] UTC 17(16/1) Prednisone 2 mg/kg alt day 2 years 3 Davis [27] CT 27(19/8) 3 Orlowski [24] UCT 50 Prednisone and immunosuppressives (NS) P/Aza/CP/chlorambucil in combinations 3 Ford [23] UCT 19 Oral/i.v. Pred (2 mg/kg) 1 ACEI 3 Faedda [28] UCT 19 3 Bergstein [29] UCT 16 i.v./p.o. Cyclo and P (in different combinations) 6 3 alt day i.v. MP, then p.o. prednisone alt day 4 Habib [25] UCT 4 McEnery [22] CS 97(28/ 69) 6 4 Arslap [30] CS 4 Emre [31] 4 4 — 79 months 8–10 week initial therapy, then 3 2–3 years tapered dose 10 months Mean duration 37 months therapy Prednisone vs. placebo — Prednisone alt day 14 years 96 Prednisone or Cyclo 6 i.v. MP 10 year follow-up CS 15 Mean 27 months (6–84) Takeda [32] CS 18 i.v. Pulse MP 30 mg/kg alt days 3 9–15 days; then oral Pred 1 mg/kg 3 1 month, tapered off 3 1 month i.v. MP, Dp and oral Pred Itaka [33] CS 41 3 therapies: low dose Pred, low dose followed by high dose alt day Pred, high dose alt day Pred alone Median 6 years (3–15.5 years) 8 years Results/comments Children only, predominantly MPGN I stable renal function 61% (Rx) vs. 12% (C) No difference found, mixed MPGN I . II MPGN III only. Nephrotic range did worse, 3/16 developed RF No effect 10 year follow-up, reduce urine protein with triple drug therapy Children; 6.5 year follow-up, early therapy, shorter course, reduce glomerular proliferation 15/19 clinical remission, 7 year follow-up Mean follow-up 5 57 months, children, type I, all parameters of renal function, proteinuria improved after 3 months. Repeat kidney bx improved acute but worse chronic changes No effect Type II, children, serial renal bx: reduce proliferative changes 37% remission, 89% 10 year renal survival If Pred failed, then Cyclo 6 i.v. MP given with 25 and 50% remission; 10 year survival 61–81% 9/15 responsive, children significant reduction proteinuria Children, 94% renal survival at 4–10 years post-clinical onset; variable group: mild urinary abnormalities (12/18), heavy proteinuria (3/18), mild renal insufficiency (3/18) Children, identified by school screening 32 Type 1, 2 Type II, 7 type III; best outcomes 5 those with high dose alt day Pred; 1/41 ESRD? utility of early screening and therapy Abbreviations are: RCT, randomized control trial; CT, controlled trial; UCT, uncontrolled trial; CS, case series; DS, descriptive study; Rx, treatment group; C, control group; fn, function; NS, nonsignificant; N, number in study; P, prednisone; MP, methylprednisolone; i.v., intravenous; p.o., oral; alt day, alternative day; ASA, asprin; Dp, dipyridamole; Chlor, Chlorambucil; CP, cyclophosphamide; sign diff, significant difference; AZA, azathioprine. S-43 Levin: Membranoproliferative GN Table 2. Non-immune modulating therapy: ASA, dipyridamole Level of evidence Author [Reference] Design N (Rx/C) 1 Donadio [19] RCT 40 (21/19) 1 Zimmerman [17] RCT, cross over 18 (8/10) 1 Zauner [21] RCT 18 (9/9) Rx (dose) Duration Dipyridamole (275 mg/day and ASA (975 mg/day vs. placebo Warfarin (target INR 1.5–2) dipyridamole (75–100 mg qid) vs. placebo Results/comments 1 12 months 12 months (2 year study) ASA (500 mg/day) and dipyridamole (75 mg/day) (both got protein restriction and HTN control) 36 months t ⁄2 platelets and renal function. Rapid decline in control group, sign diff at 3–5 year, NS 10 year renal fn Protein restriction and HTN control standardized. Significant reduction in proteinuria, ns difference in renal function Significant reduction in proteinuria Rx group (1 g Uprotein vs. 8 g Uprotein control group P , 0.5) Abbreviations are in Table 1. Table 3. Natural history studies Level of evidence Author reference Design N Treatment Duration Rx/Fup Results/comments 3 3 Schmitt [6] Confalonerri [7] DS DS 220 294 — — 59 month mean follow-up 60 month mean follow-up 4 Bennett [8] DS 27 — — 4 Cameron [9] DS 104 — 65% 10 year survival 60% 10 year survival MPGN II, progressive disease 5 nephrotic syndrome and TI changes; C3 ? predictive Adults and children, Types I and II/ nephrotic Recommendations Recommendation 1. Treatment in adults and children with idiopathic MPGN is reserved for those patients with proteinuria of greater than 3 g/day, interstitial disease on biopsy, or impaired renal function (grades B and C). Evidence. The natural history of MPGN has been studied in a number of large European studies, which have shown a 60% 10-year renal survival for idiopathic MPGN. The level 4 natural history studies of Schmitt et al [6] and Confaloneiri et al [7], with over 500 patients followed, confirmed earlier findings that the 10-year renal survival of MPGN is approximately 60 to 65%. These historical reviews describe a large number of patients followed in Italy, Germany, United Kingdom, and Australia in an attempt to define adverse prognostic factors in patients with MPGN [13–16]. Nephrotic syndrome (heavy proteinuria) and the presence of interstitial disease are the main adverse prognostic signs [14, 15]. Recommendation 2. Children with idiopathic MPGN, proteinuria (more than 3 g/day), or impaired renal function may respond to high-dose steroid therapy, which should be maintained for 6 to 12 months (grade A). Recommendation 3. Adults with idiopathic MPGN, proteinuria (more than 3 g/day), or impaired renal function should undergo a trial of therapy with dipyridamole or aspirin (grade B). Evidence. Treatment studies have compared antiplatelet agents, coumadin, steroids, and other immuno- 8 years mean follow-up suppressive agents. They are varied in study design, duration of follow-up, and inclusion of patients with different types of MPGN. Furthermore, the studies vary in definition of successful treatment (reduction in proteinuria, renal survival, and regression of lesions on biopsy), often making comparisons difficult. However, specific therapy can be recommended in certain instances (Figure 1). Of the 13 treatment trials available in the literature, eight used steroids either alone or in combination with other treatment. Regimens included alternate-day steroids, intravenous boluses of steroids, and a combination of intravenous and oral therapy. Duration of treatment was variable, ranging from less than 6 months to over 130 months. This review considered changes in renal function, as well as reduction in proteinuria as objective and clinically meaningful end points. Only five randomized clinical trials have been published with sufficient power to determine the benefits of therapy in this rare disease (level 1 data) [17–21]. Tables 1, 2, and 3 provide an overview of the studies included, grouped according to type of therapy, and subgrouped according to the level of evidence. One of these five trials exclusively studied children and showed that prednisone (40 mg/m2 alternate days) when compared with placebo was of benefit in slowing the change in glomerular filtration rate (GFR) over time and stabilizing renal function in children with MPGN. Eighty children, predominantly with MPGN type 1, were S-44 Levin: Membranoproliferative GN Fig. 1. Algorithm for the treatment and follow-up of mesangial proliferative glomerulonephritis (MPGN). enrolled in the study, with a mean duration of treatment of 130 months: 61% of the treatment group versus 12% of the placebo group had stable renal function at the end of the study period (level 1 evidence) [18]. Other less well-designed studies have corroborated the finding that steroids are useful in the treatment of MPGN in children (level 3 to 4 evidence) [22–25]. The other four randomized, controlled studies were smaller, enrolled more heterogeneous groups of patients, including both children and adults, and had different end points (proteinuria, renal function measured using variable techniques, etc.). These randomized controlled trials are offered as level 1 and 2 evidence for the use of antiplatelet agents in adults. Donadio et al’s randomized 40 patients to examine the effect of dipyridamole and aspirin on renal outcome and the t1/2 of platelets. Both measures were improved by the antiplatelet therapy compared with placebo [19]. However, in the control group, the rate of renal deterioration was quite rapid and has led to concerns about the representativeness of the control group. Zauner et al’s 1994 study of 18 people, also treated with aspirin (ASA) and dipyridamole, compared therapy with no therapy over a 36 month follow-up. Both groups received similar protein restriction and antihypertensive regimens. The study demonstrated a clinically significant effect of reduction in proteinuria in the treatment group, but no change in GFR [21]. Levin: Membranoproliferative GN Another prospective study involved 18 patients over two study years (placebo and treatment), comparing warfarin and dipyridamole against placebo. The active treatment group showed reduced proteinuria but very little impact on GFR. An increased incidence of bleeding was noted in the treatment groups [17]. Cattran et al studied the effect of cyclophosphamide and coumadin and dipyridamole therapy in patients with MPGN in 59 patients. Therapy was continued for 18 months. These investigators were unable to show a difference between control and treatment groups after this length of time. They were also unable to define any predictors of renal decline, perhaps in part because all had nephrotic range proteinuria as part of the entry criteria to the study. The authors were careful to state that their study was able to address treatment issues clearly in only patients with MPGN type I, and did not have sufficient power to offer any conclusions for those with type II disease [20]. Thus, in 215 patients involved in robust randomized controlled trial (RCT) study designs (level 1 to 2 evidence), little benefit has been found in adults with any immunosuppressive therapy, but the data do suggest a possible benefit of antiplatelet therapy. Reduction of proteinuria has been used as a surrogate measure of success and may be an important clinical outcome in this patient group. Rigorous studies comparing high-dose steroids (the effective regimen for children) with ASA and dipyridamole in high-risk adult patients have not been performed. The remaining studies include uncontrolled trials, case series, and descriptive or comparative studies [22–33]. These studies suffer from weaknesses, which include variability in the different therapies used, the end points measured, and duration of therapy (Tables 1, 2, and 3). Of note, more recent studies in children with both mild urinary abnormalities and in those with nephrotic range proteinuria demonstrate that early high-dose immunosuppressive therapy (prednisone) appears to be effective in attenuating the course of renal disease, and argue for early aggressive therapy. These observations need to be validated in properly conducted, randomized controlled trials. As in other glomerular disease, the concomitant cellmediated interstitial infiltrate, which often occurs secondarily to the glomerular injury or is triggered by it, is probably responsible for ongoing renal decline. Thus, the prognosis for MPGN is adversely affected by the presence of heavy proteinuria, the presence of tubulointerstitial disease on biopsy, and hypertension [7]. In deciding who and when to treat, the clinician must consider the expected duration and outcome of the disease, as well as the risks and benefits of treatment of renal disease, and comorbidity that arises both from progressive renal dysfunction, and potentially from drug S-45 therapy. No study has addressed these particular issues in an objective format. SUMMARY The recommendations offered here are based on the best level of evidence in an attempt to rationalize the current therapy for idiopathic MPGN. Steroid therapy has been proven effective for children but not for adults. This situation is similar to that previously existing in the focal segmental glomerulosclerosis (FSGS) literature [30]. A number of unanswered general questions in this disease remain, which include the optimal proactive management of steroid complications, the proper dosing and tapering of medications, and the projected duration of the study that would be needed to revise the current therapeutic recommendations. A number of logistical problems are inherent in the study of a rare GN, especially one with a long natural history. Despite this, patients and their physicians do require guidance, and the review of evidence is important to guide practice and follow-up. These recommendations attempt to address those needs. Future studies will need to address shortcomings of previous data and will require collaborative efforts. Reprint requests to Adeera Levin, M.D., St. Paul’s Hospital, 1081 Burrard Street, Room 6010 A Providence Wing, Vancouver, BC V6Z 1Y6, Canada. E-mail: [email protected] REFERENCES 1. 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